Whenever Harry Wuest has a doctors appointment in northern Atlantas hospital cluster dubbed Pill Hill, he makes sure to stop by the office of Dr. Douglas Doug Murphy for a quick chat.

And Murphy, unless hes tied up in the operating room, always takes a few minutes to say hello to his former patient. Remember when . . . ? is how the conversation typically starts, and its always tinged with laughter, often joyful, sometimes bittersweet.

Its a reunion of two men who shaped a piece of Georgias medical history.

Almost 35 years ago, Murphy opened the chest of Wuest and sewed in a new heart, giving him a second shot at life. Wuest was the third heart transplant patient at Emory University Hospital.

Tall, lanky, with short curly hair and a quiet demeanor, Wuest is the longest-surviving heart transplant recipient in Georgia and one of the longest-surviving in the world. The 75-year-old accountant still plays golf twice a week and only recently went from working full-time to part-time.

My heart is doing just fine, he says.

Murphy is now the chief of cardiothoracic surgery at Emory Saint Josephs Hospital and still in the operating room almost every day. He has moved on to become the worlds leading expert in robotically assisted heart surgery.

***

Harry Wuest is originally from Long Island, N.Y. After a stint in the U.S. Air Force, he moved to Florida to work and go to school. He wanted to become a physical education teacher. Then, in 1973, he fell ill. It started with some pain on his left side. He didnt think much of it, but when he got increasingly winded and fatigued, he went to see a doctor.

Several months and numerous specialists later, he received the diagnosis: Cardiomyopathy, a disease of the heart muscle that can make the heart become enlarged, thick and rigid, preventing it from pumping enough blood through the body.

They didnt know how I got it, says Wuest, sitting back in a brown leather armchair in the dark, wood-paneled living room of his Stone Mountain home. Maybe it was a virus. And back then, there wasnt much they could do to treat it, except bed rest.

For the next 12 years, Wuest lived life as best as he could. He got a degree in accounting from the University of Central Florida and worked for a real estate developer. There were good days, but there were more bad days. He was often too weak to do anything, and his heart was getting bigger and bigger.

***

The first successful human-to-human heart transplant was performed in Cape Town, South Africa, in 1967 a medical breakthrough that catapulted the surgeon, Dr. Christiaan Barnard, onto the cover of Life magazine and to overnight celebrity status.

This highly publicized event was followed by a brief surge in the procedure around the world, but overall, heart transplants had a rocky start. Most patients died shortly after the surgery, mainly due to organ rejection. Back then, immunosuppressive drugs, which can counteract rejection, were still in their infancy. Many hospitals stopped doing heart transplants in the 1970s.

That changed with the discovery of a highly effective immunosuppressive agent. Cyclosporine got FDA approval in 1983 and altered the world of organ transplants.

It was shortly thereafter when Emory University Hospital decided to launch a heart transplant program, but none of the senior surgeons wanted to do it. Even with the new drug, it was a risky surgery, and mortality was still high.

Its an all-or-nothing operation, Murphy says, as he sits down in his small office overlooking the greyish hospital compound. Hes wearing light blue scrubs from an early morning surgery. At 70, he still has boyish looks, with a lean build and an air of laid-back confidence. If you have a number of bad outcomes initially, it can be detrimental to your career as a surgeon, he says.

But Murphy didnt really have a choice. He remembers that during a meeting of Emorys cardiac surgeons in 1984, he was paged to check on a patient. When he returned, the physicians congratulated him on being appointed the head of the new heart transplant program. He was the youngest in the group and had been recruited from Harvards Massachusetts General Hospital just three years before.

Yeah, thats how I became Emorys first transplant surgeon, says Murphy.

He flew to California to shadow his colleagues at Stanford University Hospital, where most heart transplants were performed at the time. Back home at Emory, he put together a team and rigorously rehearsed the operation. The first transplant patient arrived in April 1985. The surgery was successful, as was the second operation less than a month later.

Around the same time, Harry Wuest wound up in a hospital in Orlando. He needed a transplant, but none of the medical centers in Florida offered the procedure. One of his doctors recommended Emory, and Wuest agreed. I knew I was dying. I could feel it. He was flown to Atlanta by air ambulance and spent several weeks in Emorys cardiac care unit until the evening of May 23, when Murphy walked into his room and said, Weve got a heart.

***

The heart, as the patient later learned, came from a 19-year-old sophomore at Georgia Tech who had been killed in a car crash.

Organ transplants are a meticulously choreographed endeavor, where timing, coordination and logistics are key. While Murphy and his eight-member team were preparing for the surgery, Wuest was getting ready to say farewell to his family his wife and three teenage sons and to thank the staff in the cardiac ward.

I was afraid, he recalls, especially of the anesthesia. It scared the heck out of me. He pauses during the reminiscence, choking briefly. I didnt know if I was going to wake up again.

The surgery took six hours. Transplants usually happen at night because the procurement team, the surgeons who retrieve different organs from the donor, only start working when regularly scheduled patients are out of the operating room.

Despite the cultural mystique surrounding the heart as the seat of life, Murphy says that during a transplant surgery, its not like the big spirit comes down to the operating room. Its very technical. As the team follows a precise routine, emotions are kept outside the door. We dont have time for that. Emotions come later.

After waking up from the anesthesia, Wuests first coherent memory was of Murphy entering the room and saying to a nurse, Lets turn on the TV, so Harry can watch some sports.

Wuest spent the next nine days in the ICU and three more weeks in the hospital ward. In the beginning, he could barely stand up or walk, because he had been bedridden weeks before the surgery and had lost a lot of muscle. But his strength came back quickly. I could finally breathe again, he says. Before the surgery, he felt like he was sucking in air through a tiny straw. I cannot tell you what an amazing feeling that was to suddenly breathe so easily.

Joane Goodroe was the head nurse at Emorys cardiovascular post-op floor back then. When she first met Wuest before the surgery, she recalls him lying in bed and being very, very sick. When she and the other nurses finally saw him stand up and move around, he was a whole different person.

In the early days of Emorys heart transplant program, physicians, nurses and patients were a particularly close-knit group, remembers Goodroe, whos been a nurse for 42 years and now runs a health care consulting firm. There were a lot of firsts for all of us, and we all learned from each other, she said.

Wuest developed friendships with four other early transplant patients at Emory, and he has outlived them all.

When he left the hospital, equipped with a new heart and a fresh hunger for life, Wuest made some radical changes. He decided not to return to Florida but stay in Atlanta. Thats where he felt he got the best care, and where he had found a personal support network. And he got a divorce. Four months after the operation, he went back to working full-time: first in temporary jobs and eventually for a property management company.

After having been sick for 12 years, I was just so excited to be able to work for eight hours a day, he recalls. That was a big, big deal for me.

At 50, he went back to school to get his CPA license. He also found new love.

Martha was a head nurse in the open-heart unit and later ran the cardiac registry at Saint Josephs Hospital. Thats where Wuest received his follow-up care and where they met in 1987. Wuest says for him it was love at first sight, but it took another five years until she finally agreed to go out with him. Six months later, they were married.

Having worked in the transplant office, I saw the good and the bad, Martha Wuest says. A petite woman with short, perfectly groomed silver hair, she sits up very straight on the couch, her small hands folded in her lap.Not every transplant patient did as well as Harry. And I had a lot of fear in the beginning. Now he may well outlive her, she says with a smile and a wink.

Wuests surgeon, meanwhile, went on to fight his own battles. Two and a half years into the program, Murphy was still the only transplant surgeon at Emory and on call to operate whenever a heart became available. Frustrated and exhausted, he quit his position at Emory and signed up with Saint Josephs (which at the time was not part of the Emory system) and started a heart transplant program there.

At St. Joes, Murphy continued transplanting hearts until 2005. In total, he did more than 200 such surgeries.

Being a heart transplant surgeon is a grueling profession, he says, and very much a younger surgeons subspecialty.

He then shifted his focus and became a pioneer in robotically assisted heart surgery.He has done more than 3,000 operations with the robot, mostly mitral valve repairs and replacements more than any other cardiac surgeon in the world.

***

Since Murphy sewed a new heart into Wuest, 35 years ago, there has been major progress in the field of heart transplants,but it has been uneven.

Medications to suppress the immune system have improved, says Dr. Jeffrey Miller, a transplant surgeon and heart failure specialist at Emory. As a result, we are seeing fewer cases of rejections of the donor heart.

Also, there are new methods of preserving and transporting donor hearts.

Yet patients requiring late-stage heart failure therapy, including transplantation, still exceed the number of donor hearts available. In 2019, 3,551 hearts were transplanted in the United States, according to the national Organ Procurement and Transplantation Network. But 700,000 people suffer from advanced heart failure, says the American Heart Association.

New technologies and continued research are providing hope to many of these patients. There has been significant progress in the development of partial artificial hearts, known as Left Ventricular Assist Devices, or LVADs, says Miller.

These are implantable mechanical pumps that assist the failing heart. Patients are back out in society living normal lives while theyre waiting for their donor hearts, he explains.

LVADs are used not only as bridge devices but as destination therapy as well, maintaining certain patients for the remainder of their lives.

Also, total artificial hearts have come a long way since the first artificial pump was implanted in a patient in 1969.

Long-term research continues into xenotransplantation, which involves transplanting animal cells, tissues and organs into human recipients.

Regenerative stem cell therapy is an experimental concept where stem cell injections stimulate the heart to replace the rigid scar tissue with tissue that resumes contraction, allowing for the damaged heart to heal itself after a heart attack or other cardiac disease.

Certain stem cell therapies have shown toreverse the damage to the heart by 30 to 50 percent, says Dr. Joshua Hare, a heart transplant surgeon and the director of the Interdisciplinary Stem Cell Institute at the University of Miamis Miller School of Medicine.

All of these ideas have potential, says Miller. But they have a lot of work before were ready to use them as alternatives to heart transplantation. I dont think were talking about the next few years.

Besides Emory, other health care systems in Georgia that currently have a heart transplant program are Piedmont Healthcare, Childrens Healthcare of Atlanta and Augusta University Health.

Organ rejection remains a major issue, and long-term survival rates have not improved dramatically over the past 35 years. The 10-year survival is currently around 55 percent of patients, which makes long-term-survivors like Harry Wuest rare in the world of heart transplants.

The United Network of Organ Sharing, or UNOS, which allocates donor hearts in the United States, doesnt have comprehensive data prior to 1987. An informal survey of the 20 highest-volume hospitals for heart transplants in the 1980s found only a scattering of long-term survivors.

***

Being one of the longest-living heart transplant recipients is something that Wuest sees as a responsibility to other transplant patients, but also to the donors family, which hes never met. If you as a transplant recipient reject that heart, thats like a second loss for that family.

Part of this responsibility is living a full and active life. Both he and Martha have three children from their previous marriages, and combined they have 15 grandchildren. Most of their families live in Florida, so they travel back and forth frequently. Wuest still works as a CPA during tax season, and he does advocacy for the Georgia Transplant Foundation. In addition to golf, he enjoys lifting weights and riding his bike.

Hes had some health scares over the years. In 2013, he was diagnosed with stage 1 kidney cancer, which is in remission. Also, he crossed paths with his former surgeon, and not just socially. In 2014, Murphy replaced a damaged tricuspid valve in Wuests new heart. That operation went well, too.

Murphy says there are several reasons why Wuest has survived so long. Obviously, his new heart was a very good match. But a patient can have the best heart and the best care and the best medicines and still die a few months or years after the transplantation, the surgeon says. Attitude plays a key role.

Wuest was psychologically stable and never suffered from depression or anxiety, Murphy says. Hes a numbers guy. He knew the transplant was his only chance, and he was set to pursue it.

Wuest attributes his longevity to a good strong heart from his donor; good genetics; great doctors and nurses; and a life that he loves. Im just happy to be here, he says.

Quoting his former surgeon and friend, he adds: Doug always said, Having a transplant is like running a marathon. And Im in for the long haul.

Katja Ridderbusch is an Atlanta-based journalist who reports for news organizations in the U.S. and her native Germany. Her stories have appeared in Kaiser Health News, U.S. News & World Report and several NPR affiliates.

This is a slightly modified version of the article 34 Years with a New Heart, published by Georgia Health News on February 18, 2020.

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(Image from Trinity College Dublin)

Researchers in Ireland have developed a prototype patch that they say does the same job as crucial aspects of heart tissue.

The patch was designed to withstand the mechanical demands of heart tissue and mimic the electrical signaling properties that allow the heart to pump blood throughout the body. The researchers believe it brings medtech one step closer to a functional design that could mend a broken heart.

Cardiac patches lined with heart cells can be applied surgically to restore heart tissue in patients who have had damaged tissue removed after a heart attack and to repair congenital heart defects in infants and children. Ultimately, though, the goal is to create cell-free patches that can restore the synchronous beating of the heart cells, without impairing the heart muscle movement. The bioengineers report their work in the journal Advanced Functional Materials.

Researchers are continuously looking to develop new treatments which can include stem cell treatments, biomaterial gel injections and assistive devices, said senior author Michael Monaghan, an assistant professor at Trinity College Dublin, in a news release. Ours is one of few studies that looks at a traditional material, and through effective design allows us to mimic the direction-dependent mechanical movement of the heart, which can be sustained repeatably. This was achieved through a novel method called melt electrowriting and through close collaboration with the suppliers located nationally we were able to customize the process to fit our design needs.

This work was performed in the Trinity Centre for Biomedical Engineering, based in the Trinity Biomedical Sciences Institute in collaboration with Spraybase, a subsidiary of Avectas Ltd.

The mechanical demands of heart muscle cannot be met using polyester-based thermoplastic polymers, which are predominantly the approved options for biomedical applications, according to the researchers. However, the functionality of thermoplastic polymers could be leveraged by its structural geometry. They made a patch that could control the expansion of a material in multiple directions and tune this using an engineering design approach.

The patches were manufactured via melt electrowriting, a core technology of Spraybase, which the company says is reproducible, accurate and scalable. The patches were also coated with the polymer polypyrrole to provide electrical conductivity while maintaining cell compatibility. The patch withstood repeated stretching, which is a dominant concern for cardiac biomaterials, and showed good elasticity, to accurately mimic that key property of heart muscle.

Essentially, our material addresses a lot of requirements, Monaghan said. The bulk material is currently approved for medical device use, the design accommodates the movement of the pumping heart, and has been functionalized to accommodate signaling between isolated contractile tissues. This study currently reports the development of our method and design, but we are now looking forward to furthering the next generation of designs and materials with the eventual aim of applying this patch as a therapy for a heart attack.

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Cardiac Stem Cells Comments Off on Could this patch help mend a broken heart? – Medical Design & Outsourcing | February 18th, 2020

- First conditional approval of ZYNTEGLOTM (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy for patients 12 years and older with transfusion-dependent -thalassemia who do not have 0/0 genotype in Europe achieved in 2019; Germany launch underway

- Announced positive top-line data from pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma

- Presented clinical data across studies of LentiGlobin gene therapy for -thalassemia (betibeglogene autotemcel) and LentiGlobin gene therapy for sickle cell disease (SCD) and bb21217 in multiple myeloma at American Society of Hematology (ASH) Annual Meeting

- Ended quarter with $1.24 billion in cash, cash equivalents and marketable securities

bluebird bio, Inc. (NASDAQ: BLUE) today reported financial results and business highlights for the fourth quarter and full year ended December 31, 2019.

