By Aprille HansonAssociate Editor

Aprille Hanson

Deacon Butch King and his wife Debbie (left) stand with their daughter Paula Draeger (center) in front of the Seed of Hope garden at UAMS Winthrop P. Rockefeller Cancer Institute in Little Rock Dec. 4. Last month, King was able to place a seed of hope token into the garden, signifying he is cancer-free, thanks to a stem cell donation from his daughter.

Aprille Hanson

Deacon Butch King and his wife Debbie (left) stand with their daughter Paula Draeger (center) in front of the Seed of Hope garden at UAMS Winthrop P. Rockefeller Cancer Institute in Little Rock Dec. 4. Last month, King was able to place a seed of hope token into the garden, signifying he is cancer-free, thanks to a stem cell donation from his daughter.

Deacon Butch King was given a gift in 2017. He was diagnosed with a rare disease MDS/MPN, myelodysplastic/myeloproliferative neoplasm-unclassifiable to be exact.

The hybrid disease results when bone marrow overproduces unhealthy blood cells, according to University of Arkansas for Medical Sciences in Little Rock.

The diagnosis sent the family on a harrowing journey for the next two and a half years: four changes of insurance coverage and medical facilities, 19 rounds of chemotherapy, 430 lab results, 14 bone marrow biopsies, 11.25 gallons of donated blood and the disease progressing to Acute Myeloid Leukemia.

Looking at a deadly disease as a gift takes a radical faith in God, one that King and his wife Debbie have carried with grace to his cancer-free diagnosis Nov. 4.

It was given to us as a gift. And how do we manage gifts? We care for them, we nurture them, we polish them, show them off with pride and we give thanks to God. Those are his words, our words together. We had a gift and we had to manage it, we didnt get a choice, his wife said.

King was ordained a deacon in 2012, serving at Immaculate Conception Church in North Little Rock. The couple has four children, 12 grandchildren and six great-grandchildren, with another on the way in March. After 23 years of serving in the U.S. Air Force working in secure communications, he spent 22 years with the U.S. Postal Service.

In October 2016, he had a metal stent placed in his heart and could not have any surgeries for the following six months. In November of that year, he twisted his knee at work. When he was finally ready to have knee surgery in May, his lab work was irregular. In June they learned he had developed a rare blood disorder, MDS, which later in the year progressed to MPN. It required a stem cell transplant, with only a 30 percent chance of surviving a transplant.

I was kind of stunned at first, King said. As a deacon, he had been used to visiting the sick in nursing homes and hospitals.

This is one of the stories you can say, I know how you feel because Ive been there or were praying for you and really mean it, he said.

With every roadblock of insurance not covering the procedure or a hospital turning the transplant down because he was high risk, faith prevailed.

In December 2017, their youngest daughter Paula Draeger, 38, was a perfect match for a stem cell transplant, an extremely rare result.

OK, we can do this; were going to heal him. Weve got the perfect match. If this doesnt work, nothing will. So that was just kind of the reaction, lets do it, the married mother of two said.

Debbie King said, Shes a Spina bifida baby. We were told that she would be a vegetable when she had her spinal surgery. So shes a miracle to be here; long before this ever came God had a plan.

Once Medicare kicked in, insurance would cover a transplant if a clinical trial was available. It led the family to 13 visits to University of Oklahoma Stephenson Cancer Center in Oklahoma City, though they refused the transplant.

Debbie King said they specifically chose Oklahoma City because the family had been, and still are, praying daily for Blessed Stanley Rothers intercession.

The martyr, who grew up on a farm in Okarche, Okla., was declared blessed on Sept. 23, 2017, in Oklahoma City. He was killed in 1981 while serving his people in Guatemala.

He needed a miracle. And we said God provides miracles, Debbie King said.

Before we started any treatment we would place the entire illness and what would be happening at Blessed Stanley Rothers gravesite in Oklahoma City, visiting 11 times, she said.

Whats the miracle? The miracle is the faith. And thats what Butch has said, she said.

On March 13, the Kings were told they wouldnt be continuing the trial in Oklahoma.

We were ready to just be on maintenance and enjoy the days we had, she said. On March 14, our 44th wedding anniversary we were celebrating what we thought could be our last one.

But Dr. Appalanaidu Sasapu, hematologist oncologist with the UAMS Stem Cell Transplantation and Cellular Therapy Program, never gave up on them. Because Kings disease had progressed to leukemia in April, the stem cell procedure could now be done at UAMS and covered by insurance.

Draeger said the stem cell donations, done over a weekend via a port, were simple, with no side effects aside from building her energy up in the following week.

For what youre able to give somebody, what you have to endure pales in comparison to what hes been through and what you can give him, she said.

King no longer has the blood disease and is cancer free, though he will continue at least a years worth of chemotherapy treatments.

Since his diagnosis, they attend the smaller St. Patrick Church in North Little Rock for Mass, but he cannot yet return to ministry.

We do our prayer time in the mornings and evenings, we count our blessings every night before we go to bed and we just know, what was our blessing today? Did we see somebody that we havent seen before that God put in our path? Is it a new doctor who is going to take this on? King said.

But through this whole process weve been truly blessed, had no regrets. If I had to do it over, if thats the path of my life that God wants me to take, then Ill do it.

Please read our Comments Policy before posting.

Read the original post:
Deacon Butch King learns to accept the 'gift' of cancer - Arkansas Catholic

Bone Marrow Stem Cells Comments Off on Deacon Butch King learns to accept the ‘gift’ of cancer – Arkansas Catholic | December 12th, 2019

Regardless of the advancements made in understanding the genetic and molecular landscapes of acute myeloid leukemia (AML) in young patients, bespoke treatment approaches are yet to be adopted. Based on the NCCN 2009 guidelines,2 AML risk stratification depends on genetic/cytogenetic abnormalities of AML cells and groups patients into favorable-risk" (FR), intermediate-risk (IR) or poor-risk (PR) categories. This will then determine the treatment pathway of whether they will receive allogeneic stem cell transplantation (allo-SCT). Currently, treatment of IR patients is less defined and often post-induction treatment with allo-SCT is adopted. Nevertheless, this approach does not appropriately take into account the heterogeneity in this group regarding the risk of disease relapse. Despite high complete remission (CR) rates in these patients, overall survival (OS) remains low due to the high rate of relapse incidence.3

Adriano Venditti et al., from the Gruppo Italiano Malattie EMatologiche dellAdulto (GIMEMA) Foundation investigated in a multicenter prospective clinical trial (NCT01452646) the benefit of risk-adapted, MRD-based therapy in young adults with AML. This study consisted of the integration of pre-treatment cytogenetics/genetics with post-treatment MRD assessment (detected using multiparametric flow cytometry [MFC]) to stratify patients to receive post-consolidation autologous stem cell transplantation (auto-SCT) or allo-SCT.1

In conclusion, the authors recognized that this study has intrinsic limitations due to scientific progression over time, such as better understanding of basic biology, new AML classification and increased MRD monitoring, which makes the historical control not wholly comparable. However, this is the first study that attempts to apply a prospective program of risk-adapted, MRD-driven treatment in patients with NCCN-IR AML, that integrates genetics and post-consolidation MRD status.

Patients that were in the NCCN-IR group demonstrated that allo-SCT can be avoided if patients are tested MRD-negative after induction therapy. In contrast, if patients are MRD-positive, allo-SCT improved OS and prolonged DFS to comparable levels as those of patients in the NCCN-FR group. Patients in the IR group, who could not be monitored for LAIP had an inferior 2-year OS of 50% when compared to the other risk groups. This suggests that, in this group, a number of patients could have benefited from an allo-SCT. Further studies that integrate baseline factors and monitor MRD are needed, as it could potentially be a promising tool to refine and customize outcome prediction in patients with AML.

Link:
GIMEMA AML1310 trial of risk-adapted, MRD-guided therapy for young patients with newly diagnosed AML - AML Global Portal

Bone Marrow Stem Cells Comments Off on GIMEMA AML1310 trial of risk-adapted, MRD-guided therapy for young patients with newly diagnosed AML – AML Global Portal | December 12th, 2019

AgeX Therapeutics, Inc. (NYSE American: AGE) and Lineage Cell Therapeutics, Inc. (NYSE American and TASE LCTX), announced today that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" covering what is commonly designated "induced Pluripotent Stem (iPS) cells. The issued claims include methods to manufacture pluripotent cells capable of becoming any cell in the body. The patent has an early priority date, having been filed before the first scientific publication of Shinya Yamanaka, for which he won the Nobel Prize for Physiology or Medicine in 2012.

"This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state," said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. "Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development." A video describing the significance of the patent in AgeXs product development is available on the AgeX website.

