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Sodium Selenite Improves The Therapeutic Effect Of BMSCs Via Promoting | OTT – Dove Medical Press

By daniellenierenberg

Dongmei Yan,1,* Botao Tang,2,* Lixin Yan,3 Lei Zhang,1 Meijuan Miao,1 Xi Chen,4 Guangyi Sui,5 Qi Zhang,1 Daoyuan Liu,1 Hui Wang1

1Department of Blood Transfusion, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 2Department of Cardiology, Heilongjiang Red Cross Hospital, Harbin, Peoples Republic of China; 3Department of Laboratory Medicine, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 4Department of Hematology, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 5Ethics Committee, The Tumor Hospital Affiliated to Harbin Medical University, Harbin, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Hui WangDepartment of Blood Transfusion, The Second Affiliated Hospital, Harbin Medical University, Xuefu Road No. 246, Nangang District, Harbin, Heilongjiang Province, Peoples Republic of ChinaTel +86-451-86605134Email wanghui@hrbmu.edu.cn

Purpose: Sodium selenite (Na2SeO3) has been known to restore the antioxidant capacity of bone marrow mesenchymal stem cells (BMSCs), reduce the production of reactive oxygen species (ROS) in the cells, and promote cell proliferation and inhibit cell apoptosis. However, it is still not clear whether selenium can mediate the differentiation and inhibit the induced hemagglutination of BMSCs. In this study, we attempted to explore the effect of Na2SeO3 on these aspects of BMSCs.Methods: We evaluated the fate of the MSCs isolated from the bone marrow of mice by studying their differentiation and proliferation after treatment with Na2SeO3. We also simultaneously evaluated the coagulation reaction induced by Na2SeO3-treated BMSCs in vitro.Results: While the mice-derived BMSCs expressed CD44, CD73, CD90, and CD105, they did not express CD45. The morphology of the derived cells was homogeneously elongated. These results showed that the isolated cells are indeed BMSCs. We found that 0.1 M and 1 M of Na2SeO3 promoted the proliferation and apoptosis of BMSCs, respectively. This showed that Na2SeO3 can be toxic and exert certain side effects on the BMSCs. The results of the osteogenic and adipogenic assay showed that 0.1 M Na2SeO3 could significantly promote the osteogenic and adipogenic differentiation of BMSCs by upregulating the lipid factors (LPL and PPRAG) and osteogenic factors, RUNX2, COL1, and BGP, in a concentration-dependent manner. Coagulation experiments in animals (mice and rats) revealed that Na2SeO3 can reduce the coagulation time of BMSCs in a concentration-dependent manner, which is related to the high expression of hematopoietic factors (SDF-1, GM-CSF, IL-7, IL-8, IL-11, and SCF).Conclusion: Na2SeO3 promotes the proliferation and differentiation as well as reduces the coagulation time of BMSCs, and this effect might enhance the therapeutic effect of BMSCs.

Keywords: sodium selenite, BMSCs, proliferation, differentiation, coagulation factors, clotting time

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Be the Match and Make a Difference – Fairfield Mirror

By daniellenierenberg

On Wednesday, Nov. 6, the Be the Match Club at Fairfield University hosted a donor registration drive to encourage students to sign up to be a bone marrow donor.

Be the Match is an organization that seeks to help people who are suffering from blood cancer or blood diseases and are in need of a transplant. Be the Match, operated by the National Marrow Donor Program, provides patients with a way to find a transplant match, which could be their last chance for a cure. There is a large number of diseases that could be treated with a transplant, including Hodgkin and non-Hodgkin lymphoma, different types of leukemia and severe aplastic anemia.

The Be the Match Club at Fairfield University started in the fall of 2018 when a group of studentswas inspired by a fellow Stag who had to withdraw from school when he was re-diagnosed with cancer. The clubs goal is to spread awareness about the organization and increase the database of donors, so there is a greater chance for a patient to find their match.

Were trying to get as many people as possible to sign up, Brian Gozzo 20, Vice President of the Be the Match Club, says. Currently were at 20 million. We hope to one day have basically the entire planet, ideally, on it.

A donor-patient match is found by having the donor swab the inside of their cheek to gather DNA that is tested against the patient. If the two have a similar human leukocyte antigen, they are a close match for a transplant. After signing up for the registry at Fairfield University, a cheek-swabbing kit will be sent to your house, and then will be sent back for the stem cells to be tested.

Its super simple, Gozzo says. All it takes is a cheek swab, and, like, five minutes of your time, and youre put on [the registry] till youre 61. If youre matched with someone right then, youll probably receive a phone call, and then that will take another couple weeks until you actually have to donate.

If a match is found, the actual donation process could go one of two ways. One way to donate is to give peripheral blood stem cells, which is a non-surgical procedure that extracts blood through a needle, puts it through a machine that separates the blood cells and then returns the remaining blood into the body. The other option for donation is to give bone marrow through a surgical procedure that removes liquid marrow from the pelvic bone.

Gozzo understands that this can sound scary, but the chances of getting a phone call is pretty slim. He says that only one in about four hundred people on the registry will ever have to donate.

One thing we want people to know is dont be scared that were gonna call you and say the next day you have to be here, across the country and donate, Gozzo said. Its a very lengthy process, theres a lot of people involved and its very safe.

For Gozzo, the most important thing is to spread awareness and increase the number of people on the registry and the chances of a life-saving donation. He says, You could just sit on the registry until you turn 61 and never once receive a phone call, but just know that, like, you still were there and youve still done your part.

Gozzo, a resident assistant, was motivated to form the Be the Match Club at Fairfield University with a few other RAs last year when the student had to withdraw. They reached out to Be the Match for help.

When the student had to withdraw, a couple of the RAs wanted to know what we could do to help. Could we find a match for this kid? Gozzo said. Course, thats very, very difficult.

However, last years drive was not the first appearance Be the Match has made at Fairfield University. Senior Julia Giampietro and her roommate brought a Be the Match drive themselves to Fairfield in their sophomore year. She reached out to the Connecticut Be the Match region leader, who helped them set up a drive that brought over 60 students to join the national bone marrow donor registry. Giampietro was influenced to raise awareness for this organization for a personal reason.

I wanted to bring [Be the Match] to Fairfield in honor of my cousin Christopher who passed away from AML Leukemia in October of my freshman year at Fairfield, Giampietro said via email. He went through a bone marrow transplant, was in remission and relapsed a year later. He received a second bone marrow transplant but the cancer took over his body He was and still is the biggest inspiration in my life and no matter what would always say its all good which is the motto my family and I live by now.

Giampietro continued work with Be the Match throughout her junior and senior years. She was also inspired by the Fairfield student who had to withdraw last year, so she worked with students in younger grades to put on another drive in the fall of 2018, which is when the Be the Match Club was born. Giampietro and her roommate decided to put together an event for the student at the Seagrape Cafe, where they raised almost $2,000 for his family from donations at the door and from other Fairfield Students.

That was probably the biggest accomplishment of our 3 years involved with Be the Match and was a great way to close the year and our time in the club, Giampietro said. It was also amazing to see how much support we got and the feeling of being able to make a small difference for a local peer and family.

Although her time with Be the Match at Fairfield University is over, Giampietro has high hopes for the club and the organization in the years to come.

My biggest hope really is to have students become more aware and educated about [Be the Match], Giampietro said. It is so important for people our age to be educated on this amazing cause because we are the ones who can save peoples lives.

Be the Match will hold their next donor registration drive in the spring of 2020. To learn more about the organization or become a donor, visit https://bethematch.org/.

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BrainStorm Cell Therapeutics Announces Ralph Kern MD MHSc to Present at the 7th International Stem Cell Meeting – Yahoo Finance

By daniellenierenberg

NEW YORK, Nov. 12, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Companys Chief Operating and Chief Medical Officer Ralph Kern MD MHSc will present at the 7th International Stem Cell Meeting, which is hosted by the Israel Stem Cell Society. The Conference will be held November 12-13, in Tel Aviv, Israel.

Ralph Kern, MD, MHSc, said: I welcome the opportunity to participate in the 7th International Stem Cell Meeting where I will share the advances BrainStorm has made with NurOwn. It is a privilege to participate and to exchange ideas with many of the international scientific leaders in stem cell research.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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Heartbreaking moment dad meets the woman who saved his life – he feared his son would grow up without a father – Manchester Evening News

By daniellenierenberg

This is the tear-jerking moment a dad shares a hug with the woman who saved his life.

James O'Donnell, from Burnage, feared the worst after being diagnosed with a blood disorder similar to leukaemia in 2016.

Usual treatments were failing and James was undergoing a blood transfusion every week while battling constant infections, the Liverpool Echo reports.

James was running out of options and despaired at the pain his death could cause his eight-year-old son, Harrison.

But in a stunning stroke of fortune, his saviour was only the other side of the M62 - LiverpoolCouncil admin worker Leah McDougall.

The 29-year-old mum, from Bootle, had taken the time to sign up to the register of potential stem cell donors on her lunch break at a pop-up stall, organised by blood cancer charity DKSM, the previous year.

James, who despite his Manc heritage is an avid Liverpool FC fan, told staff at the charity that he would be up for meeting his donor, who could have been anyone from a number of European countries using the register.

James, along with his wife Andrea and young Harrison, got the chance to meet Leah for the first time at a DKSM charity gala in London on Wednesday last week (November 6).

James, who says he finally feels like himself after a long period of illness, told the ECHO: "I was just getting chest infections and water infections all the time.

"I am quite a healthy person, and I was in good shape and I knew I should not be getting ill all the time."

He said after a few weeks of tests his was invited to take a bone marrow biopsy and was told the devastating news on his 40th birthday.

The disease meant James' bone marrow was not producing enough white blood cells, but doctors told him a treatment called anti-thymocite globulin (ATG) had a "75% chance" of success.

However, when that failed, fear and doubt began to creep in.

He said: "We are always saying I would get through this, we were thinking I would get better. But I started to think it's not happening, it's not going to be for me, this.