"2019 was truly a transformative year for bluebird, with our first commercial product now launched in Europe and exciting progress across our first four clinical programs and pipeline," said Nick Leschly, chief bluebird. "Notably, our data in SCD continues to build, and at the ASH annual meeting in December we presented data that showed a 99% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome (ACS) in HGB-206 Group C patients with history of VOCs and ACS who had at least six months follow-up. In -thalassemia, the consistency with which patients who do not have a 0/0 genotype in our Northstar-2 (HGB-207) study are achieving transfusion independence is very encouraging and were starting to see indications that we may be able to see similar outcomes with many patients with 0/0 genotypes as well in our Northstar-3 (HGB-212 study). These data put us in a strong position as we progress our European launch, currently underway in Germany. At the end of 2019, we also announced positive top-line data from the pivotal KarMMa study of ide-cel. We and our partners at BMS look forward to submitting these data to the FDA in the first half of this year. Amidst all of our progress in 2019, our birds demonstrated time and again their dedication to patients and ability to meet and learn from the many challenges we have faced along the way. I look forward to facing the challenges of 2020 with this amazing flock."

Story continues

Recent Highlights:

TRANSFUSION-DEPENDENT -THALASSEMIA

LAUNCH IN GERMANY In January 2020, bluebird bio announced the launch of ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent -thalassemia (TDT) who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available in Germany. The company signed its first agreements with statutory health insurances utilizing bluebirds innovative value-based payment model and providing coverage for ZYNTEGLO for up to 50% of patients in Germany, and the first qualified treatment center was established at University Hospital of Heidelberg to provide ZYNTEGLO to patients. The company anticipates treating the first commercial patient in the first half of 2020.

UPDATED LENTIGLOBIN FOR -THALASSEMIA DATA At the American Society of Hematology (ASH) meeting in December 2019, bluebird bio presented new data from its studies of LentiGlobin gene therapy for -thalassemia (betibeglogene autotemcel) in patients with TDT: long-term data from the completed Phase 1/2 Northstar study (HGB-204), updated data from the Phase 3 Northstar-2 study (HGB-207) in patients with non-0/0 genotypes, and updated data from the Phase 3 Northstar-3 study (HGB-212) in patients with 0/0 genotypes or an IVS-I-110 mutation.

BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION bluebird bio has initiated its rolling BLA submission of LentiGlobin for -thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for -thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.

SICKLE CELL DISEASE (SCD)

HGB-211 bluebird bio is announcing today plans to launch HGB-211, the companys second Phase 3 study of LentiGlobin for sickle cell disease (SCD). This study is expected to enroll approximately 18 patients ages 2-14 years with SCD and elevated stroke risk, stroke being one of the most severe complications during childhood and adolescence. The primary endpoint of the study will be transcranial doppler response without transfusion. HGB-211 is in addition to the companys previously announced Phase 3 study (HGB-210) and is intended to support potential approval of LentiGlobin for SCD in pediatric patients at elevated stroke risk. HGB-211 is expected to begin enrolling patients in 2020.

UPDATED LENTIGLOBIN FOR SCD DATA At the ASH meeting in December 2019, bluebird bio presented new data from patients in Groups A, B and C in the Phase 1/2 HGB-206 study in patients with SCD. Group C patients are being treated under a study protocol utilizing hematopoietic stem cell (HSC) mobilization and apheresis with plerixafor, and a refined manufacturing process to increase vector copy number and engraftment potential of gene-modified HSCs. The company also disclosed that the target enrollment in HGB-206 has been achieved.

MULTIPLE MYELOMA

KARMMA TOPLINE In December 2019, Bristol-Myers Squibb and bluebird bio announced positive top-line results from the pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma. The study met its primary endpoint and key secondary endpoint, demonstrating deep and durable responses in a heavily pre-treated multiple myeloma patient population. Safety results are consistent with the data presented in CRB-401 study.

BB21217 DATA At the ASH meeting in December 2019, bluebird bio and Bristol-Myers Squibb presented updated data from ongoing CRB-402 Phase 1 study of BCMA-targeted CAR T cell therapy bb21217 in relapsed and refractory multiple myeloma. The dose escalation part of CRB-402 is complete, and the dose expansion part of the study is ongoing.

COMPANY

FORTY SEVEN COLLABORATION In November 2019, bluebird bio and Forty Seven announced that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Sevens novel antibody-based conditioning regimen, FSI-174 (anti-cKIT antibody) plus magrolimab (anti-CD47 antibody), with bluebirds ex vivo lentiviral vector hematopoietic stem cell (LVV HSC) gene therapy platform. Under the terms of the agreement, bluebird bio will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its innovative antibody-based conditioning regimen for the collaboration.

Upcoming Anticipated Milestones:

Regulatory

Submission of a BLA to the U.S. FDA for ide-cel in patients with relapsed and refractory multiple myeloma in the first half of 2020, in partnership with Bristol-Myers Squibb.

Submission of a BLA to the U.S. FDA and a Marketing Authorization Application to the European Medicines Agency for Lenti-D in patients with cerebral adrenoleukodystrophy by the end of 2020.

Clinical

Submission for presentation of ide-cel clinical data from the KarMMa study in the first half of 2020, in partnership with Bristol-Myers Squibb.

Submission for presentation of ide-cel clinical data from the CRB-401 study in 2020, in partnership with Bristol-Myers Squibb.

Initiation of the Phase 3 HGB-210 study of LentiGlobin for SCD in patients with a history of vaso-occlusive crises in the first half of 2020.

Initiation of the Phase 3 HGB-211 study of LentiGlobin for SCD in patients at risk of stroke in 2020.

Updated data presentation from ALD-102 in patients with CALD by the end of 2020.

Updated data presentation from the Northstar-2 (HGB-207) clinical study in patients with transfusion-dependent -thalassemia (TDT) and non-0/0 genotypes by the end of 2020.

Updated data presentation from the Northstar-3 (HGB-212) clinical study in patients with TDT and a 0/0 genotype or an IVS-I-110 mutation by the end of 2020.

Updated data presentation from HGB-206 clinical study in patients with SCD by the end of 2020.

Commercial and Foundation Building

ZYNTEGLO first commercial patients treated in the first half of 2020.

ZYNTEGLO access and reimbursement in additional EU countries established by the end of 2020.

Fourth Quarter and Full Year 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2019 and December 31, 2018 were $1.24 billion and $1.89 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating and commercial-readiness activities.

Revenues: Collaboration and license and royalty revenues were $10.0 million for the three months ended December 31, 2019 compared to $19.2 million for the three months ended December 31, 2018. Collaboration and license and royalty revenues were $44.7 million for the year ended December 31, 2019 compared to $54.6 million for the year ended December 31, 2018. The decrease in both periods was primarily attributable to a decrease in collaboration revenue under our arrangement with Bristol-Myers Squibb, partially offset by an increase in license and royalty revenue.

R&D Expenses: Research and development expenses were $161.8 million for the three months ended December 31, 2019 compared to $119.7 million for the three months ended December 31, 2018. Research and development expenses were $582.4 million for the year ended December 31, 2019 compared to $448.6 million for the year ended December 31, 2018. The increase in both periods was primarily driven by costs incurred to advance and expand the companys pipeline.

SG&A Expenses: Selling, general and administrative expenses were $76.2 million for the three months ended December 31, 2019 compared to $53.5 million for the three months ended December 31, 2018. Selling, general and administrative expenses were $271.4 million for the year ended December 31, 2019 compared to $174.1 million for the year ended December 31, 2018. The increase in both periods was largely attributable to costs incurred to support the companys ongoing operations and growth of its pipeline as well as commercial-readiness activities.

Net Loss: Net loss was $223.3 million for the three months ended December 31, 2019 compared to $149.0 million for the three months ended December 31, 2018. Net loss was $789.6 million for the year ended December 31, 2019 compared to $555.6 million for the year ended December 31, 2018.

LentiGlobin for -thalassemia Safety

Non-serious adverse events (AEs) observed during the HGB-204, HGB-207 and HGB-212 clinical studies that were attributed to LentiGlobin for -thalassemia were hot flush, dyspnoea, abdominal pain, pain in extremities, thrombocytopenia, leukopenia, neutropenia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for -thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

With more than five years of follow-up to date, there have been no new unexpected safety events, no deaths, no graft failure and no cases of vector-mediated replication competent lentivirus or clonal dominance. In addition, there have been no new reports of veno-occlusive liver disease (VOD) as of the data cutoff presented at ASH.

About LentiGlobin for -Thalassemia (betibeglogene autotemcel)

The European Commission granted conditional marketing authorization for LentiGlobin for -thalassemia, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding A-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a 0/0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin for -thalassemia adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs). Once a patient has the A-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for -thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

bluebird bio has initiated its rolling BLA submission of LentiGlobin for -thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for -thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.

LentiGlobin for -thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit http://www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for -thalassemia. For more information visit: https://www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

ZYNTEGLO, LentiGlobin, and bluebird bio are trademarks of bluebird bio, Inc.

The full common name for ZYNTEGLO: A genetically modified autologous CD34+ cell enriched population that contains hematopoietic stem cells transduced with lentiviral vector encoding the A-T87Q-globin gene.

Forward-Looking Statements

This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the companys financial condition, results of operations, as well as statements regarding the plans for regulatory submissions and commercialization for ZYNTEGLO and the companys product candidates, including anticipated regulatory milestones, the execution of the companys commercial launch plans, planned clinical studies, as well as the companys intentions regarding the timing for providing further updates on the development and commercialization of ZYNTEGLO and the companys product candidates. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing or future clinical trials; the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates; the risk that the current or planned clinical trials of our product candidates will be insufficient to support regulatory submissions or marketing approval in the United States and European Union; the risk that regulatory authorities will require additional information regarding our product candidates, resulting in delay to our anticipated timelines for regulatory submissions, including our applications for marketing approval; the risk that we will encounter challenges in the commercial launch of ZYNTEGLO in the European Union, including in managing our complex supply chain for the delivery of drug product, in the adoption of value-based payment models, or in obtaining sufficient coverage or reimbursement for our products; the risk that our collaborations, including the collaborations with Bristol-Myers Squibb and Forty Seven, will not continue or will not be successful; and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

bluebird bio, Inc.Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except per share data)(unaudited)

For the three months endedDecember 31,

For the year endedDecember 31,

2019

2018

2019

2018

Revenue:

Collaboration revenue

$ 7,159

$ 18,382

$ 36,469

$ 52,353

License and royalty revenue

2,838

861

8,205

2,226

Total revenues

9,997

19,243

44,674

54,579

Operating expenses:

Research and development

161,821

119,722

582,413

448,589

Selling, general and administrative

76,202

53,508

271,362

174,129

Cost of license and royalty revenue

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bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress - Yahoo Finance

Cardiac Stem Cells Comments Off on bluebird bio Reports Fourth Quarter and Full Year 2019 Financial Results and Highlights Operational Progress – Yahoo Finance | February 18th, 2020

TheGlobalAutologous Stem Cell and Non-Stem Cell Based Therapies Marketis expected to reach USD113.04 billion by 2025, from USD 87.59 billion in 2017 growing at a CAGR of 3.7% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2015 & 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

For In depth Information Get Sample Copy of this Report @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market&raksh

Some of the major players operating in the global autologous stem cell and non-stem cell based therapies market areAntria (Cro), Bioheart, Brainstorm Cell Therapeutics, Cytori, Dendreon Corporation, Fibrocell, Genesis Biopharma, Georgia Health Sciences University, Neostem, Opexa Therapeutics, Orgenesis, Regenexx, Regeneus, Tengion, Tigenix, Virxsys and many more.

The data and information included in this Global Autologous Stem Cell And Non-Stem Cell Based Therapies business report helps businesses take sound decisions and plan about the advertising and sales promotion strategy more successfully. This Autologous Stem Cell And Non-Stem Cell Based Therapies market research report is generated by taking into account a range of objectives of market research that are vital for the clients success. This report also includes strategic profiling of key players in the market, systematic analysis of their core competencies, and draws a competitive landscape for the Healthcare industry. The Global Autologous Stem Cell And Non-Stem Cell Based Therapies business report includes market shares for global, Europe, North America, Asia Pacific and South America.

Market Definition:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

In autologous stem-cell transplantation persons own undifferentiated cells or stem cells are collected and transplanted back to the person after intensive therapy. These therapies are performed by means of hematopoietic stem cells, in some of the cases cardiac cells are used to fix the damages caused due to heart attacks. The autologous stem cell and non-stem cell based therapies are used in the treatment of various diseases such as neurodegenerative diseases, cardiovascular diseases, cancer and autoimmune diseases, infectious disease.

According to World Health Organization (WHO), cardiovascular disease (CVD) causes more than half of all deaths across the European Region. The disease leads to death or frequently it is caused by AIDS, tuberculosis and malaria combined in Europe. With the prevalence of cancer and diabetes in all age groups globally the need of steam cell based therapies is increasing, according to article published by the US National Library of Medicine National Institutes of Health, it was reported that around 382 million people had diabetes in 2013 and the number is growing at alarming rate which has increased the need to improve treatment and therapies regarding the diseases.

Browse Detailed TOC Herehttps://www.databridgemarketresearch.com/toc/?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market&raksh

Market Segmentation:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

Competitive Analysis:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

The global autologous stem cell and non-stem cell based therapies market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of autologous stem cell and non-stem cell based therapies market for global, Europe, North America, Asia Pacific and South America.

Major Autologous Stem Cell and Non-Stem Cell Based Therapies Market Drivers and Restraints:

Introduction of novel autologous stem cell based therapies in regenerative medicine

Reduction in transplant associated risks

Prevalence of cancer and diabetes in all age groups

High cost of autologous cellular therapies

Lack of skilled professionals

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This Autologous Stem Cell And Non-Stem Cell Based Therapies Market report will enable both of the sides in market be an established firm or a relative new entrant. It helps the established firms to know about the moves which are being performed by their competitors and also helps the new entrants by educating them about the market situations and the industry trends. This Autologous Stem Cell And Non-Stem Cell Based Therapies Market report is quite fruitful in helping to understand the market definition and all the aspects of the market including the CAGR value and key profiles.

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Autologous Stem Cell And Non-Stem Cell Based Therapies Market 2020-2025 Booming || Leadinf Players Fibrocell, Genesis Biopharma, Georgia Health...

Cardiac Stem Cells Comments Off on Autologous Stem Cell And Non-Stem Cell Based Therapies Market 2020-2025 Booming || Leadinf Players Fibrocell, Genesis Biopharma, Georgia Health… | February 17th, 2020

The medical AM segment is multifaceted, consisting of 3D printed medical devices, anatomical models, prosthetics and more. Within the industry,bioprintinghas carved out a prominent position, gaining interest across the board for its huge potentials in drug development and screening, therapeutic treatments and regenerative medicine, to name but a few. While much of the excitement surrounding bioprinting is focused on the futurewhat it could dowe want to look at what is happening now in the field that is exciting.

As part of our Medical AM Focus, we asked bioprinting leaders from across the sector what they consider to be the most exciting application for their bioprinting technologies today. In this segment, we hear from Canadian bioprinting company Aspect Biosystems.

Based in Vancouver, Aspect Biosystems is a biotech company that specializes in the microfluidic 3D bioprinting of human tissues. The company has brought to market a sophisticated bioprinting systemthe RX1 Bioprinterwhich is today used by researchers all over the globe for medical applications, including neural and cardiac research. The company also conducts its own research in-house, developing therapeutic tissue programs for orthopedic and metabolic disorders.

According to Aspect Biosystems, one of the most exciting regenerative medicine applications it is currently exploring is the bioprinting of pancreatic tissue, which could help in the treatment of diabetes patients.

The moonshot for the bioprinting industry is to create whole replacement organs, the company tells us. While this is a great goal, it is still a long way off. At Aspect, we are focused on restoration of function versus replication of entire organ structure. Many diseases and disorders are a result of missing or lost function, in which case patients dont necessarily need a whole new organthey need an implantable tissue that replaces the lost function of the damaged organ.

This is particularly true of people living with type 1 diabetes. A patient with type 1 diabetes has a pancreas that does not perform its intended functions of secreting insulin and regulating blood sugar levels. Therefore, we are not aiming to create a replacement pancreas, but rather an implantable tissue therapeutic that would restore the the intended functions of the organ.