"The issuance of this patent highlights Lineages dominant position in the field of cell therapy," stated Brian M. Culley, CEO of Lineage. "Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success."

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been "reprogrammed" or "induced" to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled "Methods of Reprogramming Animal Somatic Cells" was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

Story continues

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Certain statements contained in this release are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates" should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the "Risk Factors" section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005435/en/

Contacts

Media Contact for AgeX:Bill Douglass Gotham Communications, LLCbill@gothamcomm.com (646) 504-0890

Continue reading here:
AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells - Yahoo...

IPS Cell Therapy Comments Off on AgeX Therapeutics and Lineage Cell Therapeutics Announce Issuance of U.S. Patent for Method of Generating Induced Pluripotent Stem Cells – Yahoo… | December 12th, 2019

Lineage Cell Therapeutics and AgeX Therapeutics have been awarded a United States Patent and Trademark Office patent for Methods Of Reprogramming Animal Somatic Cells.

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr Michael D West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Patent 10,501,723 covers induced pluripotent stem cells which includes methods to manufacture iPSs cells that are capable of becoming any cell within the body. This patent has an early priority date having been filed before the first scientific publication, and was assigned to Advanced Cell Technology of Marlborough, Massachusetts and licenced to Lineage as well as being sublicensed to Age X for defined fields of use.

Original post:
Patent Granted To Lineage & AgeX - Anti Aging News

IPS Cell Therapy Comments Off on Patent Granted To Lineage & AgeX – Anti Aging News | December 12th, 2019

DUBLIN--(BUSINESS WIRE)--The "Global Blood and Bone Marrow Cancer Treatment Market Size, Market Share, Application Analysis, Regional Outlook, Growth Trends, Key Players, Competitive Strategies and Forecasts, 2019 to 2027" report has been added to ResearchAndMarkets.com's offering.

The global blood and bone marrow cancer treatment market was valued at US$ 38.8 Bn in 2018 and is expected to reach US$ 74.9 Bn by 2027, expanding at a CAGR of 7.7% from 2019 to 2027.

Market Insights

Blood cancer begins in the bone marrow which is the integral source of stem cells which later are differentiated in different types of blood cells in the human body. Researchers at Bristol Myers Squibb Company have stated that approximately 1.85 million new cases of blood cancer will be diagnosed by 2040 throughout the globe.

Lymphoma is the largest indication segment for blood and bone marrow cancer treatment market. It is prevalent in 2 types Hodgkin lymphoma and Non-Hodgkin lymphoma throughout the globe. The chief variables responsible for its rising prevalence worldwide are increasing prescription of immunosuppressant drugs for treating chronic infections and genetic mutations. Leukemia occurs when the DNA of immature white blood cells gets damaged due to exposure to ionizing radiation, hazardous chemicals, smoking, etc. The prevalence rate of leukemia is highly variable across different ethnic groups with men to women ratio of 1.4.

Chemotherapy is reigning the therapy segment for blood and bone marrow cancer treatment market. The key parameter hold responsible for its increasing demand is the availability of its generic version at affordable cost, drastically reducing the healthcare burden on ailing patients. Oncologists prefer to use them in combination therapy either with radiotherapy or immunotherapy to treat patients showing resistance to first-line drug therapy. Immunotherapy will be the fastest-growing segment during the forecast period owing to its promising drug pipeline for the treatment of blood cancer.

North America representing a market share of 34.6% is dominating the regional segment for blood and bone marrow cancer treatment market. The chief contributing factor for its market supremacy is a growing incidence of blood cancer. As per the research citings of the Leukemia and Lymphoma Society (CDC) figures after every 3 minutes, 1 person in the U.S. is diagnosed with blood cancer. In 2019, approximately 176,200 people in the U.S. are diagnosed with blood cancer in the United States. Europe holds a market share of 30.8% owing to the supportive regulatory framework provided by the European Medical Agency for the development and sale of medication for the treatment of blood cancer. The Asia Pacific accounts for 18.4% market share on account of rising public health awareness related to blood cancer & its treatment and developing healthcare infrastructure.

Key Market Movements:

Key Topics Covered:

Chapter 1. Preface

1.1. Report Scope and Description

1.1.1. Purpose of the Report

1.1.2. Target Audience

1.1.3. USP and Key Offerings

1.2. Research Scope

1.3. Research Methodology

1.3.1. Phase I-Secondary Research

1.3.2. Phase II-Primary Research

1.3.3. Approach Adopted

1.3.4. Top-down Approach

1.3.5. Bottom-up Approach

1.3.6. Phase III-Expert Panel Review

1.3.7. Assumptions

1.4. Market Segmentation

Chapter 2. Executive Summary

2.1. Global Blood and Bone Marrow Cancer Treatment Market Portraiture

2.2. Global Blood and Bone Marrow Cancer Treatment Market, by Indication, 2018 (US$ Bn)

2.3. Global Blood and Bone Marrow Cancer Treatment Market, by Therapy, 2018 (US$ Bn)

2.4. Global Blood and Bone Marrow Cancer Treatment Market, by Geography, 2018 (US$ Bn)

Chapter 3. Blood and Bone Marrow Cancer Treatment Market: Dynamics and Future Outlook

3.1. Market Overview

3.2. Drivers

3.3. Challenges

3.4. Opportunities

3.5. Attractive Investment Proposition, by Geography, 2018

3.6. Competitive Analysis: Global Blood and Bone Marrow Cancer Treatment Market, by Key Players, 2018

Chapter 4. Global Blood and Bone Marrow Cancer Treatment Market, by Indication

4.1. Overview

4.2. Multiple Myeloma

4.3. Leukemia

4.4. Lymphoma

4.5. Others

Chapter 5. Global Blood and Bone Marrow Cancer Treatment Market, by Therapy

5.1. Chemotherapy

5.2. Immunotherapy

5.3. Stem Cell Transplant

5.4. Radiotherapy

5.5. Pipeline Analysis

5.5.1. Phase III Drug

5.5.1.1. Eltrombopag

5.5.1.2. Avatrombopag

5.5.1.3. Hetrombopag

5.5.1.4. Omidubicel

5.5.1.5. Fedratinib

5.5.1.6. ATIR101

5.5.1.7. Pegylated Proline Interferon Alpha-2b

5.5.2. Tabular Representation of Phase II and I Pipeline Drugs

Chapter 6. Global Blood and Bone Marrow Cancer Treatment Market, by Geography

6.1. Overview

6.2. North America Blood and Bone Marrow Cancer Treatment Market Analysis, 2017- 2027

6.3. Europe Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.4. Asia Pacific Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.5. Latin America Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

6.6. Middle East and Africa Blood and Bone Marrow Cancer Treatment Market Analysis, 2017 - 2027

Chapter 7. Company Profiles

7.1. AstraZeneca, Plc.

7.1.1. Business Description

7.1.2. Financial Information (Subject to data availability)

7.1.3. Product Portfolio

7.1.4. News Coverage

7.2. Celgene, Inc.

7.3. Bristol Myers Squibb & Company

7.4. Eli Lilly & Company

7.5. Johnson & Johnson Company

7.6. F.Hoffman La-Roche Ltd.

7.7. Merck & Co., Inc.

7.8. Novartis AG

7.9. Pfizer, Inc.

7.10. Varian Medical Systems, Inc.

For more information about this report visit https://www.researchandmarkets.com/r/pi0qoz

Follow this link:
Global Blood and Bone Marrow Cancer Treatment Market Trends & Analysis During the Forecast Period, 2019-2027 - ResearchAndMarkets.com - Business...

Bone Marrow Stem Cells Comments Off on Global Blood and Bone Marrow Cancer Treatment Market Trends & Analysis During the Forecast Period, 2019-2027 – ResearchAndMarkets.com – Business… | December 10th, 2019

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email. "Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville. "But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the U.S. and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris. "It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection. "You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers. They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the U.S.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

Link:
2019: the year gene therapy came of age - Japan Today

Bone Marrow Stem Cells Comments Off on 2019: the year gene therapy came of age – Japan Today | December 10th, 2019

DURHAM, N.C., Dec. 10, 2019 (GLOBE NEWSWIRE) -- Chimerix, Inc. (Nasdaq: CMRX), a biopharmaceutical company focused on accelerating the development of medicines to treat cancer and other serious diseases, today announced that data relating to its dociparstat sodium (DSTAT) program, formerly known as CX-01, were presented at the 61st American Society of Hematology Annual Meeting, in Orlando, FL.

The poster, titledUpdated Study Results for CX-01, an Inhibitor of CXCL12/CXCR4, With Azacitidine for the Treatment of Hypomethylating Agent Refractory AML and MDS, was presented byEric Huselton, M.D., Assistant Professor of Medicine at the University of Rochester on December 9, 2019.