"I thought, I have been good in life, I need some luck. We were having a really hard time.

"My son was four or five then, and it was hard for him having a dad going from playing football with him to being in hospital."

Eventually doctors revealed the only option was for James to have a bone marrow transplant.

The O'Donnell's went through further disappointment when tests on his three siblings revealed none were a match, so the waiting game to find a suitable donor began.

But on a March day in 2017, he got a call to say: "We have got a perfect match, a 10 out of 10."

The operation was a success and after four weeks doctors told James the new bone marrow cells were taking effect.

He said: "We were so lucky to find a donor only about 25 miles away. Some people never find one and we had one on our doorstep."

The powerful emotion of meeting Leah last week is summed up by James: "It was the second best moment of my life after my son being born.

"What she has done means that I can see my son growing up and that he has a father."

Leah did not hesitate to agree to help a total stranger when she was asked by DKSM.

Describing the moment she met James and his family, she told the ECHO: "We were both speechless. When I walked on stage we were just hugging each other for ages.

"It is weird, we felt like we had known each other for years, I felt like I had known him my whole life.

"It just takes five minutes out of your time to sign up to the register; that's like going to the kitchen to make a drink.

"You just think about the impact it is going to have on someone, it is saving someone's life. I feel lucky to have been able to give something back."

James says his family and Leah are planning to meet up again, possibly at a Liverpool FC game.

He said: "Without her, I wouldn't have a future."

DKSM has urged anyone aged 17-55, and in general good health, to sign up to the register here.

Dr Manos Niklolousis, Haematologist at University Hospital Birmingham NHS Foundation Trust, said:"Blood stem cells can be used to treat a wide range of blood cancers and blood disorders and we urgently need more people to come forward as donors.

"Currently, only 2% of the UK population are registered so matching donors with patients isnt easy within a growing multicultural population.

"Many of those in need are unable to find a sibling match and so rely on the generosity of strangers, and a blood stem cell transplant can be some patients only hope of survival.

"As a doctor who treats people with blood cancer or disorders, it is upsetting to know that some patients could have been saved if only more potential donors were registered and available to donate.

"I look forward to the day when there will be a donor for every patient in need."

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Tearful dad meets woman who saved his life and gave him future with his son – Liverpool Echo

By daniellenierenberg

This is the poignant moment a dad and his young family broke down as they met the woman who saved his life.

James O'Donnell, 43, was running out of options after being diagnosed with aplastic anemia, a blood disorder similar to leukaemia, in 2016.

Usual treatments were failing and James was undergoing a blood transfusion every week while battling constant infections.

James, from Burnage in Manchester, feared his luck was out and despaired at the pain his death could cause his eight-year-old son Harrison.

But in a stunning stroke of fortune, his saviour was only the other side of the M62; Liverpool Council admin worker Leah McDougall.

The 29-year-old mum, from Bootle , had taken the time to sign up to the register of potential stem cell donors on her lunch break at a pop-up stall, organised by blood cancer charity DKMS, the previous year.

James, who despite his Manc heritage is an avid Liverpool FC fan, told staff at he charity that he would be up for meeting his donor, who could have been anyone from a number of European countries using the register.

James, along with his wife Andrea and young Harrison, got the chance to meet Leah for the first time at a DKMS charity gala in London on Wednesday last week (November 6).

James, who says he finally feels like himself after a long period of illness, told the ECHO: "I was just getting chest infections and water infections all the time.

"I am quite a healthy person, and I was in good shape and I knew I should not be getting ill all the time."

He said after a few weeks of tests his was invited to take a bone marrow biopsy and was told the devastating news on his 40th birthday.

The disease meant James's bone marrow was not producing enough white blood cells, but doctors told him a treatment called anti-thymocite globulin (ATG) had "75% chance" of success.

However when that failed, fear and doubt began to creep in for James.

He said: "We are always saying I would get through this, we were thinking I would get better. But I started to think it's not happening, it's not going to be for me, this.

"I thought, I have been good in life, I need some luck. We were having a really hard time. My son was four or five then, and it was hard for him having a dad going from playing football with him to being in hospital."

Eventually doctors revealed the only option was for James to have a bone marrow transplant.

The O'Donnells went through further disappointment when tests on his three siblings revealed none were a match, so the waiting game to find a suitable donor began.

But on a March day in 2017, he got a call to say: "We have got a perfect match, a 10 out of 10."

The operation was a success and after four weeks doctors told James the new bone marrow cells were taking effect.

He said: "We were so lucky to find a donor only about 25 miles away. Some people never find one and we had one on our doorstep."

The powerful emotion of meeting Leah last week is summed up by James: "It was the second best moment of my life after my son being born.

"What she has done means that I can see my son growing up and that he has a father."

Leah did not hesitate to agree to help a total stranger when she was asked by DKMS.

Describing the moment she met James and his family, she told the ECHO: "We were both speechless. When I walked on stage we were just hugging each other for ages.

"It is weird, we felt like we had known each other for years, I felt like I had known him my whole life.

"It just takes five minutes out of your time to sign up to the register; that's like going to the kitchen to make a drink.

"You just think about the impact it is going to have on someone, it is saving someone's life. I feel lucky to have been able to give something back."

James says his family and Leah are planning to meet up again, possibly at a Liverpool FC game.

He said: "Without her, I wouldn't have a future."

DKMS has urged anyone aged 17-55, and in general good health, to sign up to the register here .

Dr Manos Niklolousis, Haematologist at University Hospital Birmingham NHS Foundation Trust, said:"Blood stem cells can be used to treat a wide range of blood cancers and blood disorders and we urgently need more people to come forward as donors.

"Currently, only 2% of the UK population are registered so matching donors with patients isnt easy within a growing multicultural population.

"Many of those in need are unable to find a sibling match and so rely on the generosity of strangers, and a blood stem cell transplant can be some patients only hope of survival.

"As a doctor who treats people with blood cancer or disorders, it is upsetting to know that some patients could have been saved if only more potential donors were registered and available to donate. I look forward to the day when there will be a donor for every patient in need."

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bluebird bio and Forty Seven Announce a Research Collaboration to Study an All Antibody Conditioning Regimen for Use in Combination with Autologous…

By daniellenierenberg

CAMBRIDGE, Mass. & MENLO PARK, Calif.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) and Forty Seven, Inc. (Nasdaq:FTSV) announced today 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. This collaboration will focus on a conditioning approach aimed to deliver reduced toxicity and will initially target diseases that have the potential to be corrected with transplantation of autologous gene-modified blood-forming stem cells. If successful, the new conditioning regimen could allow for more patients to undergo gene therapy.

Autologous hematopoietic stem cell transplantation (HSCT) and most ex vivo LVV HSC gene therapies require that a patients own stem cells first be depleted from the bone marrow to facilitate the engraftment of the new (or gene-modified) HSCs through a process called conditioning. Conditioning is performed using chemotherapy or radiation, which can place patients at risk for infection and require hospitalization until bone marrow cells have recovered. In addition, conventional conditioning can place patients at risk for secondary malignancy and infertility. As a result, the overall toxicity profile of current conditioning regimens limits the types of patients who are eligible for gene therapy. It is hoped that novel antibody based conditioning regimens could avoid these toxicities.

We are excited about this collaboration, combining our industry-leading LVV HSC gene therapy platform with Forty Sevens novel antibody-based conditioning regimen, said Philip Gregory, chief scientific officer, bluebird bio. We believe that, if successful, this novel conditioning modality could not only increase the number of patients and physicians who may consider gene therapy but also improve the overall risk benefit profile for stem cell-based gene therapy, as well as potentially reduce time and costs associated with hospital visits.

Forty Seven is advancing the pioneering work on CD47 and cKIT from our scientific founder, Irv Weissmans lab. We have shown that antibody blockade of CD47 can synergize with other antibodies targeting cancer to promote tumor engulfment. Based on this experience, coupled with the results of preclinical studies, we are eager to explore this dual-antibody approach for the potential treatment of non-malignant diseases, says Jens Peter Volkmer, M.D., Founder and Vice President of Research and Development at Forty Seven.

Forty Sevens President and Chief Executive Officer, Mark McCamish, M.D., Ph.D., commented, bluebird is a leading gene therapy company and we are excited to collaborate with them. Stem cell transplantation is potentially curative for a variety of blood diseases, including genetic blood disorders like sickle cell disease and beta-thalassemia. If successful, we believe our chemo- and radiation-free, all-antibody approach could expand transplantation beyond genetic blood disorders to a range of indications for which current transplantation approaches are suboptimal. In 2020, we plan to evaluate FSI-174 in healthy volunteers, before initiating a combination study of Forty Sevens novel all-antibody conditioning regimen and bluebirds gene therapy product.

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.

About FSI-174 and MagrolimabFSI-174 is a humanized monoclonal antibody targeting cKIT, which is a receptor that is highly expressed on hematopoietic stem cells. Magrolimab is a humanized monoclonal antibody targeting CD47, which is a dont eat me signal to macrophages and is expressed on all cells. Magrolimab is currently being investigated in Phase 2 clinical trials to treat cancer and has established clinical efficacy in four indications, including myelodysplastic syndrome, acute myeloid leukemia, diffuse large B cell lymphoma and follicular lymphoma, with a favorable safety profile in over 350 patients treated, including some patients treated continuously for over two years. When combined, FSI-174 sends a positive signal to macrophages to target blood forming stem cells for removal and magrolimab disengages inhibitory signals that block phagocytosis. Combination of these antibodies has shown efficient removal of blood forming stem cells, allowing for transplantation in pre-clinical models.

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 by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent -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.

bluebird bio is a trademark of bluebird bio, Inc.