Microfluidic-based 3D bioprinting enables the creation of well-defined microstructures, thus allowing us to print a tissue structure with specific features such as immune-protection, vascularization, or production of hormones like insulin, says Aspect Biosystems. In the case of pancreatic tissue, microfluidics gives us the ability to print a multi-layered structure with a core of insulin-producing pancreatic islet cells (stem cell-derived beta-like cells) surrounded by an immuno-protective layer.

Our goal is to use these bioprinted encapsulated pancreatic beta cells as the foundation for developing an implantable 3D tissue therapeutic. Adding an outer layer that immune cells cant penetrate, but that allows diffusion of insulin, would ensure the success of the implanted tissue therapeutic as a long-term treatment for patients with type 1 diabetes without the need for immunosuppressant therapy.

As Aspect Biosystems moves forward in its bioprinting R&D, it is supported by a recent investment of $20 million, led by VC firm Radical Ventures. This injection of capital will help the company to accelerate its bioprinting workboth for the pancreatic tissue development and beyond.

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Aspect Biosystems on the restorative capacity of bioprinting today - 3DPMN

Cardiac Stem Cells Comments Off on Aspect Biosystems on the restorative capacity of bioprinting today – 3DPMN | February 17th, 2020

Scientific discoveries and research missions beyond Earths surface are quickly moving forward. Advancements in the fields of research, space medicine, life, and physical sciences, are taking advantage of the effects of microgravity to find solutions to some big problems here on Earth. Researchers in 3D printing and bioprinting have taken advantage of space facilities that are dedicated to conducting multiple experiments in orbit, such as investigating microgravitys effects on the growth of three-dimensional, human-like tissues, creating high-quality protein crystals that will help scientists develop more effective drugs, and even growing meat with 3D printing technology.

The BioFabrication Facility (BFF) by Techshot and nScrypt (Credit: Techshot)

On November 2, 2019, a Northrop Grumman Antares rocket successfully launched a Cygnus cargo spacecraft on a mission to the International Space Station (ISS). The payload aboard the Cygnus included supplies for the 3D BioFabrication Facility (BFF), like human cells, bioinks, as well as new 3D printed ceramic fluid manifolds that replaced the previously used that were printed out of polymers. According to Lithoz the company behind the 3D printed ceramic fluid manifolds they are enabling advancements in bioprinting at the ISS.

The additive manufactured ceramics have been in service since November 2019 and Lithoz claims they have proven to provide better biocompatibility than printed polymers, resulting in larger viable structures.

Lithoz, a company specializing in the development and production of materials and AM systems for 3D printing of bone replacements and high-performance ceramics, printed the ceramic manifolds using lithography-based ceramic manufacturing (LCM) on a high-resolution CeraFab printer in collaboration with Techshot, one of the companies behind the development of the BFF. Moreover, the ceramic fluid manifolds are used inside bioreactors to provide nutrients to living materials in space by the BFF.

Testing of the ceramic 3D printed manifolds is focusing on biocompatibility, precision, durability, and overall fluid flow properties; and the latest round of microgravity bioprinting in December yielded larger biological constructs than the first BFF attempts in July.

NASA engineer Christina Koch works with the BioFabrication Facility in orbit (Credit: NASA)

Techshot and Lithoz engineers and scientists worked together to optimize the design and the manufacturing processes required to make it. Techshot Senior Scientist Carlos Chang reported that its been an absolute pleasure working with Lithoz.

While Lithoz Vice President Shawn Allan suggested that their expertise in ceramic processing really made these parts happen. The success of ceramic additive manufacturing depends on working together with design, materials, and printing. Design for ceramic additive manufacturing principles was used along with print parameter control to achieve Techshots complex fluid-handling design with the confidence needed to use the components on ISS.

Headquartered in Vienna, Austria, and founded in 2011, Lithoz offers applications and material development to its customers in cooperation with renowned research institutes all over the world, benefiting from a variety of materials ranging from alumina, zirconia, silicon nitride, silica-based for casting-core applications through medical-grade bioceramics.

This work, in particular, highlighted an ideal use case for ceramic additive manufacturing to enable the production of a special compact device that could not be produced without additive manufacturing while enabling a level of bio-compatibility and strength not achievable with printable polymers. Lithoz reported that Techshot engineers were able to interface the larger bio-structures with the Lithoz-printed ceramic manifolds and that the next steps will focus on optimized integration of these components and longer culturing of the printed biological materials. While conditioned human tissues from this mission are expected to return to Earth in early 2020 for evaluation.

Back in July 2019, Gene Boland,chief scientist atTechshot, and Ken Church, chief executive officer atnScrypt, discussed the BFF at NASAs Kennedy Space Center in Port Canaveral, Florida, how they planned to use the BFF in orbit to print cells (extracellular matrices), grow them and have them mature enough so that when they return to Earth researchers can encounter a close to full cardiac strength. Church described how a tissue of this size has never been grown here on Earth, let alone in microgravity. The 3D BFF is the first-ever 3D printer capable of manufacturing human tissue in the microgravity condition of space. Utilizing adult human cells (such as pluripotent or stem cells), the BFF can create viable tissue in space through a technology that enables it to precisely place and build ultra-fine layers of bioink layers that may be several times smaller than the width of a human hair involving the smallest print nozzles in existence.

Flight engineer Andrew Morgan works with the BioFabrication Facility (Credit: NASA)

Experts suggest that bioprinting without gravity eliminates the risk of collapse, enabling organs to grow without the need for scaffolds, offering a great alternative to some of the biggest medical challenges, like supplying bioprinted organs, providing a solution to the shortage of organs.

With NASA becoming more committed to stimulating the economy in low-Earth orbit (LEO), as well as opening up the ISS research lab to scientific investigations and experiments, we can expect to learn more about some of the most interesting discoveries that could take place 220 miles above Earth. There are already quite a few bioprinting experiments taking place on the ISS, including Allevi and Made In Spaces existing Additive Manufacturing Facility on the ISS, the ZeroG bio-extruder which allow scientists on the Allevi platform to simultaneously run experiments both on the ground and in space to observe biological differences that occur with and without gravity, and CELLINKs collaboration with Made In Space to identify 3D bioprinting development opportunities for the ISS as well as for future off-world platforms. All of these approaches are expected to have an impact on the future of medicine on Earth.

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Improvements to the BioFabrication Facility on the ISS Thanks to Lithoz - 3DPrint.com

Cardiac Stem Cells Comments Off on Improvements to the BioFabrication Facility on the ISS Thanks to Lithoz – 3DPrint.com | February 17th, 2020

Early data trends from first patient dosed in the AVR-RD-04 investigational gene therapy program for cystinosis show improvements across multiple measures

Data from the Phase 1 and Phase 2 trials of AVR-RD-01 support potential long-term engraftment and durable, endogenous production of functional enzyme in patients with Fabry disease

First Phase 2 Fabry patient treated using plato gene therapy platform shows plasma enzyme activity at one month 4.0 times higher than mean activity of other Phase 2 patients treated using academic platform at same timepoint

Analyst and investor event will be webcast today, Feb. 10, 2020, at 7:00 p.m. ET, in conjunction with WORLDSymposiumTM

AVROBIO, Inc. (NASDAQ: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced new initial data from the first patient dosed in the investigational gene therapy program for cystinosis, showing improvements in early measures at three months compared to baseline. The company also unveiled new clinical data showcasing a sustained biomarker response in patients for up to 32 months after receiving the companys investigational gene therapy for Fabry disease across metrics including vector copy number (VCN), substrate levels and enzyme activity. Additionally, the company reported on the clinical debut of its platoTM gene therapy platform. These data showed improved enzyme activity, transduction efficiency and VCN in drug product manufactured using plato compared with drug product produced using the academic platform, as well as higher in vivo enzyme activity at one month in the first patient treated with plato, as compared to other patients treated using the academic platform. All these data will be presented today, during the 16th Annual WORLDSymposiumTM in Orlando, Fla.

"We have now dosed 10 patients across three trials for two lysosomal disorders and were delighted with the data were seeing. We have followed six patients in our Fabry trial for more than a year and one for nearly three years, and they are consistently producing the functional enzyme that was missing as a consequence of their genetic disease, suggesting a potentially durable effect from a single dose," said Geoff MacKay, AVROBIOs president and CEO. "Furthermore, we believe that early data from the first clinical application of plato support our decision to invest heavily from AVROBIO's earliest days in this state-of-the-art gene therapy platform. We believe these data collectively indicate that were making exciting progress toward our goal of freeing patients and families from the life-limiting symptoms and relentless progression of lysosomal disorders."

Story continues

Three-month data from first patient in investigational AVR-RD-04 trial in cystinosisAVROBIO reported initial data from the first patient dosed in the investigator-sponsored Phase 1/2 trial of the companys AVR-RD-04 investigational gene therapy for cystinosis, a progressive disease marked by the accumulation of cystine crystals in cellular organelles known as lysosomes. Patients with cystinosis accumulate the amino acid cystine, which can lead to crystal formation in the lysosomes of cells, causing debilitating symptoms including corneal damage, difficulty breathing and kidney failure, often leading to a shortened lifespan. The current standard of care for cystinosis, a burdensome treatment regimen that can amount to dozens of pills a day, may not prevent overall progression of the disease.

As of the safety data cut-off date of Jan. 27, 2020, which was approximately three months following administration of the investigational gene therapy to the first patient in the AVR-RD-04 program, there have been no reports of safety events attributed to the investigational drug product. In addition, no serious adverse events (SAEs) have been reported as of the safety data cut-off date. Adverse events did not suggest any unexpected safety signals or trends.

Three months following administration of AVR-RD-04, the first patient had a VCN of 2.0. VCN measures the average number of copies of the lentiviral-vector inserted transgene integrated into the genome of a cell and can be used to help assess the durability of a gene therapy. Initial data on another biomarker show that the patients average granulocyte cystine level -- one of the trials primary endpoints -- decreased from 7.8 nmol half cystine/mg protein two weeks after cysteamine discontinuation, to 1.5 at three months post-gene therapy.

The ongoing open-label, single-arm Phase 1/2 clinical trial evaluating the safety and efficacy of AVR-RD-04 is sponsored by AVROBIOs academic collaborators at the University of California San Diego (UCSD), led by Stephanie Cherqui, Ph.D. The trial is actively enrolling up to six participants at UCSD.

Interim data continue to support potential first line use of AVR-RD-01 in Fabry diseaseFour patients have been dosed in the Phase 2 trial (FAB-201), and five patients in the Phase 1 investigator-led trial of AVR-RD-01 in Fabry disease.

VCN data continue to be stable at 32 months following AVR-RD-01 treatment for the first patient in the Phase 1 trial, suggesting successful engraftment, which is critical to the long-term success of investigational ex vivo lentiviral gene therapies. The VCN data trend was generally consistent across the seven other Phase 1 and Phase 2 trial participants out six to 24 months.

The first three AVR-RD-01 Phase 2 patients entered the study with minimal endogenous enzyme activity. At nine, 12 and 18 months after dosing, data from these three patients indicate sustained increased leukocyte and plasma enzyme activity, suggesting that they are now producing an endogenous supply of functional alpha-galactosidase (AGA) enzyme. This enzyme is essential for breaking down globotriaosylceramide (Gb3) in cells; without it, a toxic metabolite, lyso-Gb3, may accumulate, potentially causing cardiac and kidney damage and other symptoms.

For two Phase 2 patients, data indicate that their decreased plasma lyso-Gb3 levels, a key biomarker for monitoring Fabry disease, have been sustained below their baseline at six and 18 months after dosing. The third Phase 2 patient, a cardiac variant who does not have classic Fabry disease, did not show a decrease in plasma lyso-Gb3 levels, as expected. Cardiac and kidney function measures in the Phase 2 trial remained within normal range for patients who had available 12-month data.

As previously reported, a kidney biopsy taken at 12 months post-treatment for the first patient in the Phase 2 trial showed an 87-percent reduction in Gb3 inclusions per peritubular capillary. The company believes this data point, the primary efficacy endpoint for the Phase 2 trial, supports the potential of AVR-RD-01 to reduce Gb3 levels in tissue, including in the kidney.

In the Phase 1 trial of AVR-RD-01, four of the five patients had their plasma lyso-Gb3 levels reduced between 26 and 47 percent compared to their pre-treatment baseline levels. Data from the other patient in the trial, who remains off enzyme replacement therapy (ERT), through month six showed an initial decline and at month 12 showed a 23-percent increase in lyso-Gb3 levels, as compared to pre-treatment levels. This patients lyso-Gb3 levels remain within the range for the Fabry disease patients on ERT observed in this study.

Overall, three of the five Phase 1 patients have discontinued ERT and all three remain off ERT for six, 14 and 15 months.

As of the safety data cut-off date of Nov. 26, 2019, there have been no safety events attributed to AVR-RD-01 drug product in either the Phase 1 or Phase 2 trial. Through the safety data cut-off date, four SAEs have been reported in the FAB-201 trial and two SAEs in the Phase 1 trial. The fourth Phase 2 patient, who was dosed after the safety data cut-off date, has reported an SAE, which was not attributed to AVR-RD-01 and which subsequently resolved. Across both studies, each of the SAEs has been consistent with the conditioning regimen, stem cell mobilization, underlying disease or pre-existing conditions. Pre-existing low anti-AGA antibody titers have been detected in four patients in the Phase 1 trial and a transient low titer was observed but not detectable in subsequent measures in one patient in the Phase 2 trial.

The Phase 1 trial is fully enrolled. AVROBIO continues to actively enroll the Phase 2 trial in Australia, Canada and the U.S. The FAB-201 trial is an ongoing open-label, single-arm Phase 2 clinical trial evaluating the efficacy and safety of AVR-RD-01 in eight to 12 treatment-nave patients with Fabry disease.

Successful clinical debut of platoTM gene therapy platformAVROBIO also shared preliminary results from the first two patients to receive busulfan conditioning. Conditioning is an essential step in ex vivo lentiviral gene therapy designed to clear space in the bone marrow for the cells carrying the therapeutic transgene to engraft. The conditioning regimen developed as part of AVROBIOs plato platform includes therapeutic dose monitoring to assess how rapidly the individual patient metabolizes busulfan so physicians can adjust the dose as needed, with a goal of minimizing side effects while maximizing the potential of durable engraftment.

AVROBIO is implementing its precision dosing conditioning regimen across its company-sponsored clinical trials as part of the plato platform. The fourth patient in AVROBIOs Phase 2 Fabry trial received a precision dosing conditioning regimen with busulfan as part of the plato platform, while the first patient in the investigator-led cystinosis trial received busulfan but not as part of the plato platform.

These two patients both had rapid neutrophil and platelet count recovery, with a trajectory that was similar to the patients who enrolled earlier in the Fabry trials and who received a melphalan conditioning regimen. Side effects, which included nausea, mucositis, fever, rash and hair loss, developed eight to 10 days after dosing with busulfan and then resolved quickly.

The company also reported preliminary data from the first drug product produced using the plato gene therapy platform, which was used to dose the fourth patient in the Phase 2 Fabry trial (FAB-201). Early data indicate that enzyme activity and transduction efficiency for the drug product used to dose the fourth patient were 2.2 times higher than the mean of the drug product used to dose the first three patients in FAB-201. VCN for the drug product used to dose the fourth patient was 1.8 times higher than the mean of the drug product for the first three patients dosed in FAB-201. The drug product for the first three patients in FAB-201 was manufactured using a manual process first developed by AVROBIOs academic collaborators. The automated manufacturing embedded in plato leverages optimized processes developed at AVROBIO.

At one month following administration of the plato-produced investigational gene therapy for the fourth patient in the Phase 2 Fabry trial, initial data show the patients plasma enzyme activity level to be 4.0 times higher than the mean activity level of the first three patients in the Phase 2 Fabry trial at the same timepoint.