As reported in the published study abstract, 20 patients with refractory myelodysplastic syndrome (MDS) (n = 9) or refractory acute myeloid leukemia (AML) (n = 11) were enrolled of which 15 were considered evaluable for response with a bone marrow biopsy after cycle 2. Patients received a 7-day continuous infusion of DSTAT (CX-01) at a dose of 0.25 mg/kg/hour, and azacitidine 75 mg/m2 daily days 1-7, in 28-day cycles. The primary objective of this trial was to assess the overall response rate. Half of the patients had high risk cytogenetic abnormalities and 3 had TP53 mutations. Patients had a median of 2 prior lines of therapy (range 1-3) with median of 6 prior cycles of hypomethylating agent (HMA) therapy (range 4-20). Only 4 patients had a confirmed response to prior HMA therapy.

The 15 evaluable patients received a median of 3 cycles of CX-01 and azacitidine (range 2-9). Of 15 evaluable patients, there was 1 CR (complete remission) and 3 bone marrow CRs (mCR, with incomplete peripheral blood count recovery), 9 stable disease, and 2 progressive disease for an overall response rate of 27%. Of the 3 patients with a mCR after cycle 2, two had hematologic improvement of their neutrophil and platelet counts, respectively, by the end of cycle 4. A patient with stable disease also had hematologic improvement in platelets.

The median overall survival of evaluable patients was 221 days. The median overall survival was not significantly different between AML patients at 221 days and MDS patients at 248 days.

Following a minimum of 4 cycles of prior HMA therapy, one would not expect to observe response to subsequent HMA therapy, said Dr. Huselton. These results demonstrate DSTATs potential to improve HMA therapy outcomes in terms of both response and overall survival.

"DSTATs mechanism of action is intended to enhance patient benefit when combined with an active agent, so to observe these results in HMA-refractory patients is promising. In addition to our planned Phase 3 pivotal trial in newly diagnosed AML, this study highlights the potential to develop DSTAT to enhance the benefit of multiple therapies such as azacitidine, in AML and MDS in both front-line and recurrent settings," said Mike Sherman, Chief Executive Officer of Chimerix.

AboutChimerix

Chimerixis a development-stage biopharmaceutical company dedicated to accelerating the advancement of innovative medicines that make a meaningful impact in the lives of patients living with cancer and other serious diseases. The two clinical-stage development programs are dociparstat sodium (DSTAT) and brincidofovir (BCV).

Dociparstat sodium is a potential first-in-class glycosaminoglycan biologic derived from porcine heparin that has low anticoagulant activity but retains the ability to inhibit activities of several key proteins implicated in the retention and viability of AML blasts and leukemic stem cells in the bone marrow during chemotherapy (e.g., CXCL12, selectins, HMGB1). Mobilization of AML blasts and leukemic stem cells from the bone marrow has been associated with enhanced chemosensitivity and may be a primary mechanism accounting for the observed increases in EFS and OS in Phase 2 with DSTAT versus placebo. Randomized Phase 2 data suggest that DSTAT may also accelerate platelet recovery post-chemotherapy via inhibition of platelet factor 4, a negative regulator of platelet production that impairs platelet recovery following chemotherapy. BCV is a lipid conjugate DNA polymerase inhibitor in development as a medical countermeasure for smallpox.For further information, please visit the Chimerix website,www.chimerix.com

CONTACT:

Investor Relations:Michelle LaSpaluto919-972-7115ir@chimerix.com

Will OConnorStern Investor Relations212-362-1200will@sternir.com

Link:
Chimerix Presents Updated Results from Phase 2 Clinical Trial of DSTAT in Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia at American...

Bone Marrow Stem Cells Comments Off on Chimerix Presents Updated Results from Phase 2 Clinical Trial of DSTAT in Refractory Myelodysplastic Syndrome and Acute Myeloid Leukemia at American… | December 10th, 2019

The global bone marrow aspirate concentrates market was valued around US$ 130.0 Mn in 2016 is anticipated to register a stable CAGR of over 5.0% during forecast period of 2017 to 2025, according to a new report published by Transparency Market Research (TMR) titled Bone Marrow Aspirate Concentrates Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20172025.

Growth of the global bone marrow aspirate concentrates market is driven by increased prevalence of and incidences of orthopedic diseases, and sports injuries, along with high growth of the cosmetic surgery industry and increasing applications of the BMAC products in the cosmetic and orthopedic surgeries. The bone marrow aspirate concentrates market in Asia Pacific is expanding with a high potential to grow registering a CAGR above 6.0% on the backdrop of unmet clinical needs, rising geriatric population, large patient pool, favorable government regulations, development in health care sector, and increased focus on research and developmental activities.

Request a PDF Sample of Bone Marrow Aspirate Concentrates Market Report https://www.transparencymarketresearch.com/sample/sample.php?flag=S&rep_id=40451

Increase in incidences of Osteoarthritis on the backdrop of rising geriatric population to drive market growth

According to a collaborative survey conducted by the United Nations and the World Health Organization, 1.2 billion people in China are suffering from OA, of which more than 55% are aged 60 years or above. On the backdrop of such a huge patient base, there has been several developments in the field orthopedic surgery. Bone marrow-derived stem cell treatment is considered a promising and advanced therapy. It reduces the injury healing time in orthopedic diseases to five to six weeks from four to six months in case of surgery.

Reduction in the healing time is a factor likely to propel the Bone Marrow Aspirate Concentrates market during the forecast period. However, pain associated with the treatment, lack of product approval, and preference for alternative treatments are negatively affecting the market growth. Moreover, high investments in R&D and clinical trials, slow approval processes entailing sunken costs, and marginal returns on investment (RoI) for stakeholders are primary concerns faced by manufacturer further hampering growth of the market.

Rise in the Number of BMAC Assisted Procedures to Boost Growth of Bone Marrow Aspirate Concentrates Accessories Segment

The product type segment is fragmented into bone marrow aspirate concentrates systems and bone marrow aspirate concentrates accessories. The bone marrow aspirate concentrates accessories segment is anticipated to carry major share of the market on the backdrop of rise in number of BMAC assisted procedures. Cell therapies have been used extensively over the past decade for a variety of medical applications to restore cellular function and enhance quality of life. Owing to the differentiation property, stem cells are being used for repair and regeneration of bone. Moreover, increase in awareness about hygiene and risk of cross-contamination in developing countries such as Brazil, China and India are expected to increase the use of single-use Jamshidi needles for bone marrow stem cell procedures. This is likely to fuel the growth of the accessories segment in the near future.

Request for a Discount on Bone Marrow Aspirate Concentrates Market Report https://www.transparencymarketresearch.com/sample/sample.php?flag=D&rep_id=40451

Orthopedic Surgery Application to Dominate the Global Bone Marrow Aspirate Concentrates Market

The application segment of global bone marrow aspirate concentrates market is divided into orthopedic surgery, wound healing, chronic pain, peripheral vascular disease, dermatology, and others applications. Of which, orthopedic surgery segment is anticipated to dominate the market owing to rising geriatric population, and surge in incidences of osteoarthritis around the globe.

The dermatology segment is anticipated to expand at the highest CAGR of over 6.0% during forecast period of 2017 to 2025 owing to current boom in the industry, increase in disposable income, and technological advancements in the market. The utilization of the regenerative ability of fibroblasts and keratinocytes from human skin has formed new ways to develop cell-based therapies for patients. Moreover, capacity of bone marrow derived extra-cutaneous cells is being researched for its plasticity in regenerating skin; it is likely to lead to the future growth of cell therapies in dermatology.

Rise in Healthcare Expenditure to Fuel Growth of Hospitals & Clinics End-user Segment

In terms of end-users, market is divided into hospitals & clinics, pharmaceutical & biotechnology companies, Contract Research Organizations (CROs) & Contract Manufacturing Organizations (CMOs), and academic & research institutes. The hospitals & clinics segment dominated the bone marrow aspirate concentrates market in 2016. The trend is expected to continue during the forecast period. The hospitals & clinics segment is likely to be followed by the biotechnology & biopharmaceutical companies segment in terms of market share during the forecast period. The segment is anticipated to hold more than 8.0% of market share in 2016. Growth of the segment is attributed to increasing number of biotechnology companies and rising partnerships among the market players to expand global presence.

About Us

Transparency Market Research is a next-generation market intelligence provider, offering fact-based solutions to business leaders, consultants, and strategy professionals.