About Forty Seven Inc.Forty Seven, Inc. is a clinical-stage immuno-oncology company that is developing therapies targeting cancer immune evasion pathways based on technology licensed from Stanford University. Forty Sevens lead program, magrolimab, is a monoclonal antibody against the CD47 receptor, a dont eat me signal that cancer cells commandeer to avoid being ingested by macrophages. This antibody is currently being evaluated in multiple clinical studies in patients with myelodysplastic syndrome, acute myeloid leukemia, non-Hodgkins lymphoma, ovarian cancer and colorectal carcinoma.

For more information, please visit http://www.fortyseveninc.com or contact info@fortyseveninc.com.

Follow Forty Seven on social media: @FortySevenInc, LinkedIn

Forward-Looking StatementsThis release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," potentially, and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the research and development plans for bluebird bios and Forty Sevens respective platforms and product candidates, the timing and success of Forty Sevens collaboration with bluebird bio, Forty Sevens plans to pursue clinical proof-of-concept for FSI-174 plus magrolimab with the LVV HSC gene therapy platform, the focus on diseases that have the potential to be corrected with transplantation of autologous gene-modified blood-forming stem cells, the tolerability and efficacy of FSI-174 and magrolimab, Forty Sevens plans to continue development of FSI-174 plus magrolimab, as well as related timing for clinical trials of the same.

Any forward-looking statements are based on the companies 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 exploratory antibody-based conditioning platform will not be successful or will not be safe or effective in clinical trials, the risks that the collaboration between bluebird bio and Forty Seven will not continue or be successful, and the risk that the parties will not be successful in advancing the collaboration in development, the risk that potential product candidates that bluebird bio and Forty Seven develop may not progress through clinical development or receive required regulatory approvals within expected timelines or at all, the risk that clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release and the risk that such product candidates may not be beneficial to patients or successfully commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the companies actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in each companys most recent Form 10-K as well as discussions of potential risks, uncertainties and other important factors in subsequent filings with the Securities and Exchange Commission at http://www.sec.gov. All information contained in this press release are not guarantees of future performance and speak only as of the date hereof, and each of bluebird bio and Forty Seven disclaims any obligation to update this information to reflect future events or circumstances unless required by law.

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BrainStorm Cell Therapeutics Announces Ralph Kern MD MHSc to Present at the 7th International Stem Cell Meeting – GlobeNewswire

By daniellenierenberg

NEW YORK, Nov. 12, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the Companys Chief Operating and Chief Medical Officer Ralph Kern MD MHSc will present at the 7th International Stem Cell Meeting, which is hosted by the Israel Stem Cell Society. The Conference will be held November 12-13, in Tel Aviv, Israel.

Ralph Kern, MD, MHSc, said: I welcome the opportunity to participate in the 7th International Stem Cell Meeting where I will share the advances BrainStorm has made with NurOwn. It is a privilege to participate and to exchange ideas with many of the international scientific leaders in stem cell research.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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From inspiration to innovation | UDaily – UDaily

By daniellenierenberg

Article by Karen B. Roberts Photos by Evan Krape November 11, 2019

The Merriam-Webster Dictionary defines an inventor as one who creates or introduces something new.

Thomas Edison is one. So is Emily Day.

Edison created the incandescent light bulb and the typewriter, among dozens of other things.

Day, an assistant professor in biomedical engineering at the University of Delaware, is working on technology that may one day replace bone marrow transplants by enabling nanoparticle carrier systems to deliver medication and cargo directly to stem cells without the need to remove them from the body.

The University recognized more than 225 inventors, including Day, on Tuesday, Oct. 29, for their remarkable contributions to UD and to society at large.

The event, held at the Roselle Center for the Arts and coordinated by the UDResearch Office, celebrated researchers with discoveries in engineering, health care, energy, agriculture and many other fields.

You, our inventors, have taken nuggets of ideas, of discoveries that youve made and developed them through hard work, trial and error, failure and success. Youve shown tenacity and drive, patience and persistence, and the results are what were celebrating today, said UD Provost Robin Morgan.

Enriching the environment for entrepreneurship

Since 2008, UD researchers have generated more than 500 inventions.

Working in collaboration with its partners, the University has made a concerted effort to enrich the environment for these types of efforts in Delaware, contributing to the states economic prosperity and positively impacting the greater good.

UD research expenditures for fiscal year 2019 totaled $161 million, a record-setting 10% increase over 2018, to explore pressing topics across the sciences, engineering, humanities and social sciences.

During this same time frame, UD researchers generated 33 patent applications and secured 11 patents, with the support of the Universitys Office of Economic Innovation and Partnerships (OEIP). OEIP has licensed six UD-developed technologies to outside companies and evaluated numerous other potential inventions currently under development.

Several UD-developed technologies are now featured in the Association of University Technology Managers Better World Project, which highlights successful examples where academic research and technology transfer combine to benefit the broader world. One of these is the UD-patented microbe UD10-22, a unique strain of Bacillus subtilis that helps plants grow stronger, developed by Harsh Bais, associate professor of plant and soil sciences, and Janine Sherrier, a former UD faculty member. UD licensed the technology to BASF, a global chemical company, in 2013. After completing successful trials and regulatory clearances, the technology is now available in the market as a key component of BASFs Velondis and Nodulator Duo product lines in Canada and the United States. Trials are ongoing for the product to be available in four additional product lines and for a range of crops to be sold in several countries in South America, Europe and Asia.

We are building a dynamic and rich ecosystem to support this type of activity, now and in the future, said Charles G. Riordan, UD vice president for research, scholarship and innovation.

Continued growth of UDs Science, Technology and Advanced Research (STAR) Campus through strategic partnerships and infrastructure development is one example that firmly positions the University as an innovation powerhouse for the community, state and region. The Delaware Innovation Space, the business incubator that is a public-private partnership between the state of Delaware, DuPont and UD, is another.

Riordan reported that Delaware Innovation Space, with its 130,000 square feet of lab-based tech space for startups, already is 90% occupied, hosting 13 companies including UD startups W7energy and MCET along with serving an additional dozen companies through its virtual program. The result more than 240 jobs created or retained.

Other resources available on campus to support innovators and entrepreneurs include, but are not limited to, OEIP, competitive funding opportunities, seed funding and training programs at UDs Horn Entrepreneurship,and new and existing core research facilities.

Other UD technologies that have had success in the marketplace during the past year include Avkin, a leading manufacturer of sensor-enabled, high-fidelity, wearable technology for health care simulation education founded byAmy Cowperthwait, director of Healthcare Theatre for the College of Health Sciences.The patented devices are used for training health care workers and caregivers to perform clinical procedures, such asdrawing blood, tracheostomy care or catheter insertion. Designed to be worn by a live actor, Avkin products provide a realistic, patient-centered simulation.

Today, the UD-developed products can be found in select medical and nursing schools and health systems.The company now has five products in the market, and recently launched a new package aimed at equipping todays practitioners with the knowledge and skills necessary to prevent hospital acquired infections and to improve patient outcomes.

Isao Noda, UD affiliated professor in materials science and engineering, said it is particularly important to foster innovation and invention among students. An inventor himself, Noda is named on more than 60grantedU.S. patents.

One of Nodas inventions is abio-basedplasticmade from vegetable oilsknown asNodax, which can be used to make eco-friendlyproductsranging from biodegradable plastic straws topiezoelectricnanofibers forsensors and other electronics. Nodainventedthe material while a research fellow atProcterand Gamble. Today, UDscientistsare part of theexploratorywork onNodax, collaboratingon fundamental research to see just what elsethisnovelmaterialcan do.

In industry, invention is required. If you dont invent, you will be fired. But many graduates get jobs in industry without any of the training on how to invent, so this is amazingly important, said Noda.

Day agreed and said her approach to innovation shifted in recent years, particularly when speaking with students.

In the beginning of my academic career I was more focused on publishing papers, Day said. As my group has become more established, I now tell my students, Hey, before you go present this or publish, its important for you to submit your invention disclosure to protect your ideas.

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Kiadis Pharma changes strategy to focus solely on development of Natural Killer (NK) Cell therapeutics and terminates development of ATIR101 – Yahoo…

By daniellenierenberg

Amsterdam, The Netherlands, 12 November 2019 Kiadis Pharma N.V. (Kiadis Pharma or the Company) (Euronext Amsterdam and Brussels: KDS), a clinical-stage biopharmaceutical company, today announced that it has completed a strategic portfolio review and has decided to change its strategy and focus all resources and investments on the companys NK-cell therapy platform and product candidates. The company will discontinue development of ATIR101 and stop its ongoing phase 3 trial.

Kiadis NK-cell program consists of off-the-shelf and haplo donor cell therapy products for the treatment of liquid and solid tumors. Kiadis proprietary off-the-shelf NK-cell platform is based on NK-cells from unique universal donors, expanded and activated ex vivo using our PM21 particle technology. The Kiadis off-the-shelf platform has the potential to make NK-cell therapy products rapidly and economically available for a broad patient population across a potentially wide range of indications.

The companys pipeline includes:

Arthur Lahr, CEO of Kiadis Pharma commented, We believe that our proprietary NK-cell therapy platform has broad potential as stand-alone or adjunctive treatments for patients with both liquid and solid tumors. Our off-the-shelf NK-cell platform is based on NK-cells from unique universal donors, expanded and activated with our PM21 particle technology, to make our NK-cell therapy products rapidly and economically available for patients across a potentially broad range of indications. The proof-of-concept trials for our NK pipeline programs, in which 38 patients have been treated, is very promising and was the basis for our acquisition of Cytosen Therapeutics, Inc. earlier this year. To confirm findings from these trials, we will start two Phase 1/2 clinical trials in 2020. We believe that investing in our NK platform and rapidly advancing development of our off-the-shelf and haplo donor derived NK-cell therapies in solid and liquid tumors will bring value to patients and our investors.