The investigational drug product used to dose the first patient in the AVR-RD-04 program for cystinosis, which included a four-plasmid vector but not platos automated manufacturing process, also showed increased performance in line with the increased performance recorded for the drug product in the Fabry trial. The investigational drug product and VCN assay are different for each trial.

"We believe these data are an early, but exciting, validation of our decision to invest in technological innovation rather than build expensive bricks-and-mortar manufacturing facilities," said MacKay. "The plato platform gives us control over the production and scaling of our investigational gene therapies through an efficient, automated manufacturing system that is designed to be deployed in standard contracted sites around the world. The four-plasmid vector, conditioning regimen with precision dosing and other elements of plato are designed to optimize the safety, potency and durability of our investigational lentiviral gene therapies."

About AVROBIOs ex vivo approach to gene therapyOur investigational ex vivo gene therapies start with the patients own stem cells. In the manufacturing facility, a lentiviral vector is used to insert a therapeutic gene designed to enable the patient to produce a functional supply of the protein they lack. These cells are then infused back into the patient, where they are expected to engraft in the bone marrow and produce generations of daughter cells, each containing the therapeutic gene. This approach is designed to drive durable production of the functional protein throughout the patients body, including hard-to-reach tissues such as the brain, muscle and bone. It is a distinguishing feature of this type of gene therapy that the corrected cells are expected to cross the blood-brain barrier and thereby potentially address symptoms originating in the central nervous system.

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the transgenes presence as the patients cells divide, which may improve the expected durability of the therapy and potentially enable dosing of pediatric patients, whose cells divide rapidly as they grow. Because the transgene is integrated ex vivo into patients stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the viral vector.

Analyst and investor event and webcast informationAVROBIO will host an analyst and investor event today, Monday, Feb. 10, 2020, in conjunction with the WORLDSymposiumTM, an annual scientific meeting dedicated to lysosomal disorders, in Orlando, FL. The presentation at the event will be webcast beginning at 7:00 p.m. ET. The webcast and accompanying slides will be available under "Events and Presentations" in the Investors & Media section of the companys website at http://www.avrobio.com. An archived webcast recording of the event will be available on the website for approximately 30 days.

About AVROBIOOur mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as "aims," "anticipates," "believes," "could," "designed to," "estimates," "expects," "forecasts," "goal," "intends," "may," "plans," "possible," "potential," "seeks," "will," and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, and anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled "Risk Factors" in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200210005767/en/

Contacts

Investor Contact: Christopher F. BrinzeyWestwicke, an ICR Company339-970-2843chris.brinzey@westwicke.com

Media Contact: Tom DonovanTen Bridge Communications857-559-3397tom@tenbridgecommunications.com

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AVROBIO Presents Positive Initial Data for its Investigational Cystinosis Program and Plato TM Platform, as well as Positive Data Out to 32 Months for...

Cardiac Stem Cells Comments Off on AVROBIO Presents Positive Initial Data for its Investigational Cystinosis Program and Plato TM Platform, as well as Positive Data Out to 32 Months for… | February 16th, 2020

PHILADELPHIA, Feb. 13, 2020 /PRNewswire/ --Researchers at the University of Pittsburgh and the University of Delaware have been awarded funding and support to accelerate their search for new medicines through an ongoing partnership between global biotechnology leader CSL Behring and the University City Science Center.

CSL Behring awarded Cecelia Yates, Ph.D., from the University of Pittsburgh, and Eleftherios (Terry) Papoutsakis, Ph.D., from the University of Delaware, $250,000 each and an opportunity to work alongside the plasma-based biotech's own experts in an effort to help transform their ideas into groundbreaking therapies to improve patients' health.

CSL Behring, a global leader in treating rare and serious diseases which has its global operational headquarters in King of Prussia, PA, identified the medical researchers utilizing the Science Center's, sourcing innovation framework for technology commercialization, support and infrastructure to efficiently evaluate technologies from multiple institutions.

"Congratulations Drs. Yates and Papoutsakis on being the first recipients of the CSL Behring-Science Center Research Acceleration Initiative," said Bill Mezzanotte, MD, Executive Vice President, Head of Research and Development for CSL Behring. "This initiative is another example of the strength of our partnership with the Philadelphia-based University City Science Center as we look in our 'backyard' for innovative scientific advancements that have the potential to help rare disease patients lead full lives. Our growing R&D organization looks forward to working with Dr. Yates and Dr. Papoutsakis in the years ahead to advance their scientific research."

"The Science Center couldn't be more excited about facilitating the introduction between these talented investigators and CSL Behring," says John Younger, MD, Vice President of Science & Technology at the Science Center. "Our network of universities, biotech, and pharmaceutical companies was built exactly for making these connections not just possible but easy. Supporting the development of new biologics, and new drug and gene delivery systems like those developed by Drs. Papoutsakis and Yates will continue to be an important focus of our team."

The investigators and technologies selected in this inaugural round of the program include:

Cecelia Yates, Ph.D., University of Pittsburgh, for the use of FibroKine biomimetic peptides as potential targeted therapeutic treatment of pulmonary fibrosis.

Fibrotic diseases constitute a significant health problem in the US with the ability to impact any organ that is scarred from chronic disease, including the heart, lung, liver, arteries, and skin. In some cases, progressive and life-threatening diseases follow, but effective therapies don't yet exist. In response, Dr. Yates has developed FibroKine, a chemokine-based class of biomimetic peptides that are potential therapeutic agents for the targeted treatment of tissue fibrosis. Support from CSL Behring will allow the Yates group to test FibroKine peptide ability to effectively treat and halt the progression of pulmonary fibrosis.

Eleftherios (Terry) Papoutsakis, Ph.D., University of Delaware, for exploring the use of cell derived micro-particles and vesicles (MkMPs) for the treatment of thrombocytopenias and in stem-cell targeted gene therapies

Gene delivery to or editing of Hematopoietic (blood) Stem and Progenitor Cells (HSPCs) can provide therapeutic benefit to patients for a variety of genetic hematological disorders, ranging from low platelet count diseases to primary immune deficiencies like Wiskott-Aldrich syndrome. With the support of CSL Behring, Dr. Papoutsakis will investigate the use of human MkMPs: 1) to promote in vivo platelet biogenesis as a potential treatment for thrombocytopenias, and 2) for the in vivo delivery of DNA, RNA, and proteins to HSPCs in gene therapy applications.

In October 2018, the Science Center and CSL Behring joined forces to identify promising technologies and support the commercialization pathways of potential new discoveries. Researchers at academic and research institutions throughout the region were invited to submit proposals for projects with a focus on therapeutics that fit within CSL Behring's five therapeutic areas of expertise: immunology and neurology; hematology and thrombosis; respiratory; cardiovascular and metabolic; and transplant.

Following the success of the initial pilot, the CSL Behring Science Center Research Initiative has expanded and is currently accepting applicationsfrom researchers at 28 institutions across six states with awardees to receive up to $400,000 each.

About CSL BehringCSL Behringis a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, inherited respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn. CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited(ASX: CSL; USOTC: CSLLY), headquartered in Melbourne, Australia, employs more than 25,000 people, and delivers its life-saving therapies to people in more than 70 countries. For inspiring stories about the promise of biotechnology, visit Vita CSLBehring.com/vitaand follow us on Twitter.com/CSLBehring.

About the Science CenterLocated in the heart ofuCitySquare, the Science Center is a mission-driven nonprofit that commercializes promising technology, cultivates talent and convenes people to inspire action. For over 50 years, the Science Center has supported startups, research, and economic development across the emerging technology sectors. As a result, Science Center-supported companies account for one out of every 100 jobs in the Greater Philadelphia region and drive $13 billion in economic activity in the region annually. By providing the right help at the right time, the Science Center is turning bright ideas into businesses and nurturing a workforce to support our 21st century economy. For more information about the Science Center, go towww.sciencecenter.org

View original content:http://www.prnewswire.com/news-releases/university-city-science-center-partnership-with-csl-behring-accelerates-the-search-for-new-biotherapies-at-the-university-of-pittsburgh-and-the-university-of-delaware-301004508.html

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University City Science Center partnership with CSL Behring accelerates the search for new biotherapies at the University of Pittsburgh and the...

Cardiac Stem Cells Comments Off on University City Science Center partnership with CSL Behring accelerates the search for new biotherapies at the University of Pittsburgh and the… | February 14th, 2020

Technological innovations in the area of stem cell therapy and tissue engineering has led to rapid growth of the regenerative medicine market size.

Regenerative medicine is a comparatively new area of science that involves the restoration of damaged cells, tissues or organs by applying cell therapy, tissue engineering, immunotherapy or gene therapy techniques. On contrary to the present clinical therapeutics that act on slowing the disease progression or relieve symptoms, regenerative medication has a promising therapeutic approach of restoring the function and structure of damaged organs and tissues.

The global regenerative medicine market is expected to witness significant growth during the forecast period,due to the increase in the prevalence of chronic diseases, orthopaedic injuries, genetic disorders, growing aging population, increasing government funding along with the private funding in the research & development of regenerative medicines with the advancement in nanotechnology based drug delivery system, and moderate healthcare reforms. Currently, major breakthrough in the area is the development of tissue engineered trachea, transplantation of retinal pigment differentiated by stem cell based therapy to treat age-related macular degeneration.

However, recently research labs have started to focus on regenerating solid organs such as heart, kidney, lungs and other organs to curb the problems associated with organ transplantation.

The rise in number of regulatory approvals of regenerative medications is expected to further drive the regenerative medicine market during the forecast period. Moreover, there has been strategic partnership between many companies that has encouraged increased involvement of these companies in the global market.

Improvised drug delivery systems for regenerative medicines is also expected to contribute to the growth of the global market.

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The key factors which drive the growth of the global market include increase in the demand of orthopaedic surgeries, government healthcare reforms in certain countries such as the U.S. and Canada, aging population, rise in chronic diseases, increasing prevalence of bone and joint diseases, and innovations in nanotechnology that aids in drug delivery mechanism.

Globally, North America is the largest market for regenerative medicine followed by Europe. The largest regenerative medicine market size of North America is attributed to the high rate of incidence of cardiac disorders, autoimmune diseases, and increasing prevalence of cancer patients among the American population.

Additionally, the involvement of government organization for funding in the area of R&D of regenerative medicines, technological advancement and other policies are driving the growth of the North American market.

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Regenerative Medicine Market trends, leaders, segment analysis and forecast to 2030 described in a new market report - WhaTech Technology and Markets...

Cardiac Stem Cells Comments Off on Regenerative Medicine Market trends, leaders, segment analysis and forecast to 2030 described in a new market report – WhaTech Technology and Markets… | February 14th, 2020

Regenerative Medicine Market: Snapshot

Regenerative medicine is a part of translational research in the fields of molecular biology and tissue engineering. This type of medicine involves replacing and regenerating human cells, organs, and tissues with the help of specific processes. Doing this may involve a partial or complete reengineering of human cells so that they start to function normally.

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Regenerative medicine also involves the attempts to grow tissues and organs in a laboratory environment, wherein they can be put in a body that cannot heal a particular part. Such implants are mainly preferred to be derived from the patients own tissues and cells, particularly stem cells. Looking at the promising nature of stem cells to heal and regenerative various parts of the body, this field is certainly expected to see a bright future. Doing this can help avoid opting for organ donation, thus saving costs. Some healthcare centers might showcase a shortage of organ donations, and this is where tissues regenerated using patients own cells are highly helpful.

There are several source materials from which regeneration can be facilitated. Extracellular matrix materials are commonly used source substances all over the globe. They are mainly used for reconstructive surgery, chronic wound healing, and orthopedic surgeries. In recent times, these materials have also been used in heart surgeries, specifically aimed at repairing damaged portions.

Cells derived from the umbilical cord also have the potential to be used as source material for bringing about regeneration in a patient. A vast research has also been conducted in this context. Treatment of diabetes, organ failure, and other chronic diseases is highly possible by using cord blood cells. Apart from these cells, Whartons jelly and cord lining have also been shortlisted as possible sources for mesenchymal stem cells. Extensive research has conducted to study how these cells can be used to treat lung diseases, lung injury, leukemia, liver diseases, diabetes, and immunity-based disorders, among others.

Global Regenerative Medicine Market: Overview

The global market for regenerative medicine market is expected to grow at a significant pace throughout the forecast period. The rising preference of patients for personalized medicines and the advancements in technology are estimated to accelerate the growth of the global regenerative medicine market in the next few years. As a result, this market is likely to witness a healthy growth and attract a large number of players in the next few years. The development of novel regenerative medicine is estimated to benefit the key players and supplement the markets growth in the near future.

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Global Regenerative Medicine Market: Key Trends

The rising prevalence of chronic diseases and the rising focus on cell therapy products are the key factors that are estimated to fuel the growth of the global regenerative medicine market in the next few years. In addition, the increasing funding by government bodies and development of new and innovative products are anticipated to supplement the growth of the overall market in the next few years.

On the flip side, the ethical challenges in the stem cell research are likely to restrict the growth of the global regenerative medicine market throughout the forecast period. In addition, the stringent regulatory rules and regulations are predicted to impact the approvals of new products, thus hampering the growth of the overall market in the near future.

Global Regenerative Medicine Market: Market Potential

The growing demand for organ transplantation across the globe is anticipated to boost the demand for regenerative medicines in the next few years. In addition, the rapid growth in the geriatric population and the significant rise in the global healthcare expenditure is predicted to encourage the growth of the market. The presence of a strong pipeline is likely to contribute towards the markets growth in the near future.

Global Regenerative Medicine Market: Regional Outlook

In the past few years, North America led the global regenerative medicine market and is likely to remain in the topmost position throughout the forecast period. This region is expected to account for a massive share of the global market, owing to the rising prevalence of cancer, cardiac diseases, and autoimmunity. In addition, the rising demand for regenerative medicines from the U.S. and the rising government funding are some of the other key aspects that are likely to fuel the growth of the North America market in the near future.

Furthermore, Asia Pacific is expected to register a substantial growth rate in the next few years. The high growth of this region can be attributed to the availability of funding for research and the development of research centers. In addition, the increasing contribution from India, China, and Japan is likely to supplement the growth of the market in the near future.

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Global Regenerative Medicine Market: Competitive Analysis

The global market for regenerative medicines is extremely fragmented and competitive in nature, thanks to the presence of a large number of players operating in it. In order to gain a competitive edge in the global market, the key players in the market are focusing on technological developments and research and development activities. In addition, the rising number of mergers and acquisitions and collaborations is likely to benefit the prominent players in the market and encourage the overall growth in the next few years.

Some of the key players operating in the regenerative medicine market across the globe are Vericel Corporation, Japan Tissue Engineering Co., Ltd., Stryker Corporation, Acelity L.P. Inc. (KCI Licensing), Organogenesis Inc., Medtronic PLC, Cook Biotech Incorporated, Osiris Therapeutics, Inc., Integra Lifesciences Corporation, and Nuvasive, Inc. A large number of players are anticipated to enter the global market throughout the forecast period.

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Regenerative Medicine Market Projected to Hit at a Strong CAGR Between Forecast Period 2017-2025 - Redhill Local Councillors

Cardiac Stem Cells Comments Off on Regenerative Medicine Market Projected to Hit at a Strong CAGR Between Forecast Period 2017-2025 – Redhill Local Councillors | February 14th, 2020

KENILWORTH, N.J.--(BUSINESS WIRE)--Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the pivotal Phase 3 KEYNOTE-355 trial investigating KEYTRUDA, Mercks anti-PD-1 therapy, in combination with chemotherapy met one of its dual primary endpoints of progression-free survival (PFS) in patients with metastatic triple-negative breast cancer (mTNBC) whose tumors expressed PD-L1 (Combined Positive Score [CPS] 10). Based on an interim analysis conducted by an independent Data Monitoring Committee (DMC), first-line treatment with KEYTRUDA in combination with chemotherapy (nab-paclitaxel, paclitaxel or gemcitabine/carboplatin) demonstrated a statistically significant and clinically meaningful improvement in PFS compared to chemotherapy alone in these patients. Based on the recommendation of the DMC, the trial will continue without changes to evaluate the other dual primary endpoint of overall survival (OS). The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported studies; no new safety signals were identified.