Our reports are single-point solutions for businesses to grow, evolve, and mature. Our real-time data collection methods along with ability to track more than one million high growth niche products are aligned with your aims. The detailed and proprietary statistical models used by our analysts offer insights for making right decision in the shortest span of time. For organizations that require specific but comprehensive information we offer customized solutions through adhoc reports. These requests are delivered with the perfect combination of right sense of fact-oriented problem solving method-ologies and leveraging existing data repositories.TMR believes that unison of solutions for clients-specific problems with right methodology of re-search is the key to help enterprises reach right decision.

ContactMr. Rohit BhiseyTransparency Market ResearchState Tower,90 State Street,Suite 700,Albany NY 12207United StatesTel: +1-518-618-1030USA Canada Toll Free: 866-552-3453Email: [emailprotected]Website: http://www.transparencymarketresearch.comBlog: https://theglobalhealthnews.com/

View original post here:
Bone Marrow Aspirate Concentrates Market to Expand at an Outstanding CAGR of 5% from 2017 to 2025 - VaporBlash

Bone Marrow Stem Cells Comments Off on Bone Marrow Aspirate Concentrates Market to Expand at an Outstanding CAGR of 5% from 2017 to 2025 – VaporBlash | December 10th, 2019

Researchers in China are laying claim to the first ever pig-monkey chimeras to be born in what they hope will be a breakthrough for biomedicine, not just fuel for your nightmares.

The worlds first monkey-pig hybrids have been born in a Chinese lab amid scientific attempts to grow human organs inside animals. The incredible experiment saw two chimera piglets born with DNA from both pigs and cynomolgus monkeys. However, the experiment was short-lived as the two piglets both died within a week.

According to the researchers, the baby piglets had genetic material from cynomolgus monkeys in their heart, liver, spleen, lung and skin.

However, the success rate was significantly low as only 10 piglets were born from more than 4,000 implanted in sows.

From those, only two were chimeras in a result that has been deemed discouraging by other leading stem cell scientists.

Lead researcher Tang Hai at the State Key Laboratory of Stem Cell and Reproductive Biology in Beijing said: This is the first report of full-term pig-monkey chimeras.

Ms Hai confirmed that it was unclear why the piglets died, but the non-chimeric pigs died as well.

The Chinese research team suspect that the deaths could have been due to the IVF process instead than the chimerism

According to research study, stem cells from macaque monkeys were grown in a lab and then injected into pig embryos five days after fertilisation.

Ms Hai said that the next step in their research was to create healthy animals with a higher proportion of monkey cells before creating pigs in which one organ is composed almost entirely of primate cells.

The ultimate research goal is to grow human organs inside live animals as a way to resolve the crisis of organ transplantation.

University of California stem cell biologist Paul Knoepfler said: Given the extremely low chimeric efficiency and the deaths of all the animals, I see this as fairly discouraging.

Reaction to the study has prompted many social media users to question if this is a step too far.

Read more here:
Researchers Manage to Create Pig-Monkey Chimeras for the First Time - Tdnews

Skin Stem Cells Comments Off on Researchers Manage to Create Pig-Monkey Chimeras for the First Time – Tdnews | December 10th, 2019

Theglobal market for cell harvestingshould grow from $885 million in 2018 to reach $1.5 billion by 2023 at a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023.

Report Scope:

The scope of the report encompasses the major types of cell harvesting that have been used and the cell harvesting technologies that are being developed by industry, government agencies and nonprofits. It analyzes current market status, examines drivers on future markets and presents forecasts of growth over the next five years.

The report provides a summary of the market, including a market snapshot and profiles of key players in the cell harvesting market. It provides an exhaustive segmentation analysis of the market with in-depth information about each segment. The overview section of the report provides a description of market trends and market dynamics, including drivers, restraints and opportunities. it provides information about market developments and future trends that can be useful for organizations, including wholesalers and exporters. It provides market positionings of key players using yardsticks of revenue, product portfolio, and recent activities. It further includes strategies adopted by emerging market players with strategic recommendations for new market entrants. Readers will also find historical and current market sizes and a discussion of the markets future potential. The report will help market players and new entrants make informed decisions about the production and exports of goods and services.

Request For Report Sample:https://www.trendsmarketresearch.com/report/sample/11685

Report Includes:

41 data tables and 22 additional tables Description of segments and dynamics of the cell harvesting market Analyses of global market trends with data from 2017, 2018, and projections of compound annual growth rates (CAGRs) through 2023 Characterization and quantification of market potential for cell harvesting by type of harvesting, procedure, end user, component/equipment and region A brief study and intact information about the market development, and future trends that can be useful for the organizations involved in Elaboration on the influence of government regulations, current technology, and the economic factors that will shape the future marketplace Key patents analysis and new product developments in cell harvesting market Detailed profiles of major companies of the industry, including Becton, Dickinson and Co., Corning, Inc., Fluidigm Corp., General Electric Co., Perkinelmer, Inc., and Thermo Fisher Scientific, Inc.

Summary

Stem cells are unspecialized cells that have the ability to divide indefinitely and produce specialized cells. The appropriate physiological and experimental conditions provided to the unspecialized cells give rise to certain specialized cells, including nerve cells, heart muscle cells and blood cells. Stem cells can divide and renew themselves over long periods of time. These cells are extensively found in multicellular organisms, wherein mammals, there are two types of stem cells embryonic stem cells and adult stemcells. Embryonic stem cells are derived from a human embryo four or five days old that is in the blastocyst phase of development. Adult stem cells grow after the development of the embryo and are found in tissues such as bone marrow, brain, blood vessels, blood, skin, skeletal muscles and liver. Stemcell culture is the process of harvesting the exosomes and molecules released by the stem cells for the development of therapeuticsfor chronic diseases such as cancer and diabetes. The process is widely used in biomedical applications such as therapy, diagnosis and biological drug production. The global cell harvesting market is likely to witness a growth rate of REDACTED during the forecast period of 2018-2023.The value of global cell harvesting market was REDACTED in 2017 and is projected to reach REDACTED by 2023. Market growth is attributed to factors such as increasing R&D spending in cell-based research,the introduction of 3D cell culture technology, increasing government funding, and the growing prevalence of chronic diseases such as cancer and diabetes.

The growing incidence and prevalence of cancer is seen as one of the major factors contributing to the growth of the global cell harvesting market. According to the World Health Organization (WHO), cancer is the second-leading cause of mortality globally and was responsible for an estimated 9.6 million deaths in 2018. Therefore, there is an increasing need for effective cancer treatment solutions globally. Cell harvesting is the preferred method used in cancer cell-related studies including cancer cell databases (cancer cell lines), and other analyses and drug discovery in a microenvironment. The rising prevalence of such chronic diseases has led governments to provide R&D funding to research institutes and biotechnology companies to develop advanced therapeutics. Various 3D cell culture technologies have been developed by researchers and biotechnology companies such as Lonza Group and Thermo Fischer Scientific for research applications such as cancer drug discovery. The application of cell culture in cancer research is leading to more predictive models for research, drug discovery and regenerative medicine applications.

Request For Report Discount :https://www.trendsmarketresearch.com/report/discount/11685

Platelet-rich plasma (PRP) therapy, a new biotechnology solution that has a heightened interest among researchers in tissue engineering and cell-based therapies, has various applications in the treatment of tissue healing in tendinopathy, osteoarthritis and muscle injury. It has been conventionally employed in orthopedics, maxillofacial surgery, periodontal therapy and sports medicines. PRP therapy can be used in the treatment of fat grafting, acne scars, and hair regrowth.

Major factors driving market growth include increasing healthcare costs and the high rate of adoption for modern medicines in emerging economies such as China and India. It has been estimated that India will witness a CAGR of REDACTED in the cell harvesting market during the forecast period. The active participation of foreign pharmaceutical companies has tapped the Indian healthcare sector with a series of partnerships and mergers and acquisitions, which in turn is positively impacting the growth of the market in this region. Consistent development and clinical trials for stem cell therapies, plus contribution from the government and private sectors through investments and cohesive reimbursement policies in the development of cancer biomarkers, is further fueling market growth. InSweden, a research team at Lund University has developed a device to collect fluid and harvest stem mesenchymal stem cells (MSCs). The device is developed with 3D-printed bio-inert plastics which, when used by doctors, can result in the safe extraction of fluids (medical waste) from the patients body. The liquid is then passed through a gauze filter for purifying thoroughly and MSCs are separated from the fluid by centrifugation and are grown in culture.