Lahr continued, As part of our strategic portfolio review, we reviewed progress of our phase 3 study, which was designed to show superiority of ATIR101 over the PTCy protocol. We identified that in the phase 3 a higher percentage of patients than expected dropped out of the study before receiving ATIR101. We subsequently collected additional recent external data, which show that outcomes with PTCy have better survival and lower severe GVHD than literature showed when we designed and started the phase 3 study. Based on these data, we no longer believe that the phase 3 ATIR study as currently designed with 250 patients can demonstrate superiority over PTCy and at a minimum would require a much larger trial. In the best interest of patients, we have therefore taken the decision to discontinue the ATIR101 study with immediate effect and are proceeding with close down activities.

RestructuringKiadis is implementing a restructuring program to refocus the organization on its NK-cell therapy platform, which will result in a reduction of approximately half of its workforce, a reduction in external clinical trial costs associated with the phase 3 study, and a reduced company cash burn. The company ended the third quarter of 2019 with approximately 47 million of cash.

About Kiadis K-NK-Cell Therapies Kiadis NK-cell programs consist of off-the-shelf and haplo donor cell therapy products for the treatment of liquid and solid tumors as adjunctive and stand-alone therapies.

Our NK-cell PM21 particle technology enables improved ex vivo expansion and activation of anti-cancer cytotoxic NK-cells supporting multiple high-dose infusions. Kiadis proprietary off-the-shelf NK-cell platform is based on NK-cells from unique universal donors. The Kiadis off-the-shelf K-NK platform can make NK-cell therapy product rapidly and economically available for a broad patient population across a potentially wide range of indications.

Administered as an adjunctive immunotherapeutic on top of HSCT, K-NK002 provides functional, mature and potent NK-cells from a haploidentical family member. In addition, Kiadis is developing K-NK003 for the treatment of relapse/refractory acute myeloid leukemia and has pre-clinical programs evaluating NK-cell therapy for the treatment of solid tumors.

Story continues

Kiadis Contacts:

About KiadisFounded in 1997, Kiadis Pharma, is a fully integrated biopharmaceutical company committed to developing innovative cell-based therapies for patients with life-threatening diseases. With headquarters in Amsterdam, the Netherlands, and offices and activities in the US and across Europe, Kiadis Pharma is leveraging the natural strengths of humanity and our collective immune system to source the best cells for life.

Kiadis Pharma is listed on the regulated market of Euronext Amsterdam and Euronext Brussels since July 2, 2015, under the symbol KDS. Learn more at http://www.kiadis.com.

Forward Looking Statements Certain statements, beliefs and opinions in this press release are forward-looking, which reflect Kiadis Pharmas or, as appropriate, Kiadis Pharmas directors current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial impact of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, regulation, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward-looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, Kiadis Pharma expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither Kiadis Pharma nor its advisers or representatives nor any of its subsidiary undertakings or any such persons officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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The Value and Versatility of Clinical Flow Cytometry – Technology Networks

By daniellenierenberg

What is flow cytometry and how does it work?Flow cytometry(FCM) is a scientific technique used to measure the physical and biochemical characteristics of cells.1The sample is injected into the flow cytometer instrument, where it is typically focused to flow one cell at a time past light sources and detectors. Tens of thousands of cells can be examined in seconds to determine their morphology, granularity, scattering and transmission of light, or fluorescence of biomarkers, depending on the variation of FCM used.

The first conventional fluorescence-based flow cytometer was developed and commercialized in the late 60s/early 70s in Germany.2 Over the last five decades, FCM has developed rapidly in terms of the number of its applications and the quantity and dimensionality of the data it generates.1,3 Dr. Minh Doan, formerly of the Imaging Platform of the Broad Institute (USA) and now head of Bioimaging Analytics at GlaxoSmithKline in the USA, states, There have been significant advances in all three Vs of flow cytometry data: velocity (throughput/speed of data acquisition), volume (data content), and variety (sample types and signal acquisition technology).

Michael Parsons, manager of the Flow Cytometry Core of the Lunenfeld-Tanenbaum Research Institute in Toronto, Canada, agrees. The two biggest trends in flow cytometry are high content data and the merging of technologies from separate disciplines. For example, the last five years or so have seen the emergence of mass cytometry, which merges the disciplines of flow cytometry and mass spectrometry. In its latest iteration, an image cytometry module has been incorporated to generate unprecedented amounts of content (number of measured parameters) from relatively small amounts of patient tissue. Spectral flow cytometry has also established itself as an important emerging technology. Indeed, mass cytometry can now measure up to 50 features on a single cell simultaneously using antibodies tagged with rare earth metals,4 and imaging flow cytometry allows for 1000s of morphological features and multiple fluorescence markers to be analyzed per cell.3Flow cytometry, therefore, has inarguable potential as a clinical tool for disease diagnosis, prognosis, and therapeutic monitoring. However, some challenges remain in translating the full promise of FCM into clinical practice. Here, some of the current clinical applications of FCM will be discussed, as well as some of the compelling new applications being researched.

Similarly, FCM of liquid biopsies could be used to detect circulating tumor cells in the bloodstream.3 These cells are extremely rare, and with its high sensitivity, FCM is perfectly poised to make a significant impact in this area. This approach has potential for the clinical detection of early-stage cancer as well as the detection of circulating metastatic or drug-resistant cancer cells. For example, a study published earlier this year described label-free liquid biopsy with very high throughput (> 1 million cells/second) for drug-susceptibility testing during leukemia treatment.8

Prior to an organ transplant, FCM can be used to crossmatch the patient's serum with donor lymphocytes to detect antibodies that could result in organ rejection.1 Postoperatively, the analysis of various cell markers on the peripheral blood lymphocytes can indicate early transplant rejection, detect bone marrow toxicity arising from immunosuppressive therapies, and help differentiate infections from organ rejection. For blood transfusions, FCM can be used to detect contamination of blood with residual white blood cells, which can have adverse effects such as pulmonary edema.9Groups such as Dr. Roshini Abrahams at Nationwide Childrens Hospital in Ohio, USA, are using FCM to diagnose primary immunodeficiency disorders with the use of immunophenotyping and functional assays.10 These disorders are caused by genetic mutations that result in defects in the immune system, such as X-linked (Brutons) agammaglobulinemia and X-linked hyper-IgM syndrome. Over 300 of these disorders have been identified thus far, and the causative mutations lower immune defense against the attack of infections.

HIV is, of course, an example of a secondary (acquired) immunodeficiency disorder. FCM analysis of CD4 and other markers on lymphocytes in the peripheral blood is used to monitor the treatment of HIV patients, and a CD4 count <200 cells/mL together with a positive antibody test for HIV is used as a diagnostic for AIDS.1 Secondary immunodeficiencies can also be caused by e.g., substance abuse, malnutrition, other medical conditions, and certain medical treatments. FCM of a panel of markers can be used to confirm suspected cases.1In pregnancy, when a Rhesus blood group D-negative mother carries a D-positive fetus, fetal-maternal bleeding can sensitize the mother to the D-positive blood cells from the fetus and this can be fatal to subsequent D-positive newborns.11 FCM is used to measure the degree of fetal-maternal hemorrhage to determine the correct dose of prophylactics to be administered shortly after delivery.

In addition to oncology and immunology applications, FCM is also used to diagnose a variety of rare hematologic disorders12 as well as autoimmune/autoinflammatory disorders such as spondylarthritis (arthritis of the spine).13 Another area of research that is likely to give rise to increasing clinical applications in the future is that of platelet activity, which is important in many clinical conditions.1,14

Experts suggest that it may be possible to overcome this data analysis hurdle by applying machine learning approaches coupled with further standardization of FCM workflows.3,15 The most exciting applications of high content data revolve around the use of machine learning, in particular, deep learning, to extract relevant meaning from large data sets. Machine learning, coupled with big data, has the potential for driving diagnosis and treatment options tailored to the patients disease in a timely manner, says Dr. Parsons. In addition, Prof. Sadao Ota of RCAST at the University of Tokyo, Japan, points out, We still need to figure out how to design a workflow that convincingly validates diagnostic results, especially if the diagnosis employs the power of machine learning. Such developments are necessary before the rich information content of advanced FCM technology can be fully applied in the clinic.