Triple-negative breast cancer is an aggressive malignancy. It is very encouraging that KEYTRUDA in combination with chemotherapy has now demonstrated positive results as both a first-line treatment in the metastatic setting with this trial, and as neoadjuvant therapy in the KEYNOTE-522 trial, said Dr. Roger M. Perlmutter, president, Merck Research Laboratories. We look forward to sharing these findings with the medical community at an upcoming congress and discussing them with the FDA and other regulatory authorities.

The KEYTRUDA breast cancer clinical development program encompasses several internal and external collaborative studies. In addition to KEYNOTE-355, in TNBC these include the ongoing registration-enabling studies KEYNOTE-242 and KEYNOTE-522.

About KEYNOTE-355

KEYNOTE-355 is a randomized, two-part, Phase 3 trial (ClinicalTrials.gov, NCT02819518) evaluating KEYTRUDA in combination with one of three different chemotherapies (investigators choice of either nab-paclitaxel, paclitaxel or gemcitabine/carboplatin) compared with placebo plus one of the three chemotherapy regimens for the treatment of locally recurrent inoperable or mTNBC that has not been previously treated with chemotherapy in the metastatic setting. Part 1 of the study was open-label and evaluated the safety and tolerability of KEYTRUDA in combination with either nab-paclitaxel, paclitaxel or gemcitabine/carboplatin in 30 patients. Part 2 of KEYNOTE-355 was double-blinded, with dual primary endpoints of OS and PFS in all participants and in participants whose tumors expressed PD-L1 (CPS 1 and CPS 10). The secondary endpoints include objective response rate (ORR), duration of response (DOR), disease control rate (DCR) and safety.

Part 2 of KEYNOTE-355 enrolled 847 patients who were randomized to receive KEYTRUDA (200 mg intravenously [IV] on day 1 of each 21-day cycle) plus nab-paclitaxel (100 mg/m2 IV on days 1, 8 and 15 of each 28-day cycle), paclitaxel (90 mg/m2 IV on days 1, 8 and 15 of each 28-day cycle) or gemcitabine/carboplatin (1,000 mg/m2 [gemcitabine] and Area Under the Curve [AUC] 2 [carboplatin] on days 1 and 8 of each 21-day cycle); or placebo (normal saline on day 1 of each 21-day cycle) plus nab-paclitaxel (100 mg/m2 IV on days 1, 8 and 15 of each 28-day cycle), paclitaxel (90 mg/m2 IV on days 1, 8 and 15 of each 28-day cycle) or gemcitabine/carboplatin (1,000 mg/m2 [gemcitabine] and AUC 2 [carboplatin] on days 1 and 8 of each 21-day cycle).

About Triple-Negative Breast Cancer (TNBC)

TNBC is an aggressive type of breast cancer that characteristically has a high recurrence rate within the first five years after diagnosis. While some breast cancers may test positive for estrogen receptor, progesterone receptor or human epidermal growth factor receptor 2 (HER2), TNBC tests negative for all three. As a result, TNBC does not respond to therapies targeting these markers, making it more difficult to treat. Approximately 15-20% of patients with breast cancer are diagnosed with TNBC.

About KEYTRUDA (pembrolizumab) Injection, 100 mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,000 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least 1 other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10], as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause adrenal insufficiency (primary and secondary), hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Adrenal insufficiency occurred in 0.8% (22/2799) of patients, including Grade 2 (0.3%), 3 (0.3%), and 4 (<0.1%). Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of adrenal insufficiency, hypophysitis (including hypopituitarism), thyroid function (prior to and periodically during treatment), and hyperglycemia. For adrenal insufficiency or hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 adrenal insufficiency or hypophysitis and withhold or discontinue KEYTRUDA for Grade 3 or Grade 4 adrenal insufficiency or hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients with advanced NSCLC; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC; the most common was pneumonitis (1.8%). The most common adverse reactions (20%) were decreased appetite (25%), fatigue (25%), dyspnea (23%), and nausea (20%).

Adverse reactions occurring in patients with SCLC were similar to those occurring in patients with other solid tumors who received KEYTRUDA as a single agent.

In KEYNOTE-048, KEYTRUDA monotherapy was discontinued due to adverse events in 12% of 300 patients with HNSCC; the most common adverse reactions leading to permanent discontinuation were sepsis (1.7%) and pneumonia (1.3%). The most common adverse reactions (20%) were fatigue (33%), constipation (20%), and rash (20%).

In KEYNOTE-048, when KEYTRUDA was administered in combination with platinum (cisplatin or carboplatin) and FU chemotherapy, KEYTRUDA was discontinued due to adverse reactions in 16% of 276 patients with HNSCC. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonia (2.5%), pneumonitis (1.8%), and septic shock (1.4%). The most common adverse reactions (20%) were nausea (51%), fatigue (49%), constipation (37%), vomiting (32%), mucosal inflammation (31%), diarrhea (29%), decreased appetite (29%), stomatitis (26%), and cough (22%).

In KEYNOTE-012, KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (20%) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of facial edema and new or worsening hypothyroidism.

In KEYNOTE-087, KEYTRUDA was discontinued due to adverse reactions in 5% of 210 patients with cHL. Serious adverse reactions occurred in 16% of patients; those 1% included pneumonia, pneumonitis, pyrexia, dyspnea, GVHD, and herpes zoster. Two patients died from causes other than disease progression; 1 from GVHD after subsequent allogeneic HSCT and 1 from septic shock. The most common adverse reactions (20%) were fatigue (26%), pyrexia (24%), cough (24%), musculoskeletal pain (21%), diarrhea (20%), and rash (20%).

In KEYNOTE-170, KEYTRUDA was discontinued due to adverse reactions in 8% of 53 patients with PMBCL. Serious adverse reactions occurred in 26% of patients and included arrhythmia (4%), cardiac tamponade (2%), myocardial infarction (2%), pericardial effusion (2%), and pericarditis (2%). Six (11%) patients died within 30 days of start of treatment. The most common adverse reactions (20%) were musculoskeletal pain (30%), upper respiratory tract infection and pyrexia (28% each), cough (26%), fatigue (23%), and dyspnea (21%).

In KEYNOTE-052, KEYTRUDA was discontinued due to adverse reactions in 11% of 370 patients with locally advanced or metastatic urothelial carcinoma. Serious adverse reactions occurred in 42% of patients; those 2% were urinary tract infection, hematuria, acute kidney injury, pneumonia, and urosepsis. The most common adverse reactions (20%) were fatigue (38%), musculoskeletal pain (24%), decreased appetite (22%), constipation (21%), rash (21%), and diarrhea (20%).

In KEYNOTE-045, KEYTRUDA was discontinued due to adverse reactions in 8% of 266 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reaction resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.9%). Serious adverse reactions occurred in 39% of KEYTRUDA-treated patients; those 2% were urinary tract infection, pneumonia, anemia, and pneumonitis. The most common adverse reactions (20%) in patients who received KEYTRUDA were fatigue (38%), musculoskeletal pain (32%), pruritus (23%), decreased appetite (21%), nausea (21%), and rash (20%).

In KEYNOTE-057, KEYTRUDA was discontinued due to adverse reactions in 11% of 148 patients with high-risk NMIBC. The most common adverse reaction resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.4%). Serious adverse reactions occurred in 28% of patients; those 2% were pneumonia (3%), cardiac ischemia (2%), colitis (2%), pulmonary embolism (2%), sepsis (2%), and urinary tract infection (2%). The most common adverse reactions (20%) were fatigue (29%), diarrhea (24%), and rash (24%).

Adverse reactions occurring in patients with gastric cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

Adverse reactions occurring in patients with esophageal cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

In KEYNOTE-158, KEYTRUDA was discontinued due to adverse reactions in 8% of 98 patients with recurrent or metastatic cervical cancer. Serious adverse reactions occurred in 39% of patients receiving KEYTRUDA; the most frequent included anemia (7%), fistula, hemorrhage, and infections [except urinary tract infections] (4.1% each). The most common adverse reactions (20%) were fatigue (43%), musculoskeletal pain (27%), diarrhea (23%), pain and abdominal pain (22% each), and decreased appetite (21%).

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Merck's KEYTRUDA (pembrolizumab) in Combination with Chemotherapy Met Primary Endpoint of Progression-Free Survival (PFS) as First-Line Treatment for...

Cardiac Stem Cells Comments Off on Merck’s KEYTRUDA (pembrolizumab) in Combination with Chemotherapy Met Primary Endpoint of Progression-Free Survival (PFS) as First-Line Treatment for… | February 14th, 2020

Cardiac rhythm management refers to a process of monitoring functioning of the heart through devices. Cardiac rhythm management devices are used to provide therapeutic solutions to patients suffering from cardiac disorders such as cardiac arrhythmias, heart failure, and cardiac arrests. Cardiac disorders lead to irregular heartbeat. Technological advancements and rise in the number of deaths due to increasing incidences of heart diseases and increasing aging population are some of the major factors driving the cardiac rhythm management market. Heart disease is one of the primary causes of death in the U. S. Excess of alcohol consumption; smoking, high cholesterol levels, and obesity are some of the major causes of heart diseases. Cardiac rhythm management is conducted through two major devices: implantable cardiac rhythm devices and pacemakers. Implantable cardiac rhythm devices treat patients with an improper heartbeat. Based on the device, the cardiac rhythm management market can be segmented into defibrillators, pacemakers, cardiac resynchronization therapy devices, implantable defibrillators, and external defibrillators. Pacemakers are used to treat patients with a slow heartbeat. Based on the end user, the cardiac rhythm management market can be segmented into hospitals, home/ambulatory, and others.

North America has the largest market for cardiac rhythm management due to improved healthcare infrastructure, government initiatives, rise in incidences of cardiac disorders, growing number of deaths due to cardiovascular diseases,and increasing healthcare expenditure in the region. The North America market for cardiac rhythm management is followed by Europe. Asia is expected to witness high growth rate in the cardiac rhythm management market in the next few years due to increasing incidences of cardiovascular diseases, growing disposable income, rise in awareness regarding heart disorders and relevant treatments, and improving healthcare infrastructure in the region.

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Increasing the prevalence of cardiovascular diseases, technological advancements, rise in life expectancy, increasing awareness regarding cardiac disorders, and government initiatives are some of the major factors that are expected to drive the market for cardiac rhythm management. In addition, factors such as a rise in disposable income, increasing aging population, and high cost associated with heart disease treatment are expected to drive the market for cardiac rhythm management. However, economic downturn, reimbursement issues, the importance of biologics and stem cells, and inappropriate use of the devices are some of the factors restraining the growth of the global cardiac rhythm management market.

Growing population and economies in the developing countries such as India and China are expected to drive the growth of the cardiac rhythm management market in Asia. In addition,factors such as innovations along with technological advancements such as miniaturization, introduction of MRI pacemakers, biocompatible materials and durable batteries, and continuous rise in aging population and increasing cardiovascular diseases such as arrhythmias, stroke, and high blood pressure are expected to create new opportunities for the global cardiac rhythm management market. An increasing number of mergers and acquisitions, rise in the number of collaborations and partnerships, and new product launches are some of the latest trends in the global cardiac rhythm management market.

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Some of the major companies operating in the global cardiac rhythm management market are Medtronic, Abbott Laboratories, Boston Scientific, St. Jude Medical, Altera, and Sorin. Other companies with significant presence in the global cardiac rhythm management market include

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Cardiac Rhythm Management Market to Witness Rapid Increase in Consumption During 2015 2021 - Lake Shore Gazette

Cardiac Stem Cells Comments Off on Cardiac Rhythm Management Market to Witness Rapid Increase in Consumption During 2015 2021 – Lake Shore Gazette | February 13th, 2020

CALGARY, Alberta, Feb. 10, 2020 (GLOBE NEWSWIRE) -- Hemostemix Inc. (Hemostemix or the Company) (TSXV: HEM; OTC: HMTXF) is pleased to announce the appointment of Dr. Ronnie Hershman, M.D., F.C.C.S., to its Board of Directors. Dr. Hershman is a successful, practicing cardiologist with over three decades of experience. Dr. Hershman graduated Magna Cum Laude from the Sophie Davis Center for Biomedical Research in 1980 and received his medical degree from Mount Sinai Medical Center in 1982. He then continued his medical and cardiovascular training at Mt. Sinai Medical Center.

Dr. Hershman has been an Invasive Cardiologist since 1987 and was involved in many clinical trials for emerging catheter technologies. He was a pioneer in performing laser-assisted coronary angioplasty, starting in private practice on Long Island in 1989. Presently the Medical Director of NYU Langone Long Island Cardiac Care he built and manages a large medical practice, employing cutting-edge technology and continues his practice for patients with cardiovascular and peripheral vascular diseases, employing a non-invasive therapy for patients with intractable Angina and Congestive Heart Failure.

Dr. Hershman has also been an entrepreneur and investor for more than two decades. He has been involved in life science investing and consulting for several years and previously or currently serves on the boards of medical biotechnology companies Solubest, Ltd., TheraVitae Inc., Nasus Pharma, SanoNash and Optivasive. He also serves as an advisor to a latestage, life science venture capital company that has funded 24 companies to-date. Dr. Hershman is now an investor in OurCrowd, Ltd., a leading crowd funding company and is the Co-Founder and CEO of HealthEffect, LLC and CLiHealth, LLC, SoLoyal and Nasus Pharma along with SanoNash.

Dr. Hershman continues to evaluate new medical technologies in the USA and Israel. His main interests lie in bringing improved medical technologies from the bench to the clinic, quickly and globally. He is actively seeking to commercialize technologies that improve lives and cure illnesses in the most effective and cost efficient manner.

Stem Cell therapies are the future in so many chronic illnesses and Hemostemix is an exciting company with a lot of promise in providing solutions and therapeutic options for many patients with critical Cardiovascular illnesses and ischemia, commented Dr. Hershman. As an investor and Board Member, I hope to assist in advancing these therapies further and create optimal value for patients and shareholders, alike, he said.

Dr. Hershman is replacing Mr. Yari Nieken and Mr. Bryson Goodwin who both resigned from their positions with the Company effective February 10, 2020. Ms. Natasha Sever has also resigned from the position of CFO. The Company will look for suitable replacements for both CEO and CFO positions and Mr. Smeenk will act as the interim CEO until a replacement is hired. The Company thanks Bryson, Yari and Natasha for their service and wishes them well in their future endeavors.

It is a great pleasure to welcome Dr. Hershman to the Board of Directors, said David Wood, Chairman, as he compliments us with his broad medical experience, biotechnology and business investment acumen and counsel.

I am honored and delighted to welcome Dr. Hershman to the Board of Directors and I very much look forward to his counsel, said Thomas Smeenk, President.

The Company also announces that on January 9, 2020, J.M. Wood Investment Inc. (JMWI) sent the Company a Notice of Default and Demand for the immediate repayment of the Companys previously announced convertible debenture and demand loan. Based on the repayment conditions of the debts, the Company took the position the January 9th notice was premature. On January 24th, JMWI made an application to the Court of Queens Bench of Alberta for the issuance of an order appointing a receiver. The Company responded with a 347 page affidavit including appendices, sworn on January 30th by David Wood, Chairman. The application was heard on January 31st by Madame Justice Horner, who granted a consent order to adjourn the JMWI receivership application to February 20, 2020 to enable the Company to close its financing; granted an order appointing Grant Thornton as inspector; granted an order that the costs of the application of January 31st would only be payable by the Company if the application proceeds on February 20th. On February 6, 2020 cross examinations on the Affidavits of David Wood and JMWI were heard.