See the article here:
Cell Harvesting Market a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023 - Crypto News Byte

Skin Stem Cells Comments Off on Cell Harvesting Market a compound annual growth rate (CAGR) of 11.3% for the period of 2018-2023 – Crypto News Byte | December 10th, 2019

WALTHAM, Mass., Dec. 10, 2019 /PRNewswire/ --Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq: SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that it plans to commence a Phase 2 expansion cohort based on encouraging clinical activity and a well-tolerated safety profile observed to date in the ongoing Phase 1 dose escalation trial of SNDX-6352 in patients with chronic graft versus host disease (cGVHD). SNDX-6352 is the Company's anti-CSF-1R monoclonal antibody.

The ongoing Phase 1, open-label, modified 3+3 dose escalation trial is designed to evaluate the safety and preliminary efficacy of SNDX-6352 in up to 30 patients with cGVHD who have received at least two prior lines of therapy. As of a November 25, 2019 data cutoff date, a total of five patients, all of whom received prior treatment with ibrutinib, steroids, and a calcineurin inhibitor, have been enrolled across three dose cohorts: one patient was treated at 0.15 mg/kg every two weeks (Q2W, Cohort 1), one is receiving a dose of 0.5 mg/kg Q2W (Cohort 2), and three patients are receiving 1.0 mg/kg Q2W (Cohort 3).

Responses have been observed in all evaluable patients as of the data cutoff date, with no dose limiting toxicities (DLTs) reported. Among the three patients dosed in Cohort 3 (1.0 mg/kg Q2W), one patient recently cleared the DLT period and has not yet been evaluated for efficacy, two patients experienced a partial response, and all three patients remain on therapy. The patient in Cohort 2 experienced a partial response and is currently in their ninth month of treatment with SNDX-6352 following prior treatment with ibrutinib and both Jakafi (ruxolitinib) and KD025, two agents currently being investigated for the treatment of cGVHD. The first patient (Cohort 1) achieved a partial response but discontinued in their third cycle due to elevated LFTs attributed to progression in their liver cGVHD. Cohort 4, which will explore a 3.0 mg/kg Q2W dose, is now open for enrollment.

"The initial results from our Phase 1 trial underscore the potential of SNDX-6352 to serve as an effective therapy for patients with cGVHD who are lacking alternative options," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We had not anticipated commenting on data from this initial trial until the second half of 2020, so it is quite encouraging to see the early signs of activity in patients with this difficult to treat disease. Based on these results, we have decided to advance into a Phase 2 expansion cohort to evaluate additional patients at the 1.0 mg/kg dose while we continue the dose escalation to 3.0 mg/kg. We continue to expect to present the Phase 1 trial results in the second half of 2020.

"Published preclinical data have demonstrated that CSF-1R blockade can prevent and treat disease in animal models of cGVHD1," said Peter Ordentlich, Ph.D., Chief Scientific Officer and Co-founder of Syndax."The initial data from our trial provide the first clinical evidence that targeting CSF-1R dependent macrophages may benefit patients with cGVHD."

To date, SNDX-6352 has been safe and well-tolerated, with no DLTs observed. Dose escalation is ongoing in the Phase 1 portion of the trial. The Phase 2 expansion cohort is expected to enroll up to 22 patients to further characterize the safety and efficacy at an initial dosing schedule of 1.0 mg/kg of SNDX-6352 administered every two weeks.

About Chronic Graft Versus Host Disease

Chronic graft versus host disease (cGVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation (HSCT) which can last for years. cGVHD is estimated to develop in approximately 40% of transplant recipients, and affect approximately 14,000 patients in the US. 2-4 cGVHD typically manifests across multiple organ systems, with the skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue.

About SNDX-6352

SNDX-6352 is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages and the development of cutaneous and pulmonary chronic graft versus host disease (cGVHD), as well as to lead to the depletion of cells known as Tumor Associated Macrophages, or TAMS. SNDX-6352 is currently being evaluated in a Phase 1 multiple ascending dose clinical trial in cGVHD, and a Phase 1 multiple ascending dose clinical trial as monotherapy and in combination with Infinzi (durvalumab) in solid tumors. SNDX-6352 has the potential to treat a variety of solid tumor and immune-related diseases.

About Syndax Pharmaceuticals, Inc.

Syndax Pharmaceuticalsis a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. The Company's lead product candidate, entinostat, a once-weekly, oral, small molecule, class I HDAC inhibitor, is being tested in a Phase 3 combination trial with exemestane for treatment of advanced HR+, HER2- breast cancer and has been evaluated in combination with several approved PD-1/PD-(L)1 antagonists. The Company's pipeline also includes SNDX-6352, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor, and SNDX-5613, a highly selective inhibitor of the MeninMLL binding interaction. For more information, please visitwww.syndax.comor follow the Company on TwitterandLinkedIn.

Syndax's Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate," "estimate," "intend," "believe" and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials and the reporting of clinical data for Syndax's product candidates, and the potential use of our product candidates to treat various cancer indications. Many factors may cause differences between current expectations and actual results including unexpected safety or efficacy data observed during preclinical or clinical trials, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, failure of Syndax's collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

References

1. Alexander, KA. et al. J Clin Invest. 2014;124(10):42664280.

2.Kantar GVHD Expert Interviews N=8 interviews

3. SmartAnalyst 2017 SmartImmunology Insights chronic GVHD report.

4. Bachier, CR. et al. ASH annual meeting 2019; abstract #2109 Epidemiology and Real-World Treatment of Chronic Graft-Versus-Host Disease Post Allogeneic Hematopoietic Cell Transplantation: A U.S. Claims Analysis.

Syndax Contacts

Investor ContactMelissa ForstArgot Partnersmelissa@argotpartners.com212.600.1902

Media ContactCraig HeitGCI Healthcraig.Heit@gcihealth.com347.451.4733

SNDX-G

SOURCE Syndax Pharmaceuticals, Inc.

Home

Read more here:
Syndax Pharmaceuticals Announces Plans to Commence Phase 2 Expansion Cohort of SNDX-6352 for the Treatment of Chronic Graft Versus Host Disease -...

Skin Stem Cells Comments Off on Syndax Pharmaceuticals Announces Plans to Commence Phase 2 Expansion Cohort of SNDX-6352 for the Treatment of Chronic Graft Versus Host Disease -… | December 10th, 2019

The issuance of this patent highlights Lineages dominant position in the field of cell therapy, stated Brian M. Culley, CEO of Lineage. Our efforts to develop new treatments rely on well-characterized and NIH-approved human cell lines. These lines are not genetically manipulated, which avoids the safety concerns associated with genetic aberrations arising from the creation of iPS cells. We believe the Lineage cell lines provide the safest option for our current clinical-stage programs, particularly in immune-privileged anatomical sites such as the eye (OpRegen for the treatment of dry AMD) and spinal cord (OPC1, for the treatment of spinal cord injury). However, the vast intellectual property estate which underlies our cell therapy platform has never been limited to these particular cell lines. As one example, this newly-issued patent provides us with proprietary methods for producing induced pluripotent stem cells, or, as it was practiced by us prior to Yamanaka, Analytical Reprogramming Technology (ART). In certain settings, an ART/iPS approach might offer important advantages, such as for an autologous treatment or when the selection of preferential attributes from a series of iPS lines is desirable. Questions as to which stem cell technology is preferred ultimately will be answered by clinical safety and efficacy and likely will be indication-specific, so we believe it is in the best interest of our shareholders to generate patented technology which enables us to pursue programs in either or both formats which we believe will ensure the highest probability of success.

This patent broadly describes multiple techniques for reprogramming cells of the body back to the all-powerful stem cell state, said Dr. Michael D. West, CEO of AgeX and first inventor on the patent. Perhaps more significantly, it includes certain factors that address some of the difficulties currently encountered with iPS cells. It also reflects the foundational work our scientists have undertaken to apply reprogramming technology to age-reversal, specifically, induced Tissue Regeneration (iTR) which is currently a focus of AgeX product development.

Induced Pluripotent Stem Cells (iPS) are typically derived from adult skin or blood cells which have been reprogrammed or induced to retrace their developmental age and regain the potential to form all of the young cell and tissue types of the body. In 2010 inventors of the -723 patent issued today demonstrated that this reversal of developmental aging even extended to the telomere clock of cell aging. This reprogramming technology provides an alternate source of starting material for the manufacture of potentially any type of human cell needed for therapeutic purposes. Because iPSCs can be derived directly from adult tissues, they can be used to generate pluripotent cells from patients with known genetic abnormalities for drug discovery or as an alternative source of cell types for regenerative therapies.

U.S. Patent No. 10,501,723, entitled Methods of Reprogramming Animal Somatic Cells was assigned to Advanced Cell Technology of Marlborough, Massachusetts (now Astellas Institute for Regenerative Medicine) and licensed to Lineage and sublicensed to AgeX Therapeutics for defined fields of use. Inventors of the patent include Michael D. West, CEO of AgeX and previous CEO of Advanced Cell Technology, Karen B. Chapman, Ph.D., and Roy Geoffrey Sargent, Ph.D.