In terms of other future advances in the field, Prof. Ota specifically makes mention of the potential of cell sorters combined with FCM.16 There are exciting and unique applications of sorters in fields such as cell therapy and regenerative medicine. Also, creating key applications of imaging cell sorters in pharmaceutical fields may accelerate global drug discovery. Dr. Doan concurs, Disease heterogeneity makes it hard to validate findings. Perhaps the use of flow cytometry with sorting capability can help such validation, where events-of-interest collected by flow cytometry can be validated with other downstream assays. Finally, as Dr. Doan notes, With multiple layers of data(types) incorporated altogether, there are now possibilities to do more with less, i.e., label-free sample measurement, which could lead to more direct, faster, and smarter diagnoses. Rare events (e.g., metastatic cancer cells) may soon be detected better than before.References1.Bakke A.C. Clinical Applications of Flow Cytometry. Laboratory Medicine. 2000; 31(2): 97104. doi: 10.1309/FC96-DDY4-2CRA-71FK.2.Herzenberg L.A., Parks D., Sahaf B., Perez O., Roederer M., Herzenberg L.A. The history and future of the fluorescence activated cell sorter and flow cytometry: a view from Stanford. Clinical Chemistry. 2002;48(10):181918273.Doan M., Vorobjev I., Rees P., Filby A., Wolkenhauer O., Goldfeld A.E., Lieberman J., Barteneva N., Carpenter A.E., Hennig H. Diagnostic potential of imaging flow cytometry. Trends in Biotechnology. 2018;36(7):649652. doi: 10.1016/j.tibtech.2017.12.008.4.Olsen L.R, Leipold M.D., Pedersen C.B., Maecker H.T. The anatomy of single cell mass cytometry data. Cytometry Part A. 2019;95(2):156172. doi: 10.1002/cyto.a.23621.5.Laerum O.D., Farsund T. Clinical application of flow cytometry: a review. Cytometry. 1981;2(1):113. doi: 10.1002/cyto.990020102.6.Li J., Wertheim G., Paessler M., Pillai V. Flow cytometry in pediatric hematopoietic malignancies. Clinics in Laboratory Medicine. 2017;37(4):879893. doi: 10.1016/j.cll.2017.07.009.7.Gupta S., Devidas M., Loh M.L., Raetz E.A., Chen S., Wang C., Brown P., Carroll A.J., Heerema N.A., Gastier-Foster J.M., Dunsmore K.P., Larsen E.C., Maloney K.W., Mattano L.A. Jr., Winter S.S., Winick N.J., Carroll W.L., Hunger S.P., Borowitz M.J., Wood B.L. Flow-cytometric vs. -morphologic assessment of remission in childhood acute lymphoblastic leukemia: a report from the Childrens Oncology Group (COG). Leukemia. 2018;32(6):13701379. doi: 10.1038/s41375-018-0039-7.8.Kobayashi H., Lei C., Wu Y., Huang C-J., Yasumoto A., Jona M., Li W., Wu Y., Yalikun Y., Jiang Y., Guo B., Sun C-W., Tanaka Y., Yamada M., Yatomi Y., Goda K. Intelligent whole-blood imaging flow cytometry for simple, rapid, and cost-effective drug-susceptibility testing of leukemia. Lab on a Chip. 2019;19(16):26882698. doi: 10.1039/c8lc01370e.9.Castegnaro S., Dragone P., Chieregato K., Alghisi A., Rodeghiero F., Astori G. Enumeration of residual white blood cells in leukoreduced blood products: Comparing flow cytometry with a portable microscopic cell counter. Transfusion and Apheresis Science. 2016;54(2):266270. doi: 10.1016/j.transci.2015.10.001.10.Abraham R.S., Aubert G. Flow cytometry, a versatile tool for diagnosis and monitoring of primary immunodeficiencies. Clinical and Vaccine Immunology. 2016;23(4):254271. doi: 10.1128/CVI.00001-16.11.Kim Y.A., Makar R.S. Detection of fetomaternal hemorrhage. American Journal of Hematology. 2012;87(4):417423. doi: 10.1002/ajh.22255.12.Bn M.C., Le Bris Y., Robillard N., Wuillme S., Fouassier M., Eveillard M. Flow cytometry in hematological nonmalignant disorders. International Journal of Laboratory Hematology. 2016;38(1):516. doi: 10.1111/ijlh.12438.13.Duan Z., Gui Y., Li C., Lin J., Gober H.J., Qin J., Li D., Wang L. The immune dysfunction in ankylosing spondylitis patients. Bioscience Trends. 2017;11(1):6976. doi: 10.5582/bst.2016.01171.14.Pasalic L. Assessment of platelet function in whole blood by flow cytometry. Methods in Molecular Biology. 2017;1646:349367. doi: 10.1007/978-1-4939-7196-1_27.15.Doan M., Carpenter A.E. Leveraging machine vision in cell-based diagnostics to do more with less. Nature Materials. 2019;18(5):414418. doi: 10.1038/s41563-019-0339-y.16.Ota S., Horisaki R., Kawamura Y., Ugawa M., Sato I., Hashimoto K., Kamesawa R., Setoyama K., Yamaguchi S., Fujiu K., Waki K., Noji H. Ghost cytometry. Science. 2018;360(6394):12461251. doi: 10.1126/science.aan0096.

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An introduction to the use of transplantation for the treatment of AML – AML Global Portal

By daniellenierenberg

Transplant strategies for AML

Today, the only curative approach to treat patients with AML is the administration of high-dose chemotherapy followed by allogeneic HSCT (allo-SCT). Although autologous HSCT (auto-SCT) may still be an option for certain patients with favorable or intermediate risk AML, its use has been debated due to the fact that AML is a blood and bone marrow malignancy, thus transplantation with the patients own cells runs the risk of giving back some of the patients leukemia cells.1 In contrast, during the process of allo-SCT, cells from a donor are infused. This provides an additional benefit, called the graft-versus-leukemia or tumor (GvL or GvT) effect, whereby the donor immune cells have the potential of recognising and eradicating remaining leukemia cells in the host, thus reducing the risk of relapse.2

Various donors can be used for an allo-SCT, while the best stem cell source remains to be from a human leukocyte antigen (HLA)-matched sibling donor (MSD). However, in approximately 70% of the cases such donors are unavailable, leaving the next best option of an HLA-matched unrelated donor (MUD).3 For patients where MSD or MUD are unavailable, a partially HLA-matched related donor can be used. This is referred to as haploidentical transplant (HD) and is usually a 50% HLA-match. In the past, HD has been associated with a slow immune reconstitution and high mortality from infections. Today, the use of post-transplant management treatments, like post-transplant cyclophosphamide (PTCy), reduces mortality and has made HD a viable option for patients with AML. This was further discussed by Arnon Nagler in his interview4 with the AML Global Portal (AGP) during the 2019 European Society for Blood and Marrow Transplantation (EBMT) meeting (video below). Nevertheless, HD still leads to inferior outcomes when compared to MSD in patients with AML (read AGP article here). Other donor types include cells from umbilical cord (read AGP article here) or from HLA-mismatched unrelated donors (MMUD).2 The impact of donor type on the outcomes of allo-SCT has recently been explored in an article by the AGP here. The authors of the study concluded that the traditional hierarchy of donors (MSD, MUD, and then others) remains true in patients with AML and should be used as a treatment algorithm.

VIDEO INTERVIEW: EBMT 2019 | Haploidentical hematopoietic transplantation: current status and future perspectives

How to choose the right patients for transplantation

Choosing the right patient to receive transplantation following chemotherapy is crucial for maximising outcomes and reducing the risk of relapse and toxicity. As mentioned by Uwe Platzbecker in his AGP interview during the 2019 EBMT meeting (video below), there are two main considerations when choosing the right candidate for allo-SCT:

According to the ELN guidelines, patients are classified as favorable-risk, intermediate-risk, or poor-risk depending on the possibility of disease relapse. Patients with favorable-risk are usually not considered for allo-SCT after achieving their first complete remission (CR1), as the risk of toxicity and serious side effects outweighs the potential benefit from allo-SCT. For these patients, auto-SCT instead of chemotherapy after CR1 could be beneficial (read AGP article here). On the contrary, allo-SCT at CR1 is a common strategy for poor-risk patients with AML. In the case of intermediate-risk patients (the majority of patients with AML), the most suitable treatment option is less clear.6 Due to the high relapse rates seen in AML, allo-SCT has also been considered as a potential treatment strategy in second remission (CR2), although outcome is inferior compared to allo-SCT performed in CR1.7 In a recent study, summarized here by the AGP, it seems that myeloablative conditioning (MAC) and reduced intensity conditioning (RIC) lead to similar outcomes after allo-SCT in CR2, however more prospective trials are needed to tailor them for maximum efficacy and minimum toxicity.7 To date, it is evident that clinical decisions to perform transplantation need to be made on an individual basis. Recently, measurable residual disease (MRD) as a marker for disease severity and relapse risk has emerged as an important factor that can guide treatment decisions in the context of HSCT and has been reviewed in depth here by the AGP.

VIDEO INTERVIEW: EBMT 2019 | Considerations for transplantation in AML

Post-transplant issues & how to tackle them

Regardless of the advances in the transplantation field, allo-SCTs are associated with two main post-transplant issues: disease relapse and graft-versus-host disease (GvHD).

There is still a considerable number of patients that relapse after HSCT. At the moment, the best strategies to decrease the risk of post-transplantation relapse include:

Such agents include the use of FMS-like tyrosine kinase-3 (FTL3) inhibitors that are shown to delay disease relapse and to potentiate the GvL effect in patients with FLT3 mutations after allo-SCT.7 Multiple pre-clinical and clinical trials are currently underway to examine the efficacy of other targeted inhibitors, like sorafenib, lestaurtinib, sunitinib, tandutinib, quizartinib, and midostaurin, amongst others.8 Another drug that has been shown to prevent disease relapse and to potentially increase the GvL effect is azacitidine. This is a hypomethylating agent that is currently used as a safe and effective prophylactic therapy in high-risk patients following allo-SCT (read AGP article here).9

Charlie Craddock provided an extensive presentation on the strategies for GvL effect optimization at the 2019 EBMT meeting (see full article on the AGP here):

VIDEO INTERVIEW: American Society of Clinical Oncology (ASCO) 2019 | Who should get azacitidine after transplant?

GvHD remains a major post-transplant challenge, occurring when transplanted donor cells start attacking host cells and tissues.10 There are two main strategies used today to prevent GvHD:

In a clinical trial, PTCy has shown superior outcomes when compared to ATG in patients undergoing HD transplant, leading to improved overall survival, leukemia-free survival, and GvHD-relapse free survival. The results of this study were discussed by Arnon Nagler in his interview with the AGP at European Hematology Association (EHA) 2019. A comprehensive review on available treatments for GvHD prophylaxis and their efficacies has been published here by our GvHD Hub.

VIDEO INTERVIEW: EHA 2019 | Should we use PTCy or ATG as GvHD prophylaxis in haploidentical stem cell transplantation?

In patients who develop severe GvHD, systemic administration of steroids remains the first choice of treatment. Treating GvHD is considered by many as a double-edged sword, since on one side it is necessary, but on the other hand it may hinder the GvL effect, thus contributing to potential disease relapse. Further research is needed to clarify the role of GvHD treatment on the GvL effect and to establish the best agent to treat GvHD without hindering the benefits of graft transplantation to the host. Some patients do not respond to post allo-SCT corticosteroids and are classified as steroid-refractory GvHD patients. These patients have a high mortality rate after allo-SCT with a 1-year survival between 30-35%. Many novel approaches are being tested for these patients with the Janus kinase 1/2 (JAK1/2) inhibitor, ruxolitinib, and the Brutons tyrosine kinase (BTK) inhibitor, ibrutinib, being recently approved by the Food and Drug Administration (FDA) for steroid-refractory GvHD.11,12

VIDEO INTERVIEW: EHA 2019 | What are the current treatment recommendations for acute GvHD and the promotion of the GvL effect?