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Also, on February 3, 2020 the Company received an action from Aspire Health Science, LLC filed with the Ninth Judicial Circuit Court for Orange County, State of Florida, in connection with the Amended and Restated License Agreement rescinded by Hemostemix on December 5, 2019 due to Aspires failure to meet the Condition Precedent of paying US$1,000,000 within 30 business days of September 30, 2019. The Company believes the action is frivolous, without merit, and it intends to vigorously defend its position.

The Company intends to effect repayment of the secured debts and it will provide a further update to the market at that time. Although the Company is optimistic that it will be successful in raising sufficient funds to meet its obligations, there can be no assurance that the financing will close as anticipated or within the time frames required.

ABOUT HEMOSTEMIX INC.

Hemostemix is a publicly traded autologous stem cell therapy company, founded in 2003. A winner of the World Economic Forum Technology Pioneer Award, the Company developed and is commercializing its lead product ACP-01 for the treatment of CLI, PAD, Angina, Ischemic Cardiomyopathy, Dilated Cardiomyopathy and other heart conditions. ACP-01 has been used to treat over 300 patients, including no-option end-stage heart disease patients, and it has been the subject of four open label phase II clinical studies which proved its safety and efficacy.

On October 21, 2019, the Company announced the results from its presentation from its Phase II CLI trial abstract presentation entitled Autologous Stem Cell Treatment for CLI Patients with No Revascularization Options: An Update of the Hemostemix ACP-01 Trial With 4.5 Year Followup which noted healing of ulcers and resolution of ischemic rest pain occurred in 83% of patients, with outcomes maintained for up to 4.5 years. The Companys clinical trial for CLI is ongoing at 20 clinical sites in North America and 56 of 95 subjects have been enrolled to-date.

The Company owns 91 patents across five patent families titled: Regulating Stem Cells, In Vitro Techniques for use with Stem Cells, Production from Blood of Cells of Neural Lineage, and Automated Cell Therapy. For more information, please visit http://www.hemostemix.com.

Contact:

Thomas Smeenk, President & CEO Suite 1150, 707 7th Avenue S.W.Calgary, Alberta T2P 3H6Tel: 905-580-4170

Neither the TSX Venture Exchange nor its Regulation Service Provider (as that term is defined under the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Forward-Looking Statements

This release may contain forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words expects, plans, anticipates, believes, intends, estimates, projects, potential, and similar expressions, or that events or conditions will, would, may, could, or should occur. Although Hemostemix believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and actual results may differ materially from those in forward-looking statements. Forward-looking statements are based on the beliefs, estimates, and opinions of Hemostemix management on the date such statements were made. By their nature forward-looking statements are subject to known and unknown risks, uncertainties, and other factors which may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, but are not limited to, the Companys ability to fund operations and access the capital required to continue operations and repay its secured debts, the Companys stage of development, the ability to complete its current CLI clinical trial, complete a futility analysis and the results of such, future clinical trials and results, long-term capital requirements and future developments in the Companys markets and the markets in which it expects to compete, risks associated with its strategic alliances and the impact of entering new markets on the Companys operations. Each factor should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. Hemostemix expressly disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events, or otherwise. Additional information identifying risks and uncertainties are contained in the Companys filing with the Canadian securities regulators, which filings are available at http://www.sedar.com.

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Hemostemix Announces the Appointment of Dr. Ronnie Hershman to the Board of Directors and Provides a Corporate Update - Yahoo Finance

Cardiac Stem Cells Comments Off on Hemostemix Announces the Appointment of Dr. Ronnie Hershman to the Board of Directors and Provides a Corporate Update – Yahoo Finance | February 11th, 2020

Autologous stem cell and non-stem cell based therapies is a significant therapeutic intervention that makes use of an individuals cells which are then cultured and reintroduced into the donor patients body. In the context of China-US trade war and global economic volatility and uncertainty, it will have a big influence on this market. Autologous Stem Cell and Non-Stem Cell Based Therapies Report by Material, Application, and Geography Global Forecast to 2023 is a professional and comprehensive research report on the worlds major regional market conditions, focusing on the main regions (North America, Europe and Asia-Pacific) and the main countries (United States, Germany, United Kingdom, Japan, South Korea and China).

In this report, the global Autologous Stem Cell and Non-Stem Cell Based Therapies market is valued at USD XX million in 2019 and is projected to reach USD XX million by the end of 2023, growing at a CAGR of XX% during the period 2019 to 2023.

The report firstly introduced the Autologous Stem Cell and Non-Stem Cell Based Therapies basics: definitions, classifications, applications and market overview; product specifications; manufacturing processes; cost structures, raw materials and so on. Then it analyzed the worlds main region market conditions, including the product price, profit, capacity, production, supply, demand and market growth rate and forecast etc. In the end, the report introduced new project SWOT analysis, investment feasibility analysis, and investment return analysis.

The major players profiled in this report include:U.S. STEM CELL, INC.Brainstorm Cell TherapeuticsCytoriDendreon CorporationFibrocellLion BiotechnologiesCaladrius BiosciencesOpexa TherapeuticsOrgenesisRegenexxGenzymeAntriaRegeneusMesoblastPluristem Therapeutics IncTigenixMed cell EuropeHolostemMiltenyi Biotec

The end users/applications and product categories analysis:On the basis of product, this report displays the sales volume, revenue (Million USD), product price, market share and growth rate of each type, primarily split into-Embryonic Stem CellResident Cardiac Stem CellsAdult Bone MarrowDerived Stem CellsUmbilical Cord Blood Stem Cells

On the basis on the end users/applications, this report focuses on the status and outlook for major applications/end users, sales volume, market share and growth rate of Autologous Stem Cell and Non-Stem Cell Based Therapies for each application, including-Neurodegenerative DisordersAutoimmune DiseasesCancer and TumorsCardiovascular Diseases

Table of Contents

Part I Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Overview?Chapter One Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Overview1.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Definition1.2 Autologous Stem Cell and Non-Stem Cell Based Therapies Classification Analysis1.2.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Main Classification Analysis1.2.2 Autologous Stem Cell and Non-Stem Cell Based Therapies Main Classification Share Analysis1.3 Autologous Stem Cell and Non-Stem Cell Based Therapies Application Analysis1.3.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Main Application Analysis1.3.2 Autologous Stem Cell and Non-Stem Cell Based Therapies Main Application Share Analysis1.4 Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Chain Structure Analysis1.5 Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Development Overview1.5.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Product History Development Overview1.5.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Product Market Development Overview1.6 Autologous Stem Cell and Non-Stem Cell Based Therapies Global Market Comparison Analysis1.6.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Global Import Market Analysis1.6.2 Autologous Stem Cell and Non-Stem Cell Based Therapies Global Export Market Analysis1.6.3 Autologous Stem Cell and Non-Stem Cell Based Therapies Global Main Region Market Analysis1.6.4 Autologous Stem Cell and Non-Stem Cell Based Therapies Global Market Comparison Analysis1.6.5 Autologous Stem Cell and Non-Stem Cell Based Therapies Global Market Development Trend Analysis

Chapter Two Autologous Stem Cell and Non-Stem Cell Based Therapies Up and Down Stream Industry Analysis2.1 Upstream Raw Materials Analysis2.1.1 Proportion of Manufacturing Cost2.1.2 Manufacturing Cost Structure of Autologous Stem Cell and Non-Stem Cell Based Therapies Analysis2.2 Down Stream Market Analysis2.2.1 Down Stream Market Analysis2.2.2 Down Stream Demand Analysis2.2.3 Down Stream Market Trend Analysis

Part II Asia Autologous Stem Cell and Non-Stem Cell Based Therapies Industry (The Report Company Including the Below Listed But Not All)

Chapter Three Asia Autologous Stem Cell and Non-Stem Cell Based Therapies Market Analysis3.1 Asia Autologous Stem Cell and Non-Stem Cell Based Therapies Product Development History3.2 Asia Autologous Stem Cell and Non-Stem Cell Based Therapies Competitive Landscape Analysis3.3 Asia Autologous Stem Cell and Non-Stem Cell Based Therapies Market Development Trend

Chapter Four 2014-2019 Asia Autologous Stem Cell and Non-Stem Cell Based Therapies Productions Supply Sales Demand Market Status and Forecast4.1 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Overview4.2 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Market Share Analysis4.3 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Demand Overview4.4 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Supply Demand and Shortage4.5 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Import Export Consumption4.6 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Cost Price Production Value Gross Margin

Chapter Five Asia Autologous Stem Cell and Non-Stem Cell Based Therapies Key Manufacturers Analysis5.1 Company A5.1.1 Company Profile5.1.2 Product Picture and Specification5.1.3 Product Application Analysis5.1.4 Capacity Production Price Cost Production Value5.1.5 Contact Information5.2 Company B5.2.1 Company Profile5.2.2 Product Picture and Specification5.2.3 Product Application Analysis5.2.4 Capacity Production Price Cost Production Value5.2.5 Contact Information5.3 Company C5.3.1 Company Profile5.3.2 Product Picture and Specification5.3.3 Product Application Analysis5.3.4 Capacity Production Price Cost Production Value5.3.5 Contact Information5.4 Company D5.4.1 Company Profile5.4.2 Product Picture and Specification5.4.3 Product Application Analysis5.4.4 Capacity Production Price Cost Production Value5.4.5 Contact InformationChapter Six Asia Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Development Trend6.1 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Overview6.2 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Market Share Analysis6.3 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Demand Overview6.4 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Supply Demand and Shortage6.5 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Import Export Consumption6.6 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Cost Price Production Value Gross Margin

Part III North American Autologous Stem Cell and Non-Stem Cell Based Therapies Industry (The Report Company Including the Below Listed But Not All)

Chapter Seven North American Autologous Stem Cell and Non-Stem Cell Based Therapies Market Analysis7.1 North American Autologous Stem Cell and Non-Stem Cell Based Therapies Product Development History7.2 North American Autologous Stem Cell and Non-Stem Cell Based Therapies Competitive Landscape Analysis7.3 North American Autologous Stem Cell and Non-Stem Cell Based Therapies Market Development Trend

Chapter Eight 2014-2019 North American Autologous Stem Cell and Non-Stem Cell Based Therapies Productions Supply Sales Demand Market Status and Forecast8.1 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Overview8.2 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Market Share Analysis8.3 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Demand Overview8.4 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Supply Demand and Shortage8.5 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Import Export Consumption8.6 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Cost Price Production Value Gross Margin

Chapter Nine North American Autologous Stem Cell and Non-Stem Cell Based Therapies Key Manufacturers Analysis9.1 Company A9.1.1 Company Profile9.1.2 Product Picture and Specification9.1.3 Product Application Analysis9.1.4 Capacity Production Price Cost Production Value9.1.5 Contact Information9.2 Company B9.2.1 Company Profile9.2.2 Product Picture and Specification9.2.3 Product Application Analysis9.2.4 Capacity Production Price Cost Production Value9.2.5 Contact InformationChapter Ten North American Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Development Trend10.1 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Overview10.2 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Market Share Analysis10.3 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Demand Overview10.4 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Supply Demand and Shortage10.5 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Import Export Consumption10.6 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Cost Price Production Value Gross Margin

Part IV Europe Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Analysis (The Report Company Including the Below Listed But Not All)

Chapter Eleven Europe Autologous Stem Cell and Non-Stem Cell Based Therapies Market Analysis11.1 Europe Autologous Stem Cell and Non-Stem Cell Based Therapies Product Development History11.2 Europe Autologous Stem Cell and Non-Stem Cell Based Therapies Competitive Landscape Analysis11.3 Europe Autologous Stem Cell and Non-Stem Cell Based Therapies Market Development Trend

Chapter Twelve 2014-2019 Europe Autologous Stem Cell and Non-Stem Cell Based Therapies Productions Supply Sales Demand Market Status and Forecast12.1 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Overview12.2 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Market Share Analysis12.3 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Demand Overview12.4 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Supply Demand and Shortage12.5 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Import Export Consumption12.6 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Cost Price Production Value Gross Margin

Chapter Thirteen Europe Autologous Stem Cell and Non-Stem Cell Based Therapies Key Manufacturers Analysis13.1 Company A13.1.1 Company Profile13.1.2 Product Picture and Specification13.1.3 Product Application Analysis13.1.4 Capacity Production Price Cost Production Value13.1.5 Contact Information13.2 Company B13.2.1 Company Profile13.2.2 Product Picture and Specification13.2.3 Product Application Analysis13.2.4 Capacity Production Price Cost Production Value13.2.5 Contact InformationChapter Fourteen Europe Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Development Trend14.1 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Overview14.2 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Market Share Analysis14.3 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Demand Overview14.4 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Supply Demand and Shortage14.5 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Import Export Consumption14.6 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Cost Price Production Value Gross Margin

Part V Autologous Stem Cell and Non-Stem Cell Based Therapies Marketing Channels and Investment Feasibility

Chapter Fifteen Autologous Stem Cell and Non-Stem Cell Based Therapies Marketing Channels Development Proposals Analysis15.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Marketing Channels Status15.2 Autologous Stem Cell and Non-Stem Cell Based Therapies Marketing Channels Characteristic15.3 Autologous Stem Cell and Non-Stem Cell Based Therapies Marketing Channels Development Trend15.2 New Firms Enter Market Strategy15.3 New Project Investment Proposals

Chapter Sixteen Development Environmental Analysis16.1 China Macroeconomic Environment Analysis16.2 European Economic Environmental Analysis16.3 United States Economic Environmental Analysis16.4 Japan Economic Environmental Analysis16.5 Global Economic Environmental Analysis

Chapter Seventeen Autologous Stem Cell and Non-Stem Cell Based Therapies New Project Investment Feasibility Analysis17.1 Autologous Stem Cell and Non-Stem Cell Based Therapies Market Analysis17.2 Autologous Stem Cell and Non-Stem Cell Based Therapies Project SWOT Analysis17.3 Autologous Stem Cell and Non-Stem Cell Based Therapies New Project Investment Feasibility Analysis

Part VI Global Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Conclusions

Chapter Eighteen 2014-2019 Global Autologous Stem Cell and Non-Stem Cell Based Therapies Productions Supply Sales Demand Market Status and Forecast18.1 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Overview18.2 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Market Share Analysis18.3 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Demand Overview18.4 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Supply Demand and Shortage18.5 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Import Export Consumption18.6 2014-2019 Autologous Stem Cell and Non-Stem Cell Based Therapies Cost Price Production Value Gross Margin

Chapter Nineteen Global Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Development Trend19.1 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Overview19.2 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Production Market Share Analysis19.3 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Demand Overview19.4 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Supply Demand and Shortage19.5 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Import Export Consumption19.6 2019-2023 Autologous Stem Cell and Non-Stem Cell Based Therapies Cost Price Production Value Gross Margin

Chapter Twenty Global Autologous Stem Cell and Non-Stem Cell Based Therapies Industry Research Conclusions

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Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020: Industry Analysis By Emerging Trends, Key Companies, Regional Outlook and...

Cardiac Stem Cells Comments Off on Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020: Industry Analysis By Emerging Trends, Key Companies, Regional Outlook and… | February 11th, 2020

BOTHELL, Wash. and TOKYO, Feb. 10, 2020 /PRNewswire/ --Seattle Genetics, Inc.(Nasdaq: SGEN) and Astellas Pharma Inc.(TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") today announced updated results from the phase 1b/2 clinical trial EV-103 in previously untreated patients with locally advanced or metastatic urothelial cancer who were ineligible for treatment with cisplatin-based chemotherapy. Forty-five patients were treated with the combination of PADCEV (enfortumab vedotin-ejfv) and pembrolizumab and were evaluated for safety and efficacy. After a median follow-up of 11.5 months, the study results continue to meet outcome measures for safety and demonstrate encouraging clinical activity for this platinum-free combination in a first-line setting. Updated results will be presented during an oral session on Friday, February 14 at the 2020 Genitourinary Cancers Symposium in San Francisco (Abstract #441). Initial results from the study were presented at the European Society of Medical Oncology Congress in September 2019.