About Lineage Cell Therapeutics, Inc.

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineages programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineages clinical assets include (i) OpRegen, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. Lineage is also evaluating potential partnership opportunities for Renevia, a facial aesthetics product that was recently granted a Conformit Europenne (CE) Mark. For more information, please visit http://www.lineagecell.com or follow the Company on Twitter @LineageCell.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms. For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as believe, may, will, estimate, continue, anticipate, design, intend, expect, could, plan, potential, predict, seek, should, would, contemplate, project, target, tend to, or the negative version of these words and similar expressions. Such statements include, but are not limited to, Lineages exploration of alternative cell therapy platforms. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineages actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineages business and other risks in Lineages filings with the Securities and Exchange Commission (the SEC). Lineages forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading Risk Factors in Lineages periodic reports with the SEC, including Lineages Annual Report on Form 10-K filed with the SEC on March 14, 2019 and its other reports, which are available from the SECs website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005404/en/

See the rest here:
Lineage Cell Therapeutics and AgeX Therapeutics Announce Issuance of US Patent for Method of Generating Induced Pluripotent Stem Cells - BioSpace

Spinal Cord Stem Cells Comments Off on Lineage Cell Therapeutics and AgeX Therapeutics Announce Issuance of US Patent for Method of Generating Induced Pluripotent Stem Cells – BioSpace | December 10th, 2019

Brian Bowman, who presides over a city increasingly defined by rising crime, chaos, and a spiralling meth epidemic, apparently thought it would be smart to tweet out a photo of him looking happy to meet Chinas ambassador to Canada, Cong Peiwu.

Pleased to visit with Chinese Ambassador to Canada, Cong Peiwu. Thank you for a productive discussion about our Sister City Chengdu, trade, and Winnipegs goal of becoming a leader in the protection and promotion of human rights.

Pleased to visit with Chinese Ambassador to Canada, Cong Peiwu. Thank you for a productive discussion about our Sister City Chengdu, trade, and Winnipeg's goal of becoming a leader in the protection and promotion of human rights. pic.twitter.com/xe4hqpHrUI Mayor Brian Bowman (@Mayor_Bowman) December 5, 2019

You can already see the problems here.

First, if youre going to talk about the protection and promotion of human rights, you probably shouldnt be meeting with an ambassador from a country that has a horrific human rights record like China.

China has at least one million innocent Muslim people in concentration camps, is committing a cultural genocide, is breaking their One Country, Two Systems pledge in Hong Kong, and is threatening their neighbours.

Also, to thank Chinas ambassador after that same ambassador threatened Canada, and while China continues to jail the two Michaels is an absolute disgrace.

Unsurprisingly, Bowman is being ripped following the Tweet.

Below, you can see how badly Bowman got ratioed, with just 26 retweets and 482 comments.

Here is some of what people said:

How much money did communists pay you to make you praise their human rights disaster?! Swear you dont know Tiananmen Square Massacre, live organ harvesting, Uyghurs in concentration camps, HK protests being brutally cracked down! Where is your conscience sir?!

How much money did communists pay you to make you praise their human rights disaster?! Swear you dont know Tiananmen Square Massacre, live organ harvesting, Uyghurs in concentration camps, HK protests being brutally cracked down! Where is your conscience sir?! pic.twitter.com/RvowTsYR0C

Whose going to tell him they have two of our people in torture like conditions?

#TheMichaels will appreciate the irony in the Human Rights part

#TheMichaels will appreciate the irony in the Human Rights part Mr. Omega Man -1965 (@Keepershiny) December 7, 2019

Human rights and industrial scale murder for organs are not compatible

Its time to drop Chengdu as a sister city. And certainly time to revoke any future invitations to ANYONE who denies the existence of these concentration camps. Oh, and youve officially lost my vote.

Its time to drop Chengdu as a sister city. And certainly time to revoke any future invitations to ANYONE who denies the existence of these concentration camps. Oh, and youve officially lost my vote. Long time listener, first time caller (@EdVorst) December 7, 2019

Bowman was also ripped by some Canadian leaders, including Conservative MP James Bezan, and former Canadian Ambassador to China David Mulroney:

Statements like this play into the PRCs agenda of obfuscation, and actually undermine efforts to protect human rights. They reflect the false assumption that diplomacy cannot cope with plain-speaking and hard truths

Statements like this play into the PRC's agenda of obfuscation, and actually undermine efforts to protect human rights. They reflect the false assumption that diplomacy cannot cope with plain speaking and hard truths David Mulroney (@David_Mulroney) December 7, 2019

Too bad @Mayor_Bowman was played. The only discussions with Chinese officials should be about the release of Michael Kovrig & Michael Spavor, removing Chinese sanctions on our agriculture products, & respecting human rights in China. Until then, everything else is off the table.

Too bad @Mayor_Bowman was played. The only discussions with Chinese officials should be about the release of Michael Kovrig & Michael Spavor, removing Chinese sanctions on our agriculture products, & respecting human rights in China. Until then, everything else is off the table. https://t.co/v0H999derC James Bezan (@jamesbezan) December 8, 2019

Bowman has made a fool of himself, and sent a message that will encourage China to treat Canada even worse, as they once again see that the only response from Canadas political elites is pathetic weakness.

If Bowman had any integrity he would listen to Yaakov Pollak and resign.

Read the original:
Canadians are filing for multiple bankruptcies and it's costing everyone - The Post Millennial

Spinal Cord Stem Cells Comments Off on Canadians are filing for multiple bankruptcies and it’s costing everyone – The Post Millennial | December 10th, 2019

Many of us biologists conduct fieldwork in diverse places, from Alaska to the tropics, from aiming to understand how microbes are responding to climate change in the boreal soils to learning about life history strategies and co-evolutionary arms races of bats, their ectoparasitic flies, and the ectoparasitic fungi living on those flies.

The days before fieldwork tend to be hectic: make a checklist to make sure you have everything you need, think about a plan B (and a plan C, just in case), anticipate drawbacks and plan on how to address them, and the list goes on and on. The day comes. You make it to your field site, you collect the samples you want, obtain the data you need, everything works out just like planned, and you make it back to the lab safe, on time, and without going over your planned budget. This is how it should be, but it never really goes like that.

Fieldwork is one of the most exciting experiences about doing research. It is also, in many cases, high-risk. During fieldwork, many things can go wrong, and most of those things cannot be helped. We cannot control the appearances of massive puddles in the middle of the road, critically damaging our transportation vehicles. We cannot control the thunderstorm that makes our study organisms disappear when we finally arrive at a remote field site after hours of climbing a mud-covered mountain.

Sadly, this is not always the case for threats to our integrity as human beings, and we, as a scientific community, have done far too little to address this problem. People from underrepresented groups in the sciences such as people of color, women, and those who identify as LGBTQIA+ or gender nonconforming often are at higher risk of suffering abuse during fieldwork. This comes in the form of sexual harassment, sexual abuse, discrimination, and intimidation. Scientists who have experienced abuse often fear talking about it because they are traumatized and because they fear retaliation and backlash, especially if the perpetrators of abuse are colleagues or superiors advisers and people at higher career stage.

In Spring 2018, we carried out an anonymous survey to collect testimonies of what scientists, specifically from the LGBTQIA+ community, experience during fieldwork. The idea for such a survey sprouted from concerns that sexual orientation or gender identity may play an unwanted or unwarranted role in peoples professional career. Especially during fieldwork, when Diversity and Inclusion Offices from our university campuses are far away, LGBTQIA+ researchers are exposed to people who may not agree with their sexual orientation or who do not understand why he may want to be addressed as they.

Responses revealed experiences ranging from discrimination to situations that made researchers decide to no longer perform fieldwork outside of safe places. This adds a whole new level to fieldwork stress, namely having to evaluate sites for their tolerance towards LGBTQIA+. In one story from fieldwork, men voiced discomfort because an openly gay man would share a room with them while, simultaneously, women felt uncomfortable due to the possibility of having to share a room with someone from the opposite sex. Another survey respondent described that they were fearful to carry out fieldwork in places that are recognized for their homophobic culture. These experiences leave people feeling isolated and rejected.

We present a few strategies that we can instill in STEM fields to avoid cases like these:

1) INFORM PEOPLE ABOUT LGBTQIA+. Erase any misinformation that may exist. For example, a gay man is not a threat to the sexuality of cisgender males. Institutions can facilitate trainings on diversity and inclusiveness and provide information on the LGBTQIA+ community to eliminate negative stereotypes.