Can the new treatments for AML reduce the need for transplantation?

With the recent therapeutic advances in the field of AML, one major question arises: Can these advances in diagnostics and new therapies replace allo-SCT? During the 1st National Cancer Research Institute (NCRI) AML academy meeting, AGP was pleased to film the headline debate on recently licensed drugs versus recent advances in transplantation, which can be accessed here. Although an unresolved issue, it is evident that some of the new treatments lack the toxicity associated with allo-SCT and have demonstrated improved survival rates. Moreover, with new diagnostic tools, the identification of the right subgroups of patients who may benefit from a transplant-free and more targeted approach will be feasible. One such novel approach to AML treatment is the use of CAR-T cells. Their use as monotherapy or in combination with allo-SCT for the treatment of relapsed or refractory AML is currently under consideration and of great interest in the field. More details on the potential of CAR-T cell therapy for AML can be found here in a recently published article by the AGP. However, it is too early to say whether these new treatment approaches can replace allo-SCT as a curative approach to treat AML. This topic was discussed by Gert Ossenkoppele in the interview with the AGP shown below.

VIDEO INTERVIEW: EHA 2019 | What is the clinical value of new drugs in AML?

Conclusions

Despite the curative potential conferred by allo-SCT in patients with AML, there is still a high risk of non-relapse mortality (mostly due to severe GvHD) in addition to the risk of relapse associated with transplantation. This warrants the need for the development of either novel management and prophylactic therapies that can improve post-transplantation outcomes or of transplantation-free approaches for the treatment of AML. With numerous clinical trials underway with novel targeted agents as monotherapy or in combination, the future of AML treatment starts to look more promising.

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An introduction to the use of transplantation for the treatment of AML - AML Global Portal

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Stem Cell Therapy Market by Treatment,Application,End Users and Geography Forecast To 2026 – Markets Gazette 24

By daniellenierenberg

Stem Cell Therapy Market is expected to reach 202.77 billion by 2026 from 12.25 billion in 2017 at CAGR of 42.02%.(Detailed analysis of the market CAGR is provided in the report) stands for use of stem cells to treat or prevent disease or condition.

Bone marrow transplant and some therapies derived from umbilical cord blood are mainly used in stem cell therapy. Advancement, in order to establish new sources for stem cells, and to apply stem-cell treatments for neurodegenerative diseases and conditions such as diabetes, heart disease, and other conditions, are increased in recent years. Stem Cell Therapy Market Researchers are making efforts to discover novel methods to create human stem cells. This will increase the demand as well as supply for stem cell production and potential investigation in disease management. Increasing investment & research grants for developing safe and effective stem cell therapy products, the growing patient base for target diseases, concentrated product pipelines, increasing approval of the new clinical trials, rapid technological advancement in genomics, and the rising awareness about the stem cell are expected to drive the growth of the Stem Cell Therapy solutions market during the forecast period.

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However, improper infrastructure, insufficient storage systems, nascent technology in underdeveloped economies, Ethical issues related to an embryonic stem cell, low patient acceptance rate, Difficulty in the preservation of stem cell are expected to restrain the market growth. North America is expected to be the largest growing region by 2026; the reason behind that is extensive funding by Government. However, Emerging countries like India, china, Korea have low growth rate as compared to Developed regions in 2017 but increase in awareness about stem cell therapy will lead the Asia Pacific to generate a significant level of revenue by 2026.

Key Highlights of Stem Cell Therapy Market report

Detailed quantitative analysis of the current and future trends from 2017 to 2026, which helps to identify the prevailing market opportunities.Comprehensive analysis of factors instrumental in changing the market scenario, rising prospective opportunities, market shares, core competencies in terms of market development, growth strategies and identification of key companies that can influence this market on a global and regional scale.Assessment of Market definition along with the identification of key drivers, restraints opportunities and challenges for this market during the forecast period.Complete analysis of micro-markets with respect to individual growth trends, prospects, and contributions to the overall Stem Cell Therapy Solutions market.Stem Cell Therapy market analysis and comprehensive segmentation with respect to the Application, End users, Treatment, and geography to assist in strategic business planning.Stem Cell Therapy market analysis and forecast for five major geographies-North America, Europe, Asia Pacific, Middle East & Africa, Latin America, and their key regions.For company profiles, 2017 has been considered as the base year. In cases, wherein information was unavailable for the base year, the years prior to it have been considered.

Research Methodology:

The market is estimated by triangulation of data points obtained from various sources and feeding them into a simulation model created individually for each market. The data points are obtained from paid and unpaid sources along with paid primary interviews with key opinion leaders (KOLs) in the market. KOLs from both, demand and supply side were considered while conducting interviews to get an unbiased idea of the market. This exercise was done at a country level to get a fair idea of the market in countries considered for this study. Later this country-specific data was accumulated to come up with regional numbers and then arrive at a global market value for the stem cell therapy market.

Key Players in the Stem Cell Therapy Market are:

Chiesi Farmaceutici S.P.A Are:Gamida CellReNeuron Group, plcOsiris Therapeutics, Inc.Stem Cells, Inc.Vericel Corporation.Mesoblast, Ltd.

Key Target Audience:

Stem Cell Associations and OrganizationsGovernment Research Boards and OrganizationsResearch and consulting firmsStem Cell Therapy Market InvestorsHealthcare Service Providers (including Hospitals and Diagnostic Centers)Stem Cell Therapeutic Product Manufacturing OrganizationsResearch LabsClinical research organizations (CROs)Stem Cell Therapy Marketing PlayersPharmaceutical Product Manufacturing Companies

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Scope of the Stem Cell Therapy Market Report:

Stem Cell Therapy market research report categorizes the Stem Cell Therapy market based on Application, End users, Treatment, and geography (region wise). Market size by value is estimated and forecasted with the revenues of leading companies operating in the Stem Cell Therapy market with key developments in companies and market trends.

Stem Cell Therapy Market, By Treatments:

Allogeneic Stem Cell TherapyAutologous Stem Cell Therapy

Stem Cell Therapy Market, By End Users:

HospitalsAmbulatory Surgical Centers

Stem Cell Therapy Market, By Application:

OncologyCentral Nervous System DiseasesEye DiseasesMusculoskeletal DiseasesWound & InjuriesMetabolic DisordersCardiovascular DisordersImmune System Disorders

Stem Cell Therapy Market, By Geography:

North AmericaEuropeAsia PacificMiddle East & AfricaLatin America

Available Customization:

With the given market data, Maximize Market Research offers customization of report and scope of the report as per the requirement

Regional Analysis:

Breakdown of the North America stem cell therapy marketBreakdown of the Europe stem cell therapy marketBreakdown of the Asia Pacific stem cell therapy marketBreakdown of the Middle East & Africa stem cell therapy marketBreakdown of the Latin America stem cell therapy market

Company Information:Detailed analysis and profiles of addition

Browse Full Report with Facts and Figures of Stem Cell Therapy Market Report at: https://www.maximizemarketresearch.com/market-report/stem-cell-therapy-market/522/

MAJOR TOC OF THE REPORT

Chapter One: Stem Cell Therapy Market Overview

Chapter Two: Manufacturers Profiles

Chapter Three: Global Stem Cell Therapy Market Competition, by Players

Chapter Four: Global Stem Cell Therapy Market Size by Regions

Chapter Five: North America Stem Cell Therapy Revenue by Countries

Chapter Six: Europe Stem Cell Therapy Revenue by Countries

Chapter Seven: Asia-Pacific Stem Cell Therapy Revenue by Countries

Chapter Eight: South America Stem Cell Therapy Revenue by Countries

Chapter Nine: Middle East and Africa Revenue Stem Cell Therapy by Countries

Chapter Ten: Global Stem Cell Therapy Market Segment by Type

Chapter Eleven: Global Stem Cell Therapy Market Segment by Application

Chapter Twelve: Global Stem Cell Therapy Market Size Forecast (2019-2026)

About Us:

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Myelofibrosis Treatment Market To Witness an Outstanding Growth During 2016-2022 – Zebvo

By daniellenierenberg

Myelofibrosis or osteomyelofibrosis is a myeloproliferative disorder which is characterized by proliferation of abnormal clone of hematopoietic stem cells. Myelofibrosis is a rare type of chronic leukemia which affects the blood forming function of the bone marrow tissue. National Institute of Health (NIH) has listed it as a rare disease as the prevalence of myelofibrosis in UK is as low as 0.5 cases per 100,000 population. The cause of myelofibrosis is the genetic mutation in bone marrow stem cells. The disorder is found to occur mainly in the people of age 50 or more and shows no symptoms at an early stage. The common symptoms associated with myelofibrosis include weakness, fatigue, anemia, splenomegaly (spleen enlargement) and gout. However, the disease progresses very slowly and 10% of the patients eventually develop acute myeloid leukemia. Treatment options for myelofibrosis are mainly to prevent the complications associated with low blood count and splenomegaly.

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The global market for myelofibrosis treatment is expected to grow moderately due to low incidence of a disease. However, increasing incidence of genetic disorders, lifestyle up-gradation and rise in smoking population are the factors which can boost the growth of global myelofibrosis treatment market. The high cost of therapy will the growth of global myelofibrosis treatment market.

The global market for myelofibrosis treatment is segmented on basis of treatment type, end user and geography:

As myelofibrosis is considered as non-curable disease treatment options mainly depend on visible symptoms of a disease. Primary stages of the myelofibrosis are treated with supportive therapies such as chemotherapy and radiation therapy. However, there are serious unmet needs in myelofibrosis treatment market due to lack of disease modifying agents. Approval of JAK1/JAK2 inhibitor Ruxolitinib in 2011 is considered as a breakthrough in myelofibrosis treatment. Stem cell transplantation for the treatment of myelofibrosis also holds tremendous potential for market growth but high cost of therapy is foreseen to limits the growth of the segment.