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.1,2

"Cisplatin-basedchemotherapy is the standard treatment for first-line advanced urothelial cancer; however, it isn't an option for many patients,"said Jonathan E. Rosenberg, M.D., Medical Oncologist and Chief, Genitourinary Medical Oncology Service at Memorial Sloan Kettering Cancer Center in New York."I'm encouraged by these interim results, including a median progression-free survival of a year for patients who received the platinum-free combination of PADCEV and pembrolizumab in the first-line setting."

In the study, 58 percent (26/45) of patients had a treatment-related adverse event greater than or equal to Grade 3: increase in lipase (18 percent; 8/45), rash (13 percent; 6/45), hyperglycemia (13 percent; 6/45) and peripheral neuropathy (4 percent; 2/45); these rates were similar to those observed with PADCEV monotherapy.3Eighteen percent (8/45) of patients had treatment-related immune-mediated adverse events of clinical interest greater than or equal to Grade 3 that required the use of systemic steroids (arthralgia, dermatitis bullous, pneumonitis, lipase increased, rash erythematous, rash maculo-papular, tubulointerstitial nephritis, myasthenia gravis). None of the adverse events of clinical interest were Grade 5 events. Six patients (13 percent) discontinued treatment due to treatment-related adverse events, most commonly peripheral sensory neuropathy. As previously reported, there was one death deemed to be treatment-related by the investigator attributed to multiple organ dysfunction syndrome.

The data demonstrated the combination of PADCEV plus pembrolizumab shrank tumors in the majority of patients, resulting in a confirmed objective response rate (ORR) of 73.3 percent (33/45; 95% Confidence Interval (CI): 58.1, 85.4) after a median follow-up of 11.5 months (range,0.7 to 19.2). Responses included 15.6 percent (7/45) of patients who had a complete response (CR)and 57.8 percent (26/45) of patients who had a partial response. Median duration of response has not yet been reached (range 1.2 to 12.9+ months). Eighteen (55%) of 33 responses were ongoing at the time of analysis, with 83.9% of responses lasting at least 6 months and 53.7% of responses lasting at least 12 months (Kaplan-Meier estimate).The median progression-free survival was 12.3 months (95% CI: 7.98, -) and the 12-month overall survival (OS) rate was 81.6 percent (95% CI: 62 to 91.8 percent); median OS has not been reached.

"These updated data are encouraging and provide support for the recently initiated phase 3 trial EV-302 that includes an arm evaluating PADCEV in this platinum-free combination in the first-line setting," said Roger Dansey, M.D., Chief Medical Officer at Seattle Genetics.

"These additional results support continued evaluation of PADCEV in combination with other agents and at earlier stages of treatment for patients withurothelial cancer," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head at Astellas.

About the EV-103 TrialEV-103 is an ongoing, multi-cohort, open-label, multicenter phase 1b/2 trial of PADCEV alone or in combination, evaluating safety, tolerability and efficacy in muscle invasive, locally advanced and first- and second-line metastatic urothelial cancer.

The dose-escalation cohort and expansion cohort A include locally advanced or metastatic urothelial cancer patients who are ineligible for cisplatin-based chemotherapy. Patients were dosed in a 21-day cycle, receiving an intravenous (IV) infusion of enfortumab vedotin on Days 1 and 8 and pembrolizumab on Day 1. At the time of this initial analysis, 45 patients (5 from the dose-escalation cohort and 40 from the dose-expansion cohort A) with locally advanced and/or metastatic urothelial cancer had been treated with enfortumab vedotin (1.25 mg/kg) plus pembrolizumab in the first-line setting.

The primary outcome measure of the cohorts included in this analysis is safety. Key secondary objectives related to efficacy include objective response rate (ORR), disease control rate (DCR), duration of response (DoR), progression free survival (PFS) and overall survival (OS). DoR,PFS and OS are not yet mature.

Additional cohorts in the EV-103 study will evaluate enfortumab vedotin:

More information about PADCEV clinical trials can be found at clinicaltrials.gov.

About Bladder and Urothelial CancerIt is estimated that approximately 81,000 people in the U.S. will be diagnosed with bladder cancer in 2020.5 Urothelial cancer accounts for 90 percent of all bladder cancers and can also be found in the renal pelvis, ureter and urethra.6 Globally, approximately 549,000 people were diagnosed with bladder cancer in 2018, and there were approximately 200,000 deaths worldwide.7

The recommended first-line treatment for patients with advanced urothelial cancer is a cisplatin-based chemotherapy. For patients who are ineligible for cisplatin, such as people with kidney impairment, a carboplatin-based regimen is recommended. However, fewer than half of patients respond to carboplatin-based regimens and outcomes are typically poorer compared to cisplatin-based regimens.8

About PADCEV PADCEV (enfortumabvedotin-ejfv) was approved by the U.S. Food and Drug Administration (FDA) in December 2019 and is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a platinum-containing chemotherapy before (neoadjuvant) or after (adjuvant) surgery or in a locally advanced or metastatic setting. PADCEV was approved under the FDA's Accelerated Approval Program based on tumor response rate. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.9

PADCEV is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.2,9Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4 expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).9PADCEV is co-developed by Astellas and Seattle Genetics.

Important Safety Information

Warnings and Precautions

Adverse ReactionsSerious adverse reactions occurred in 46% of patients treated with PADCEV. The most common serious adverse reactions (3%) were urinary tract infection (6%), cellulitis (5%), febrile neutropenia (4%), diarrhea (4%), sepsis (3%), acute kidney injury (3%), dyspnea (3%), and rash (3%). Fatal adverse reactions occurred in 3.2% of patients, including acute respiratory failure, aspiration pneumonia, cardiac disorder, and sepsis (each 0.8%).

Adverse reactions leading to discontinuation occurred in 16% of patients; the most common adverse reaction leading to discontinuation was peripheral neuropathy (6%). Adverse reactions leading to dose interruption occurred in 64% of patients; the most common adverse reactions leading to dose interruption were peripheral neuropathy (18%), rash (9%) and fatigue (6%). Adverse reactions leading to dose reduction occurred in 34% of patients; the most common adverse reactions leading to dose reduction were peripheral neuropathy (12%), rash (6%) and fatigue (4%).

The most common adverse reactions (20%) were fatigue (56%), peripheral neuropathy (56%), decreased appetite (52%), rash (52%), alopecia (50%), nausea (45%), dysgeusia (42%), diarrhea (42%), dry eye (40%), pruritus (26%) and dry skin (26%). The most common Grade 3 adverse reactions (5%) were rash (13%), diarrhea (6%) and fatigue (6%).

Lab AbnormalitiesIn one clinical trial, Grade 3-4 laboratory abnormalities reported in 5% were: lymphocytes decreased, hemoglobin decreased, phosphate decreased, lipase increased, sodium decreased, glucose increased, urate increased, neutrophils decreased.

Drug Interactions

Specific Populations

For more information, please see the full Prescribing Information for PADCEV here.

About Seattle GeneticsSeattle Genetics, Inc. is a global biotechnology company that discovers, develops and commercializes transformative medicines targeting cancer to make a meaningful difference in people's lives. The company is headquartered in Bothell, Washington, and has offices in California, Switzerland and the European Union. For more information on our robust pipeline, visit https://www.seattlegenetics.comand follow @SeattleGenetics on Twitter.

About AstellasAstellas Pharma Inc., based in Tokyo, Japan, is a company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products. For more information, please visit our website at https://www.astellas.com/en.

About the Astellas and Seattle Genetics CollaborationSeattle Genetics and Astellas are co-developing enfortumab vedotin-ejfv under a collaboration that was entered into in 2007 and expanded in 2009. Under the collaboration, the companies are sharing costs and profits on a 50:50 basis worldwide.

Seattle Genetics Forward-Looking StatementsCertain statements made in this press release are forward looking, such as those, among others, relating to the EV-103 and EV-302 clinical trials; clinical development plans relating to enfortumab vedotin; the therapeutic potential of enfortumab vedotin; and its possible safety, efficacy, and therapeutic uses, including in the first-line setting. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the possibility that ongoing and subsequent clinical trials of enfortumab vedotin may fail to establish sufficient efficacy; that adverse events or safety signals may occur and that adverse regulatory actions or other setbacks could occur as enfortumab vedotin advances in clinical trials even after promising results in earlier clinical trials. More information about the risks and uncertainties faced by Seattle Genetics is contained under the caption "Risk Factors" included in the company's Annual Report on Form 10-K for the year ended December 31, 2019 filed with the Securities and Exchange Commission. Seattle Genetics disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Astellas Cautionary NotesIn this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties.

Information about pharmaceutical products (including products currently in development), which is included in this press release is not intended to constitute an advertisement or medical advice.

1 PADCEV [package insert]. Northbrook, IL: Astellas, Inc.2 Challita-Eid P, Satpayev D, Yang P, et al. Enfortumab Vedotin Antibody-Drug Conjugate Targeting Nectin-4 Is a Highly Potent Therapeutic Agent in Multiple Preclinical Cancer Models. Cancer Res 2016;76(10):3003-13.3 Rosenberg JE, O'Donnell PH, Balar AV, et al. Pivotal Trial of Enfortumab Vedotin in Urothelial Carcinoma After Platinum and Anti-Programmed Death 1/Programmed Death Ligand 1 Therapy. J Clin Oncol 2019;37(29):2592-600.4 ClinicalTrials.gov. A Study of Enfortumab Vedotin Alone or With Other Therapies for Treatment of Urothelial Cancer (EV-103). https://clinicaltrials.gov/ct2/show/NCT03288545.5 American Cancer Society. Cancer Facts & Figures 2020. https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2020/cancer-facts-and-figures-2020.pdf. Accessed 01-23-2020.6National Cancer Institute. Surveillance, Epidemiology, and End Results Program. Cancer stat facts: bladder cancer. https://seer.cancer.gov/statfacts/html/urinb.html. Accessed 05-01-2019.7International Agency for Research on Cancer. Cancer Tomorrow: Bladder. http://gco.iarc.fr/tomorrow. 8 National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology: Bladder Cancer. Version 4; July 10, 2019. https://www.nccn.org/professionals/physician_gls/pdf/bladder.pdf.9 PADCEV [package insert]. Northbrook, IL: Astellas, Inc.

SOURCE Astellas

http://www.seattlegenetics.com

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Seattle Genetics and Astellas Announce Updated Results from Phase 1b/2 Trial of PADCEV (enfortumab vedotin-ejfv) in Combination with Immune Therapy...

Cardiac Stem Cells Comments Off on Seattle Genetics and Astellas Announce Updated Results from Phase 1b/2 Trial of PADCEV (enfortumab vedotin-ejfv) in Combination with Immune Therapy… | February 11th, 2020

Regenerative Medicine Market: Snapshot

Regenerative medicine is a part of translational research in the fields of molecular biology and tissue engineering. This type of medicine involves replacing and regenerating human cells, organs, and tissues with the help of specific processes. Doing this may involve a partial or complete reengineering of human cells so that they start to function normally.

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Regenerative medicine also involves the attempts to grow tissues and organs in a laboratory environment, wherein they can be put in a body that cannot heal a particular part. Such implants are mainly preferred to be derived from the patients own tissues and cells, particularly stem cells. Looking at the promising nature of stem cells to heal and regenerative various parts of the body, this field is certainly expected to see a bright future. Doing this can help avoid opting for organ donation, thus saving costs. Some healthcare centers might showcase a shortage of organ donations, and this is where tissues regenerated using patients own cells are highly helpful.

There are several source materials from which regeneration can be facilitated. Extracellular matrix materials are commonly used source substances all over the globe. They are mainly used for reconstructive surgery, chronic wound healing, and orthopedic surgeries. In recent times, these materials have also been used in heart surgeries, specifically aimed at repairing damaged portions.

Cells derived from the umbilical cord also have the potential to be used as source material for bringing about regeneration in a patient. A vast research has also been conducted in this context. Treatment of diabetes, organ failure, and other chronic diseases is highly possible by using cord blood cells. Apart from these cells, Whartons jelly and cord lining have also been shortlisted as possible sources for mesenchymal stem cells. Extensive research has conducted to study how these cells can be used to treat lung diseases, lung injury, leukemia, liver diseases, diabetes, and immunity-based disorders, among others.

Global Regenerative Medicine Market: Overview

The global market for regenerative medicine market is expected to grow at a significant pace throughout the forecast period. The rising preference of patients for personalized medicines and the advancements in technology are estimated to accelerate the growth of the global regenerative medicine market in the next few years. As a result, this market is likely to witness a healthy growth and attract a large number of players in the next few years. The development of novel regenerative medicine is estimated to benefit the key players and supplement the markets growth in the near future.

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Global Regenerative Medicine Market: Key Trends

The rising prevalence of chronic diseases and the rising focus on cell therapy products are the key factors that are estimated to fuel the growth of the global regenerative medicine market in the next few years. In addition, the increasing funding by government bodies and development of new and innovative products are anticipated to supplement the growth of the overall market in the next few years.

On the flip side, the ethical challenges in the stem cell research are likely to restrict the growth of the global regenerative medicine market throughout the forecast period. In addition, the stringent regulatory rules and regulations are predicted to impact the approvals of new products, thus hampering the growth of the overall market in the near future.

Global Regenerative Medicine Market: Market Potential

The growing demand for organ transplantation across the globe is anticipated to boost the demand for regenerative medicines in the next few years. In addition, the rapid growth in the geriatric population and the significant rise in the global healthcare expenditure is predicted to encourage the growth of the market. The presence of a strong pipeline is likely to contribute towards the markets growth in the near future.

Global Regenerative Medicine Market: Regional Outlook

In the past few years, North America led the global regenerative medicine market and is likely to remain in the topmost position throughout the forecast period. This region is expected to account for a massive share of the global market, owing to the rising prevalence of cancer, cardiac diseases, and autoimmunity. In addition, the rising demand for regenerative medicines from the U.S. and the rising government funding are some of the other key aspects that are likely to fuel the growth of the North America market in the near future.

Furthermore, Asia Pacific is expected to register a substantial growth rate in the next few years. The high growth of this region can be attributed to the availability of funding for research and the development of research centers. In addition, the increasing contribution from India, China, and Japan is likely to supplement the growth of the market in the near future.

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Global Regenerative Medicine Market: Competitive Analysis

The global market for regenerative medicines is extremely fragmented and competitive in nature, thanks to the presence of a large number of players operating in it. In order to gain a competitive edge in the global market, the key players in the market are focusing on technological developments and research and development activities. In addition, the rising number of mergers and acquisitions and collaborations is likely to benefit the prominent players in the market and encourage the overall growth in the next few years.

Some of the key players operating in the regenerative medicine market across the globe are Vericel Corporation, Japan Tissue Engineering Co., Ltd., Stryker Corporation, Acelity L.P. Inc. (KCI Licensing), Organogenesis Inc., Medtronic PLC, Cook Biotech Incorporated, Osiris Therapeutics, Inc., Integra Lifesciences Corporation, and Nuvasive, Inc. A large number of players are anticipated to enter the global market throughout the forecast period.

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Regenerative Medicine Market Analysis Trends, Growth Opportunities, Size, Type, Dynamic Demand and Drives with Forecast to 2025 - Jewish Life News

Cardiac Stem Cells Comments Off on Regenerative Medicine Market Analysis Trends, Growth Opportunities, Size, Type, Dynamic Demand and Drives with Forecast to 2025 – Jewish Life News | February 11th, 2020

A research team featuring an expert from City University of Hong Kong (CityU) has developed a novel dual approach for the first time for concurrently rejuvenating both the cardiac muscle and vasculature of the heart by utilising two types of stem cells. The results give hope for a new treatment for repairing myocardial infarction (MI) heart.