2) HAVE SUFFICIENT FUNDING AVAILABLE FOR FIELDWORK. Although sometimes it's unavoidable to share rooms due to limited budget or space, if there is the possibility to do so, provide individual lodging for people traveling to fieldwork or conferences. Especially for those who ask for it.

3) DEVELOP AN EMERGENCY PROTOCOL. As a lab, department, or institution, develop a protocol that scientists can follow as a response to experiencing a threat to their integrity. Protocols like this should be part of a broader departmental or university-wide mission statement about equity in field work. The bar has been set high by this example of a mission statement written by University of California Irvine professor Kathleen Treseder.

4) AVOID INTOLERANT AREAS. It is important to note that this does not only apply to countries like Niger and Tunisia where discriminatory laws expose LGBTQIA+ individuals to the risk of death penalty. It also applies close to home, in the USA, where there is an ongoing debate about public restrooms and which one transgender people and people who identify as gender-nonconforming should use.

5) IMPLEMENT A ZERO-TOLERANCE POLICY. Inform everyone in your lab, department and institution that there is a zero-tolerance policy regarding abuse. A code of conduct with expected versus unaccepted behavior and practices should always be made available through trainings and in field stations.

Continue reading here:
Your Apple smartwatch may be the key to detecting heart issues before they happen - Massive Science

Cardiac Stem Cells Comments Off on Your Apple smartwatch may be the key to detecting heart issues before they happen – Massive Science | December 10th, 2019

In the 4th edition of MarketsandMarkets Bioprocessing of Advanced Cellular Therapies & Regenerative Medicine, we would be focusing on the pre-clinical, manufacturing, clinical and regulatory aspects of cell therapies and regenerative medicine. This Congress event will be held on 10th and 11th March 2020 in London -UK

Since the past three editions of Bioprocessing of Advanced Cellular Therapies and Regenerative Medicine, MarketsandMarkets aims to provide a demonstrative approach to the latest developments in technologies of bioprocessing of cellular therapies.

What to expect:

The 4th edition of MarketsandMarkets Bioprocessing of Advanced Cellular Therapies & Regenerative Medicine would be concentrating on the pre-clinical, manufacturing, clinical and regulatory facets of cell therapies and regenerative medicine. The prime importance would be given on discussing topics such as tissue engineering, car-T cell-based immunotherapies, automated manufacturing, allogeneic therapies, from challenges in supply chain management and regulatory concern, point of view.

The conference will be useful for all the respective stakeholders of Advanced Cellular Therapies, majorly Pharma/Biotech delegates, Solution provider Delegates and Academic Delegates. The event will host VPs, directors, managers, leaders, engineers, scientists, academic heads, students which will boost the networking capacity of the attendees.

Download Agenda at https://www.reportsnreports.com/events/4th-annual-marketsandmarkets-bioprocessing-of-advanced-cellular-therapies-regenerative-medicine-congress/

Conference Agenda:

The two-day conference will have a list of agenda:

Key Pointers 4th Annual MarketsandMarkets Bioprocessing of Advanced Cellular Therapies & Regenerative Medicine Congress

Conference Registration

Lets get you sorted! Choose which applies best to you @ https://www.reportsnreports.com/events/4th-annual-marketsandmarkets-bioprocessing-of-advanced-cellular-therapies-regenerative-medicine-congress/register

Continued here:
MarketsandMarkets 4th Annual Bioprocessing of Advanced Cellular Therapies & Regenerative Medicine Congress - Galus Australis

Cardiac Stem Cells Comments Off on MarketsandMarkets 4th Annual Bioprocessing of Advanced Cellular Therapies & Regenerative Medicine Congress – Galus Australis | December 9th, 2019

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, today announced updated results from a Phase 1 clinical study of GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy for the treatment of patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (MM), at the 61st Annual Meeting of the American Society of Hematology (ASH), which is being held December 710 in Orlando, FL. Data from 22 patients in the ongoing study showed GDA-201 in combination with monoclonal antibodies was generally well tolerated and demonstrated early evidence of clinical activity in heavily pre-treated patients, including five complete responses observed among nine patients with NHL. Gamida Cell plans to initiate a Phase 1/2 multi-dose, multi-center study of GDA-201 in patients with NHL in 2020.

NK cells are increasingly recognized as a potential breakthrough approach in immunotherapy, and the data reported today provide early evidence that GDA-201 has the potential to be an important new treatment option, said Veronica Bachanova, M.D., Ph.D., Associate Professor of Medicine in the Division of Hematology, Oncology and Transplantation at the University of Minnesota and principal investigator of the study through the Masonic Cancer Center. Given the population of heavily pre-treated patients with advanced disease, its particularly encouraging to witness multiple complete responses. I look forward to the continued development of this investigational therapy.

New research was also presented today on the mechanism of action of Gamida Cells NAM-based cell expansion platform, which is designed to enhance the number and functionality of allogeneic donor cells. These data provide further scientific rationale for the favorable stem cell engraftment and patient outcomes observed in the Phase 1/2 clinical study of omidubicel, the companys advanced cell therapy currently in Phase 3 clinical development as a potential life-saving treatment option for patients in need of an allogeneic bone marrow transplant.

These mechanism of action data reinforce the transformative potential of our NAM therapeutic platform, which can be used to expand multiple cell types. Specifically for omidubicel, this research suggests that NAM modulates certain gene expression pathways that, collectively, mimic the hypoxic environment of the bone marrow to help preserve stem cell function and long-term engraftment ability, said Tracey Lodie, Ph.D., chief scientific officer of Gamida Cell. We expect to build on our findings by characterizing the metabolites produced when we expand stem cells to make omidubicel, and we are also beginning to conduct similar mechanism of action studies with GDA-201.

GDA-201 Phase 1 Clinical Data Presented at ASH

The oral presentation, Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer Cells (NAM-NK) in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma (MM) (Abstract #777), described data from the Phase 1 clinical study of GDA-201 in heavily pre-treated patients with advanced NHL and MM. Twenty-two patients were enrolled in the study, including nine patients with NHL and 13 patients with MM. Of these 22 patients, all were evaluable for safety and 21 were evaluable for response (NHL = 9; MM = 12).

In the study, cell therapy using GDA-201 with monoclonal antibodies was generally well tolerated and demonstrated early evidence of clinical activity. Of the nine patients with NHL, five achieved a complete response and one achieved a partial response. Among the patients with MM, one patient achieved a complete response, and five patients achieved stable disease.

GDA-201 was generally well tolerated, with no graft vs. host disease (GvHD), no tumor lysis syndrome, no neurotoxicity and no marrow aplasia observed. No dose limiting toxicities were observed. Hypertension and hematologic events were the most common Grade 3/4 adverse events observed. Most non-hematologic toxicities were attributed to cyclophosphamide/fludarabine, which was used as a pre-conditioning treatment.

NAM Therapeutic Platform Mechanism of Action Data Presented at ASH

The poster presentation, Nicotinamide (NAM) Modulates Transcriptional Signature of Ex Vivo Cultured UCB CD34+ Cells (Omidubicel) and Preserves Their Stemness and Engraftment Potential (Abstract #3718), included transcriptome, transcription factor, and pathway analysis to elucidate the pathways leading to the preservation of engraftment after ex vivo expansion of CD34+ hematopoietic stem cells derived from umbilical cord blood (the starting point for omidubicel) compared to CD34+ cells grown in the absence of NAM.

Analyses showed that the presence of NAM reduced the expression of genes involved in the production of reactive oxygen and nitrogen species, suggesting that cell stress was minimized during expansion. In addition, NAM also decreased growth factor pathways responsible for activation and differentiation of hematopoietic stem cells, suggesting NAM expanded cells while keeping them in an undifferentiated state. The presence of NAM also led to a decrease in the expression of genes responsible for matrix-metallo proteinase secretion, simulating the microenvironment of the bone marrow. Additionally, NAM led to an increased expression of telomerase genes, which is believed to enable cells to remain in a more quiescent, stem-like state. These data provide further scientific rationale for the favorable stem cell engraftment and patient outcomes that were observed in the Phase 1/2 clinical study of omidubicel.

About GDA-201GDA-201 (formerly known as NAM-NK) is being developed as an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies. NK cells have the ability to kill tumor cells, representing a novel immunotherapeutic approach to cancer treatment. GDA-201 is designed to address key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development in patients with refractory non-Hodgkin lymphoma and multiple myeloma.1 For more information on the clinical study of GDA-201, please visit http://www.clinicaltrials.gov.