On the basis of treatment type, the global myelofibrosis treatment market has been segmented into blood transfusion, chemotherapy, androgen therapy and stem cell or bone marrow transplantation. Chemotherapy segment is expected to contribute major share due to easy availability of chemotherapeutic agents. Ruxolitinib is the only chemotherapeutic agent approved by the USFDA specifically for the treatment of myelofibrosis, which will drive the global myelofibrosis treatment market over the forecast period.

Geographically, global myelofibrosis treatment market is segmented into five regions viz. North America, Latin America, Europe, Asia Pacific and Middle East & Africa. Northe America is anticipated to lead the global myelofibrosis treatment market due to comparatively high prevalence of the disease in the region.

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Some of the key market players in the global myelofibrosis treatment market are Incyte Corporation, Novartis AG, Celgene Corporation, Mylan Pharmaceuticals Ulc., Bristol-Myers Squibb Company, Eli Lilly and Company, Taro Pharmaceuticals Inc., AllCells LLC, Lonza Group Ltd., ATCC Inc. and others.

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First Patient in CytoDyn’s Triple-Negative Metastatic Breast Cancer Trial Shows Significant Reduction in Circulating Tumor Cells (CTC) and Reduced…

By daniellenierenberg

VANCOUVER, Washington, Nov. 11, 2019 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today encouraging initial results from the first patient in a Phase 1b/2 clinical trial with metastatic triple-negative breast cancer (mTNBC). Circulating tumor cells (CTC) in the patients blood decreased significantly after leronlimab therapy at both two-week and five-week time points. Furthermore, a reduction in CCR5 expression on presumed metastatic tumor cells was evident.

We are excited to be involved with CytoDyn in evaluating the efficacy of leronlimab in mTNBC," stated IncellDx CEO, Bruce Patterson, M.D. These results at both two-week and five-week time intervals post-leronlimab therapy indicate initial efficacy against this most aggressive tumor type. Moreover, the reduction of CCR5 expression on EMT cells may prove to be significant, as high CCR5 expression is believed to be crucial for metastases.

The treatment of mTNBC with leronlimab in this Phase 1b/2 trial is in addition to metastatic breast cancer (MBC) patients treated with leronlimab under an emergency use IND. Results from both of the ongoing trials in MBC will dictate the Companys regulatory pathway, including the potential to seek Breakthrough Therapy designation and accelerated approval with the U.S. FDA for the use of leronlimab in MBC. Leronlimab has been granted Fast Track designation for mTNBC by the FDA based on a greater than 98% reduction of metastatic tumor volume in a murine xenograft model.

Today marks yet another significant milestone in our Companys history, advancing CytoDyns clinical development in oncology. Although these are early results in our first patient, we are encouraged by the reduction in both CTC and tumor size. Our safety record with leronlimab, and preclinical results in multiple oncology trials in various cancer indications, solidifies our vision to explore oncology indications. We are optimistic about the opportunity to provide a potential new therapeutic option for the women that are diagnosed with invasive breast cancer each year in the United States. We wish to thank the women who have agreed to participate in our trials and will endeavor to provide each of them with clinical benefit," stated CytoDyn President and CEO, Nader Pourhassan, Ph.D.

About Leronlimab (PRO 140)The U.S. Food and Drug Administration (FDA) has granted a "Fast Track" designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with highly active antiretroviral therapy (HAART) for HIV-infected patients, and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including non-alcoholic steatohepatitis (NASH). Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 plays a vital role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. CytoDyn is conducting additional research with leronlimab in the setting of oncology and NASH with plans to conduct further clinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD. Blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted "orphan drug" designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a crucial role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in 2019 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication, which, if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs).Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients. Some patients on leronlimab monotherapy have viral suppression for more than four years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and has received clearance to initiate a clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.

Forward-Looking StatementsThis press release contains certain forward-looking statements that involve risks, uncertainties, and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements but, their absence does not mean that a statement is not forward-looking. The Company's forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CONTACTSInvestors: Nader Pourhassan, Ph.D.President & CEOnpourhassan@cytodyn.com

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First Patient in CytoDyn's Triple-Negative Metastatic Breast Cancer Trial Shows Significant Reduction in Circulating Tumor Cells (CTC) and Reduced...

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Gary Pesselt: Vitality Healthcare is it worth the cost? – The Union of Grass Valley

By daniellenierenberg

Another seminar is again advertised in The Union. I first thought it might help my wife with neuropathy until I did some extensive research.

First off, Medicare does not cover stem cell injections. Bone marrow stem cell injections range from $2,000 to $5,000 or more. Read Consumer Research report at: https://www.consumerreports.org/medical-treatments-procedures/trouble-with-stem-cell-therapy.

Stem cell treatments are widely accepted only for two broad medical indications: to help treat a handful of blood disorders including leukemia and some forms of anemia and in some cases to help burn victims. Ask questions. Any doctor who offers stem cell therapy should be able to explain where the cells will come from, what will be done to them before theyre injected into your body, and how, exactly, they will resolve your illness or injury. He or she should also be able to offer you proof of safety and efficacy, even for experimental treatments. Dont rely on patient testimonials.

Stem cells survive much longer than ordinary cells, increasing the chance that they might accumulate genetic mutations. It might take only a few mutations for one cell to lose control over its self-renewal and growth and become the source of cancer. Please do your own research.

Gary Pesselt

Grass Valley

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Perfect match: How Birthright alumni saved the lives of 100s of strangers – The Jerusalem Post

By daniellenierenberg

Whoever saves a single life is considered by the Talmud to have saved the whole world.

In August 2013, Jeffrey Altadonna, who was on a Birthright trip, was tested at a bone marrow testing drive at the Jerusalem Gate Hotel.

It was perfectly ordinary summer day when the 29-year-old accountant from Sherman Oaks, California received the fateful phone call.

A 77-year-old woman from Los Angeles was the perfect stranger that he was deemed to save.

Diane Gebel, a widow from Cyprus, California was diagnosed with Acute Myeloid Leukemia (AML). Her husband had passed away right before she was diagnosed with cancer.

For an entire year, the donor and recipient need to remain anonymous to each other, but last week, the time came for the two to finally meet.

The two were honored at the Los Angeles One Huge Night Gala event hosted by Gift of Life.

In a statement, Birthright Israel explained that the gala also celebrated the successful 15-year partnership of Birthright Israel and Gift of Life.

This partnership has so far resulted in 83,000 Birthright donors joining the registry, with 1,900 matches made between patients and Birthright donors, and 241 life-saving transplants to date.

Prior to the meeting, Altadonna recalled his Birthright trip explaining that it was really great to see that part of the world, to go to where its our given right to visit.

It had a profound effect on my friendships and cultural Jewish identity to see that Jewish people are one people, and we have each others backs, he said. It left me with the feeling that I had backing in anything that I wanted to do in my life. It really felt like a family.

He recalled that after being swabbed at the drive, he didnt really think too much of it because everyone did it.

I got the donation call 15 months ago, he explained, adding that he immediately decided to donate. I find it very bizarre, that everyone is telling me Its such a great thing that you are doing. For me, it wasnt an option to say yes or no, its just, Okay, lets do this, Im a match. Im surprised that more people dont donate.

After doing preliminary tests, he took the plunge and donated.

I had to do it early in the morning, it lasted 6-8 hours a marathon blood donation and it was finished,Altadonna continued. It didnt seem all that hard to me.

He made it clear that this opportunity to help only came about because of the Birthright Israel and Gift of Life collaboration.

It wasnt a mission of mine. I wouldnt have gone out of my way to get swabbed, so it only happened as a result of their collaboration, he said.

In an emotional meeting, the two finally met. Of the meeting, Gebel stressed that she is here because of my selfless and generous donor.

For me, it was easy, I just had an infusion, but for him it was hard, she said. It takes a very special person to do that, to actually give the gift of life.

Gebel said she had been waiting to meet him.

I didnt know he was such a good looking guy, Gebel joked. Im here because of him. I was not ready to die. I had too much to live for.

She stressed that she has changed because of my new life.

I take risks, I live my life fully, she explained. My motto has always been that I want to go through life with a Cosmopolitan in one hand and a travel book in the other. Because of my donor, I can do that, so I thank him from the bottom of my heart.

Altadonna called on others to also take the plunge by getting tested and donating.

I stand here for my recipients valor, for her victory. I ask you to do the same: sit and swab today, so someone can swim and live tomorrow, he said.

Birthrights International CEO Gidi Mark said he was proud of our participants who register as donors and the powerful impact of such a simple choice when they get the call that they are the perfect match for a perfect stranger.

It is a great honor that Birthright Israel is able to assist in this mission, he added.

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An artist and a transplant researcher discuss the heart – Harvard Gazette

By daniellenierenberg

Doris A. Taylors so-called replacement ghost heart suggests something otherworldly, but the eerie-looking form is far from an apparition. Its an innovative approach to organ transplantation that has inspired many in the medical community and at least one artist.

The Texas researchers process piggybacks on natures sophisticated design. Together she and a team of researchers strip cells off human and animal cadaver hearts with a soapy solution, leaving ghostly white protein shells that retain the form of the organ. They inject them with a patients blood or bone-marrow stem cells, and the ghost hearts act as scaffolding on which the newly introduced cells can slowly transform into a beating muscle.

What we said was, Wouldnt it be really cool if we could wash the sick cells out and put the healthy cells back in? said Taylor, director of Regenerative Medicine Research and director of the Center for Cell and Organ Biotechnology at the Texas Heart Institute, during a recent talk at the Radcliffe Institute for Advanced Study.

The hope is that one day these regenerated hearts will resolve the most challenging issues transplant patients currently face: the lack of a permanent artificial replacement, concerns about rejection, and the shortage of viable donor hearts.