Dr Ban Ki-won, Assistant Professor of the Department of Biomedical Sciences and his research team, including researchers from Konkuk University, The Catholic University of Korea, Pohang University of Science and Technology and T&R Biofab in South Korea, have conducted the first study of two distinct stem cell effects for cardiac repair. The two major types of stem cells employed are human bone marrow derived mesenchymal stem cells (hMSCs) and cardiomyocytes derived from human induced pluripotent stem cells (hiPSC-CMs). The research findings have been published in Nature Communications in a paper titled Dual stem cell therapy synergistically improves cardiac function and vascular regeneration following myocardial infarction.

Both cardiac muscles and vasculatures are severely damaged following MI, and so the therapeutic strategies should focus on comprehensive repair of both at the same time. But the current strategies only focus on either one, Dr Ban said.

Dr Ban said that, with limited therapeutic options for severe MI and advanced heart failure, a heart transplant was the last resort. However, such an operation is very risky, costly and subject to limited supply of suitable donors. Therefore, stem cell-based therapy has emerged as a promising therapeutic option.

In the study, the hiPSC-CMs were injected directly into the border zone of the rats heart, while the hMSCs-loaded patch was implanted on top of the infarct area, like a bandage. The results showed that this dual approach led to a significant improvement of cardiac function and an enhancement of vessel formation on a MI heart.

We believe this novel dual approach can potentially provide translational and clinical benefit to the field of cardiac regeneration. Based on the same principle, the protocol may also be utilised for repairing other organs including the brain, liver and pancreas in which multiple types of stem cells co-exist, Dr Ban added.

The research team is working on follow-up studies in larger animal models such as pigs. The patent application for this research result has been submitted.

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First dual stem cell therapy jointly developed by City University of Hong Kong brings new hope for cardiac repair - QS WOW News

Cardiac Stem Cells Comments Off on First dual stem cell therapy jointly developed by City University of Hong Kong brings new hope for cardiac repair – QS WOW News | February 10th, 2020

CALGARY, Alberta, Feb. 10, 2020 (GLOBE NEWSWIRE) --Hemostemix. (Hemostemix or the Company) (TSXV: HEM; OTC: HMTXF) is pleased to announce the appointment of Dr. Ronnie Hershman, M.D., F.C.C.S., to its Board of Directors. Dr. Hershman is a successful, practicing cardiologist with over three decades of experience. Dr. Hershman graduated Magna Cum Laude from the Sophie Davis Center for Biomedical Research in 1980 and received his medical degree from Mount Sinai Medical Center in 1982. He then continued his medical and cardiovascular training at Mt. Sinai Medical Center.

Dr. Hershman has been an Invasive Cardiologist since 1987 and was involved in many clinical trials for emerging catheter technologies. He was a pioneer in performing laser-assisted coronary angioplasty, starting in private practice on Long Island in 1989. Presently the Medical Director of NYU Langone Long Island Cardiac Care he built and manages a large medical practice, employing cutting-edge technology and continues his practice for patients with cardiovascular and peripheral vascular diseases, employing a non-invasive therapy for patients with intractable Angina and Congestive Heart Failure.

Dr. Hershman has also been an entrepreneur and investor for more than two decades. He has been involved in life science investing and consulting for several years and previously or currently serves on the boards of medical biotechnology companies Solubest, Ltd., TheraVitae Inc., Nasus Pharma, SanoNash and Optivasive. He also serves as an advisor to a latestage, life science venture capital company that has funded 24 companies to-date. Dr. Hershman is now an investor in OurCrowd, Ltd., a leading crowd funding company and is the Co-Founder and CEO of HealthEffect, LLC and CLiHealth, LLC, SoLoyal and Nasus Pharma along with SanoNash.

Dr. Hershman continues to evaluate new medical technologies in the USA and Israel. His main interests lie in bringing improved medical technologies from the bench to the clinic, quickly and globally. He is actively seeking to commercialize technologies that improve lives and cure illnesses in the most effective and cost efficient manner.

Stem Cell therapies are the future in so many chronic illnesses and Hemostemix is an exciting company with a lot of promise in providing solutions and therapeutic options for many patients with critical Cardiovascular illnesses and ischemia, commented Dr. Hershman. As an investor and Board Member, I hope to assist in advancing these therapies further and create optimal value for patients and shareholders, alike, he said.

Dr. Hershman is replacing Mr. Yari Nieken and Mr. Bryson Goodwin who both resigned from their positions with the Company effective February 10, 2020. Ms. Natasha Sever has also resigned from the position of CFO. The Company will look for suitable replacements for both CEO and CFO positions and Mr. Smeenk will act as the interim CEO until a replacement is hired. The Company thanks Bryson, Yari and Natasha for their service and wishes them well in their future endeavors.

It is a great pleasure to welcome Dr. Hershman to the Board of Directors, said David Wood, Chairman, as he compliments us with his broad medical experience, biotechnology and business investment acumen and counsel.

I am honored and delighted to welcome Dr. Hershman to the Board of Directors and I very much look forward to his counsel, said Thomas Smeenk, President.

The Company also announces that on January 9, 2020, J.M. Wood Investment Inc. (JMWI) sent the Company a Notice of Default and Demand for the immediate repayment of the Companys previously announced convertible debenture and demand loan. Based on the repayment conditions of the debts, the Company took the position the January 9th notice was premature. On January 24th, JMWI made an application to the Court of Queens Bench of Alberta for the issuance of an order appointing a receiver. The Company responded with a 347 page affidavit including appendices, sworn on January 30th by David Wood, Chairman. The application was heard on January 31st by Madame Justice Horner, who granted a consent order to adjourn the JMWI receivership application to February 20, 2020 to enable the Company to close its financing; granted an order appointing Grant Thornton as inspector; granted an order that the costs of the application of January 31st would only be payable by the Company if the application proceeds on February 20th. On February 6, 2020 cross examinations on the Affidavits of David Wood and JMWI were heard.

Also, on February 3, 2020 the Company received an action from Aspire Health Science, LLC filed with the Ninth Judicial Circuit Court for Orange County, State of Florida, in connection with the Amended and Restated License Agreement rescinded by Hemostemix on December 5, 2019 due to Aspires failure to meet the Condition Precedent of paying US$1,000,000 within 30 business days of September 30, 2019. The Company believes the action is frivolous, without merit, and it intends to vigorously defend its position.

The Company intends to effect repayment of the secured debts and it will provide a further update to the market at that time. Although the Company is optimistic that it will be successful in raising sufficient funds to meet its obligations, there can be no assurance that the financing will close as anticipated or within the time frames required.

ABOUT HEMOSTEMIX INC.

Hemostemix is a publicly traded autologous stem cell therapy company, founded in 2003. A winner of the World Economic Forum Technology Pioneer Award, the Company developed and is commercializing its lead product ACP-01 for the treatment of CLI, PAD, Angina, Ischemic Cardiomyopathy, Dilated Cardiomyopathy and other heart conditions. ACP-01 has been used to treat over 300 patients, including no-option end-stage heart disease patients, and it has been the subject of four open label phase II clinical studies which proved its safety and efficacy.

On October 21, 2019, the Company announced the results from its presentation from its Phase II CLI trial abstract presentation entitled Autologous Stem Cell Treatment for CLI Patients with No Revascularization Options: An Update of the Hemostemix ACP-01 Trial With 4.5 Year Followup which noted healing of ulcers and resolution of ischemic rest pain occurred in 83% of patients, with outcomes maintained for up to 4.5 years. The Companys clinical trial for CLI is ongoing at 20 clinical sites in North America and 56 of 95 subjects have been enrolled to-date.

The Company owns 91 patents across five patent families titled: Regulating Stem Cells, In Vitro Techniques for use with Stem Cells, Production from Blood of Cells of Neural Lineage, and Automated Cell Therapy. For more information, please visit http://www.hemostemix.com.

Contact:

Thomas Smeenk, President & CEOSuite 1150, 707 7th Avenue S.W.Calgary, Alberta T2P 3H6Tel: 905-580-4170

Neither the TSX Venture Exchange nor its Regulation Service Provider (as that term is defined under the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Forward-Looking Statements

This release may contain forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words expects, plans, anticipates, believes, intends, estimates, projects, potential, and similar expressions, or that events or conditions will, would, may, could, or should occur. Although Hemostemix believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and actual results may differ materially from those in forward-looking statements. Forward-looking statements are based on the beliefs, estimates, and opinions of Hemostemix management on the date such statements were made. By their nature forward-looking statements are subject to known and unknown risks, uncertainties, and other factors which may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, but are not limited to, the Companys ability to fund operations and access the capital required to continue operations and repay its secured debts, the Companys stage of development, the ability to complete its current CLI clinical trial, complete a futility analysis and the results of such, future clinical trials and results, long-term capital requirements and future developments in the Companys markets and the markets in which it expects to compete, risks associated with its strategic alliances and the impact of entering new markets on the Companys operations. Each factor should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. Hemostemix expressly disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events, or otherwise. Additional information identifying risks and uncertainties are contained in the Companys filing with the Canadian securities regulators, which filings are available at http://www.sedar.com.

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Hemostemix Announces the Appointment of Dr. Ronnie Hershman to the Board of Directors and Provides a Corporate Update - BioSpace

Cardiac Stem Cells Comments Off on Hemostemix Announces the Appointment of Dr. Ronnie Hershman to the Board of Directors and Provides a Corporate Update – BioSpace | February 10th, 2020

The Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market is expected to reach USD113.04 billion by 2025, from USD 87.59 billion in 2017 growing at a CAGR of 3.7% during the forecast period of 2018 to 2025. The upcoming market report contains data for historic years 2015 & 2016, the base year of calculation is 2017 and the forecast period is 2018 to 2025.

Get Exclusive FREE Sample Copy of This Report Herehttps://www.databridgemarketresearch.com/request-a-sample?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market

Some of the major players operating in the global autologous stem cell and non-stem cell based therapies market are Antria (Cro), Bioheart, Brainstorm Cell Therapeutics, Cytori, Dendreon Corporation, Fibrocell, Genesis Biopharma, Georgia Health Sciences University, Neostem, Opexa Therapeutics, Orgenesis, Regenexx, Regeneus, Tengion, Tigenix, Virxsys and many more.

Autologous Stem Cell and Non-Stem Cell Based Therapies market analysis document contains basic, secondary and advanced information related to the global status, recent trends, market size, sales volume, market share, growth, future trends analysis, segment and forecasts from 2020 2025. Market research data included in this report lend a hand to businesses for planning of strategies related to investment, revenue generation, production, product launches, costing, inventory, purchasing and marketing. Furthermore, Autologous Stem Cell and Non-Stem Cell Based Therapies report presents the data and information for actionable, most recent, and real-time market insights which makes it easier to even reach to the critical business decisions.

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Market Definition:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

In autologous stem-cell transplantation persons own undifferentiated cells or stem cells are collected and transplanted back to the person after intensive therapy. These therapies are performed by means of hematopoietic stem cells, in some of the cases cardiac cells are used to fix the damages caused due to heart attacks.

The autologous stem cell and non-stem cell based therapies are used in the treatment of various diseases such as neurodegenerative diseases, cardiovascular diseases, cancer and autoimmune diseases, infectious disease. According to World Health Organization (WHO), cardiovascular disease (CVD) causes more than half of all deaths across the European Region. The disease leads to death or frequently it is caused by AIDS, tuberculosis and malaria combined in Europe.

With the prevalence of cancer and diabetes in all age groups globally the need of steam cell based therapies is increasing, according to article published by the US National Library of Medicine National Institutes of Health, it was reported that around 382 million people had diabetes in 2013 and the number is growing at alarming rate which has increased the need to improve treatment and therapies regarding the diseases.

Market Segmentation:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

Major Autologous Stem Cell and Non-Stem Cell Based Therapies Market Drivers and Restraints:

Competitive Analysis:Global Autologous Stem Cell and Non-Stem Cell Based Therapies Market

The global autologous stem cell and non-stem cell based therapies market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of autologous stem cell and non-stem cell based therapies market for global, Europe, North America, Asia Pacific and South America.

Read Complete Details with TOC Herehttps://www.databridgemarketresearch.com/toc?dbmr=global-autologous-stem-cell-and-non-stem-cell-based-therapies-market&raksh

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Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020-2025 Business Analysis || Leading Players Fibrocell, Genesis Biopharma, Georgia...

Cardiac Stem Cells Comments Off on Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020-2025 Business Analysis || Leading Players Fibrocell, Genesis Biopharma, Georgia… | February 10th, 2020

National Research (NASDAQ:NRC) and US Stem Cell (OTCMKTS:USRM) are both small-cap business services companies, but which is the better investment? We will compare the two businesses based on the strength of their earnings, dividends, valuation, profitability, institutional ownership, risk and analyst recommendations.

Analyst Recommendations

This is a breakdown of current ratings and target prices for National Research and US Stem Cell, as reported by MarketBeat.

Valuation & Earnings

This table compares National Research and US Stem Cells revenue, earnings per share (EPS) and valuation.

National Research has higher revenue and earnings than US Stem Cell.

Institutional & Insider Ownership

39.6% of National Research shares are owned by institutional investors. 4.5% of National Research shares are owned by company insiders. Comparatively, 16.7% of US Stem Cell shares are owned by company insiders. Strong institutional ownership is an indication that large money managers, hedge funds and endowments believe a company is poised for long-term growth.

Profitability

This table compares National Research and US Stem Cells net margins, return on equity and return on assets.

Risk & Volatility

National Research has a beta of 0.77, indicating that its stock price is 23% less volatile than the S&P 500. Comparatively, US Stem Cell has a beta of 5.08, indicating that its stock price is 408% more volatile than the S&P 500.

Summary

National Research beats US Stem Cell on 7 of the 9 factors compared between the two stocks.

National Research Company Profile

National Research Corporation (NRC) is a provider of analytics and insights that facilitate revenue growth, patient, employee and customer retention and patient engagement for healthcare providers, payers and other healthcare organizations. The Companys portfolio of subscription-based solutions provides information and analysis to healthcare organizations and payers across a range of mission-critical, constituent-related elements, including patient experience and satisfaction, community population health risks, workforce engagement, community perceptions, and physician engagement. The Companys clients range from acute care hospitals and post-acute providers, such as home health, long term care and hospice, to numerous payer organizations. The Company derives its revenue from its annually renewable services, which include performance measurement and improvement services, healthcare analytics and governance education services.

US Stem Cell Company Profile

U.S. Stem Cell, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of autologous cellular therapies for the treatment of chronic and acute heart damage, and vascular and autoimmune diseases in the United States and internationally. Its lead product candidates include MyoCell, a clinical therapy designed to populate regions of scar tissue within a patient's heart with autologous muscle cells or cells from a patient's body for enhancing cardiac function in chronic heart failure patients; and AdipoCell, a patient-derived cell therapy for the treatment of acute myocardial infarction, chronic heart ischemia, and lower limb ischemia. The company's product development pipeline includes MyoCell SDF-1, an autologous muscle-derived cellular therapy for improving cardiac function in chronic heart failure patients. It is also developing MyoCath, a deflecting tip needle injection catheter that is used to inject cells into cardiac tissue in therapeutic procedures to treat chronic heart ischemia and congestive heart failure. In addition, the company provides physician and patient based regenerative medicine/cell therapy training, cell collection, and cell storage services; and cell collection and treatment kits for humans and animals, as well operates a cell therapy clinic. The company was formerly known as Bioheart, Inc. and changed its name to U.S. Stem Cell, Inc. in October 2015. U.S. Stem Cell, Inc. was founded in 1999 and is headquartered in Sunrise, Florida.

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Reviewing National Research (NASDAQ:NRC) and US Stem Cell (NASDAQ:USRM) - Slater Sentinel

Cardiac Stem Cells Comments Off on Reviewing National Research (NASDAQ:NRC) and US Stem Cell (NASDAQ:USRM) – Slater Sentinel | February 9th, 2020