Taylors efforts are driving what could become a revolution in organ transplants, and they have sparked the creativity of transdisciplinary artist Dario Robleto, whose latest work, on view at the Johnson-Kulukundis Family Gallery in Radcliffes Byerly Hall, recreates in images and sounds the original pulse wave of the heart first captured in visual form by scientists in the 1900s. Robleto and Taylor, longtime friends and Texas residents, explored those connections during Mondays Radcliffe discussion, which was moderated by Jennifer Roberts, Elizabeth Cary Agassiz Professor of the Humanities.

Robletos exhibit, Unknown and Solitary Seas, touches on the overlap between the medical mysteries and workings of the vascular pump, and the metaphor for the heart as the emotional center of the soul. It includes a video installation that features recreated sounds of a beating heart from the 19th century, reconstructed images of how the earliest pulse waves first appeared on the page, and a series of heart waveform sculptures in brass-plated stainless steel.

Roberts said that with his work, Robleto acknowledges the pulse waves promise, their profundity, their scientific value, but he also reclaims some of their ambiguity and asks us to wonder whether we can or should accept that these waveforms have escaped the realms of art, culture, and emotional communication.

Taylor similarly views her work as a blend of the scientific and the human. It transcends complicated, complex science, she says, in that her ghost hearts require a kind of passion, commitment, care, attention, and nurturing similar to whats required by a small child. Its really about building hearts at the emotional, mental, spiritual, and physical level that I think is going to get them to work, she said.

For Robleto, big ideas, like the creation of a new human heart, require multiple perspectives.

The artist called Taylors work one of the most fascinating and definitely one of the most emotional things Ive ever seen. As an object, he added, the ghost heart is stunningly beautiful but it also raises questions about the self, identity, emotion, the notions of form and where memory is truly held, questions he thinks artists can help address. He cited two of the nations earliest heart transplants, after which the patients wives asked their husbands, who had received donor hearts, if they still loved them.

Taylors work, Robleto said, is right at the edge of identity and materiality and so when the day comes when someone says the first ghost heart transplant I think we will have a similar moment where perhaps we will be forced to re-evaluate what we ask from our heart metaphor.

Dario Robletos Unknown and Solitary Seas is on view in Byerly Halls Johnson-Kulukundis Family Gallery through Jan. 18, 2020.

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An artist and a transplant researcher discuss the heart - Harvard Gazette

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‘I’ve become best friends with the woman who saved my life’ – Telegraph.co.uk

By daniellenierenberg

When Nicky Turkoz (aboveleft) was diagnosed with leukaemia, her only hope was an anonymous stem-cell donation. That donor was Annette Hamson (above right) and the two, once strangers, now share an unbreakable bond

I was wrestling the Christmas decorations down from the loft when I got the call. I can remember hearing the landline ring and telling my daughter, Meltem, who was steadying the ladder, to leave it. They can call back if its urgent, I said. My mobile rang and we left that too, but the landline went again. I better get it, Mum, said Meltem. Someone clearly wants to talk to you. She answered it as I was hauling the last of the decorations down. Its the surgery, she said, passing the phone to me.

That morning Id had a blood test. Id been feeling strange for months and had finally made an appointment to see the GP. I told her I was feeling lethargic and depressed, which was very unlike me, but Id put it down to ageing and a spot of empty-nest syndrome, as my youngest daughter Zeynep had just left home to work abroad. Odd things, such as a recurring gum infection, were making me wonder if something else was going on. My GP seemed unconcerned but sent me for blood tests anyway. A few hours later she was telling me to pack an overnight bag and get to hospital. Your results show youre very anaemic, she said. Theyll need to do some more tests. You might be there a while.

She never used the word cancer. Looking back, I think she knew then but was sugar- coating it for me. We left the decorations.

Three hours later, after more prodding and poking, a consultant told me I had acute myeloid leukaemia, a blood cancer. It seems unbelievable now but I can remember thinking, Well, thank goodness theyve found something wrong with me, I knew I wasnt feeling great. I had no idea, of course, what was coming down the track.

It was a Wednesday evening and I was told they would start my chemotherapy on the Friday, but Id need to stay in hospital in the meantime. So while I was being installed on a ward, my poor daughter was handed a leaflet about myeloid leukaemia and drove home to our undecorated tree.

The first round of chemo lasted 10 days, which meant I had to miss Meltems 23rd birthday on 21 December. Zeynep left Gran Canaria, where she had just started working as a holiday rep, and flew home to be with us. It has always been just the three of us, ever since they were tiny. Im a very can-do sort of person and have always just got on with life whatever it has thrown at us. I couldnt bear it when my girls suddenly had to look after me. It all just seemed so unfair. I suppose everyone feels like that though.

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Exercise found to block chronic inflammation in mice – Harvard Gazette

By daniellenierenberg

Scientists at Harvard-affiliated Massachusetts General Hospital (MGH) have identified a previously unknown biological pathway that promotes chronic inflammation and may help explain why sedentary people have an increased risk for heart disease and strokes.

In a study to be published in the November issue ofNature Medicine, MGH scientists and colleagues at several other institutions found that regular exercise blocks this pathway. This discovery could aid the development of new therapies to prevent cardiovascular disease.

Regular exercise protects the cardiovascular system by reducing risk factors such as cholesterol and blood pressure. But we believe there are certain risk factors for cardiovascular disease that are not fully understood, said Matthias Nahrendorf of the Center for Systems Biology at MGH. In particular, Nahrendorf and his team wanted to better understand the role of chronic inflammation, which contributes to the formation of artery-clogging blockages called plaques.

Nahrendorf and colleagues examined how physical activity affects the activity of bone marrow, specifically hematopoietic stem and progenitor cells (HSPCs). HSPCs can turn into any type of blood cell, including white blood cells called leukocytes, which promote inflammation. The body needs leukocytes to defend against infection and remove foreign bodies.

When these [white blood] cells become overzealous, they start inflammation in places where they shouldnt, including the walls of arteries.

Matthias Nahrendorf

But when these cells become overzealous, they start inflammation in places where they shouldnt, including the walls of arteries, said Nahrendorf.

Nahrendorf and his colleagues studied a group of laboratory mice that were housed in cages with treadmills. Some of the mice ran as much as six miles a night on the spinning wheels. Mice in a second group were housed in cages without treadmills. After six weeks, the running mice had significantly reduced HSPC activity and lower levels of inflammatory leukocytes than the mice that simply sat around their cages all day.

Nahrendorf explains that exercising caused the mice to produce less leptin, a hormone made by fat tissue that helps control appetite, but also signaled HSPCs to become more active and increase production of leukocytes. In two large studies, the team detected high levels of leptin and leukocytes in sedentary humans who have cardiovascular disease linked to chronic inflammation.

This study identifies a new molecular connection between exercise and inflammation that takes place in the bone marrow and highlights a previously unappreciated role of leptin in exercise-mediated cardiovascular protection, said Michelle Olive, program officer at the National Heart, Lung, and Blood Institute Division of Cardiovascular Sciences. This work adds a new piece to the puzzle of how sedentary lifestyles affect cardiovascular health and underscores the importance of following physical-activity guidelines.

Reassuringly, the study found that lowering leukocyte levels by exercising didnt make the running mice vulnerable to infection. This study underscores the importance of regular physical activity, but further focus on how exercise dampens inflammation could lead to novel strategies for preventing heart attacks and strokes. We hope this research will give rise to new therapeutics that approach cardiovascular disease from a completely new angle, said Nahrendorf.

The primary authors of theNature Medicinepaper are Nahrendorf, who is also a professor of radiology at Harvard Medical School; Vanessa Frodermann, a former postdoctoral fellow at MGH who is now a senior scientist at Novo Nordisk; David Rohde, a research fellow in the Department of Radiology at MGH; and Filip K. Swirski, an investigator in the Department of Radiology at MGH.

The work was funded bygrantsHL142494 andHL139598from the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health.

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Rocket Pharmaceuticals to Present Preliminary Phase 1 Data of RP-L102 Process B for Fanconi Anemia at the 61st American Society of Hematology Annual…

By daniellenierenberg

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces presentations at the upcoming 61st American Society of Hematology (ASH) Annual Meeting being held December 7-10, 2019 in Orlando, Florida. The two poster presentations will highlight clinical data from the Phase 1 study of RP-L102 utilizing Process B for the treatment of Fanconi Anemia (FA), as well as long-term follow-up data from the Phase 1/2 EUROFANCOLEN trial.

Details for Rockets poster presentations are as follows:Title: Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor CellsSession Title: Gene Therapy and Transfer: Poster IIPresenter: Sandeep Soni, M.D.Session Date: Sunday, December 8, 2019Session Time: 6:00 p.m. 8:00 p.m. ESTLocation: Orange County Convention Center, Hall B

Title: Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene TherapySession Title: Gene Therapy and Transfer: Poster IIIPresenter: Paula Ro, Ph.D.Session Date: Monday, December 9, 2019Session Time: 6:00 p.m. 8:00 p.m. ESTLocation: Orange County Convention Center, Hall B

The Sunday poster session will be followed by a breakout session to give investors and analysts the opportunity to ask questions and discuss the data. The breakout session, hosted by Rocket management, will be held on Sunday, December 8th at 8:30 p.m. EST, directly after Dr. Sonis presentation. At the event, Dr. Soni, Clinical Associate Professor of Stem Cell Transplantation and Regenerative Medicine at the Stanford University School of Medicine and principal investigator of the U.S. Phase 1 trial of RP-L102 and Paula Ro, Ph.D., Senior Scientist, Divisin de Terapias Innovadoras en el Sistema Hematopoytico, CIEMAT/CIBERER Unidad Mixta de Terapias Avanzadas CIEMAT/IIS Fundacin Jimnez Daz will be participating in a Q&A panel. For further information, please contact investors@rocketpharma.com.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a FANC-A gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANC-A gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natures gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline program is for Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals to Present Preliminary Phase 1 Data of RP-L102 Process B for Fanconi Anemia at the 61st American Society of Hematology Annual...

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