About OmidubicelOmidubicel (formerly known as NiCord), the companys lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.2 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Latin America, Europe and Asia.3 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.4 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

GDA-201 and omidubicel are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About the NAM Therapeutic PlatformGamida Cells proprietary NAM-based cell expansion platform is designed to enhance the number and functionality of donor cells in culture, enabling the creation of potentially transformative therapies that move beyond what is possible with existing approaches. The NAM therapeutic platform leverages the unique properties of nicotinamide to enable the expansion of multiple cell types including stem cells and natural killer (NK) cells with appropriate growth factors to maintain the cells original phenotype and potency. This can enable the administration of a therapeutic dose of cells with the potential to improve patient outcomes.

About Gamida CellGamida Cell is an advanced cell therapy company committed to finding cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com.

Cautionary Note Regarding Forward Looking StatementsThis press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the initiation of new clinical trials and the continuation of the Companys clinical development program, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope and progress of Gamida Cells clinical trials and other clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of Gamida Cells public filing on Form 20-F, filed with the SEC on February 25, 2019, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

1ClinicalTrials.gov identifier NCT03019666.2 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.3 ClinicalTrials.gov identifier NCT027302994 ClinicalTrials.gov identifier NCT03173937

Continued here:
Gamida Cell Announces Results from Phase 1 Study of GDA-201 and New Mechanism of Action Data at ASH 2019 Annual Meeting - Business Wire

Bone Marrow Stem Cells Comments Off on Gamida Cell Announces Results from Phase 1 Study of GDA-201 and New Mechanism of Action Data at ASH 2019 Annual Meeting – Business Wire | December 9th, 2019

ORLANDO, Fla.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a new biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies, today announced that initial results from an ongoing Phase 1 dose-escalation study of its lead product candidate, JSP191 (formerly AMG191), will be presented today in an oral session at the American Society of Hematology (ASH) Annual Meeting & Exposition.

JSP191, a humanized antibody targeting CD117, is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplantation. The Phase 1 clinical trial is evaluating JSP191 as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that had poor outcomes.

Life-threatening disorders such as SCID, and other conditions including autoimmune diseases and hematologic cancers, can be cured by hematopoietic cell transplantation, and those with certain genetic diseases can be cured with stem cell-directed gene therapies. However, the number of patients who can benefit from these approaches is limited because of the severe toxicity of the chemotherapy used for pre-transplant conditioning that is needed to allow room in the bone marrow for the stem cells to engraft, said Judith Shizuru, M.D., Ph.D., co-founder and member of the Board of Directors of Jasper Therapeutics. We are encouraged by the initial Phase 1 study results of JSP191 in these fragile patients with SCID and plan to expand clinical development of this antibody beyond patients with SCID. We expect to initiate clinical trials of JSP191 in 2020 to evaluate it as a conditioning agent in patients undergoing hematopoietic cell therapy for acute myeloid leukemia, myelodysplastic syndrome and Fanconi anemia, and IND-enabling studies for sickle cell disease and autoimmune indications.

Details of the oral presentation follow:

Abstract Title: Non-Genotoxic Anti-CD117 Antibody Conditioning Results in Successful Hematopoietic Stem Cell Engraftment in Patients with Severe Combined Immunodeficiency (abstract #800) Session Name: 721. Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Innovative Approaches in Allogeneic Transplantation for Pediatric or Nonmalignant DisordersPresenter: Rajni Agarwal, M.D., Associate Professor of Pediatrics and Stem Cell Transplantation, the Stanford University School of MedicineTime: 3:00 p.m. ETLocation: W311EFGH, Level 3, Orange County Convention Center

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow is currently achieved by subjecting patients to toxic treatment with radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191

JSP191 (formerly AMG191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1 dose-escalation trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at http://www.jaspertherapeutics.com.

See the original post here:
Jasper Therapeutics Announces Upcoming Data Presentation on Lead Program, JSP191, at 61st American Society of Hematology (ASH) Annual Meeting &...

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics Announces Upcoming Data Presentation on Lead Program, JSP191, at 61st American Society of Hematology (ASH) Annual Meeting &… | December 9th, 2019

NEW ORLEANS, Dec. 9, 2019 /PRNewswire/ --Autoimmune Technologies LLC, a biotechnology company developing diagnostics and therapies for non-infectious and infectious disease, has established a new subsidiary, Stadius Biopharma LLC, to focus on proprietary anti-infective antibody therapeutics for diseases that are inadequately addressed by current standard-of-care medicines.

"Within this standalone entity we'll concentrate on our unique fully human antibodies and our stem cell antibody delivery platform to treat opportunistic viral, bacterial, and fungal infections," said Michael Charbonnet, who will serve as CEO of the newly formed business unit in addition to his continuing responsibilities for the parent company. Current Stadius antibody targets include invasive candidiasis and cytomegalovirus (CMV) infection.

"Ourantibodies bind and disrupt the function of conserved sequences of novel virulence factors associated with pathology of various species of Candida," said CSO Russell Wilson. "Candida aurisis an emerging fungus that presents a serious global health threat, and we'reencouraged by our initial preclinical data that indicates activity against C. auris as well as C. albicans, which is prevalent in high risk patients," he said.

The CDC is monitoring the spread of C. auris colonization and infection in the United States.C. aurisis associated with a high rate of morbidity and mortality and is resistant to current standard of care antifungal treatments. Healthy individualsunknowingly colonized with C. aurisor other Candida species and can spread the fungal cells to surfaces in hospitals, long-term-care facilities, and other healthcare environments, where they pose a threat to people with weakened immune systems.

Also under development are antibody therapeutics to CMV infection. More than 50% of individuals in the U.S. over 40 years of age have been exposed to CMV and carry it without symptoms. It can re-activate in patients with subpar immune systems with the potential for multi-organ involvement, and it can be transmitted through body fluids such as breast milk and saliva. Over time, individuals can become re-infected with different strains of CMV, further complicating prophylaxis and treatment. CMV is the most frequent infectious complication following both solid organ transplantation and bone marrow transplantation.

The company is also developing its proprietary non-viral gene modified allogeneic stem cell platform for delivering antibodies in chronically immune compromised patients. The somatic adipose-derived mesenchymal stem cells, which have unique properties to allow prevention of rejection, are transfected with the genetic information needed to enable the implanted stem cells to produce inside the patient the antibodies needed to prevent or treat infection. Antibody delivery to patients via this platform is intended to provide continuous protection while reducing the requirement for more frequent dosing.

Follow Stadius Biopharma on http://www.stadiusbio.com.

View original content to download multimedia:http://www.prnewswire.com/news-releases/autoimmune-technologies-announces-new-subsidiary-to-develop-novel-antibody-and-allogeneic-somatic-stem-cell-therapeutics-for-the-treatment-of-invasive-candidiasis-and-other-infectious-disease-300971143.html

SOURCE Autoimmune Technologies LLC

See more here:
Autoimmune Technologies Announces New Subsidiary to Develop Novel Antibody and Allogeneic Somatic Stem Cell Therapeutics For the Treatment of Invasive...

Bone Marrow Stem Cells Comments Off on Autoimmune Technologies Announces New Subsidiary to Develop Novel Antibody and Allogeneic Somatic Stem Cell Therapeutics For the Treatment of Invasive… | December 9th, 2019

PALO ALTO, Calif.--(BUSINESS WIRE)--Jasper Therapeutics, Inc., a new biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies, today announced the launch of the company with a $35 million total Series A financing. Abingworth LLP and Qiming Venture Partners USA served as lead investors, with further investment from Surveyor Capital (a Citadel company) and participation from Alexandria Venture Investments, LLC. The proceeds will be used to advance the clinical development of the companys lead product candidate, JSP191, which is designed to replace or reduce the toxicity of chemotherapy and radiation therapy as a conditioning regimen to prepare patients for hematopoietic cell transplant.

Jaspers development of JSP191 is also supported by a collaboration with the California Institute for Regenerative Medicine (CIRM), which has been funding the program and is committed to providing a total of $23 million in grant support. As part of the Series A financing, Amgen, which discovered JSP191 (formerly AMG191), has licensed worldwide rights to Jasper that also include translational science and materials from Stanford University.

Jasper was co-founded by Judith Shizuru, M.D., Ph.D., a hematopoietic stem cell transplant expert at Stanford University, and Susan Prohaska, Ph.D., a Stanford University-trained immunologist, stem cell biologist and early-stage drug development professional. Dr Shizurus CIRM-funded lab advanced the understanding of the ability of anti-CD117 to impact hematopoietic stem cells and, together with the Lucile Packard Childrens Hospital Stanford and University of California, San Francisco (UCSF) pediatric transplant teams, was the first to study an anti-CD117 antibody in the clinic as a conditioning agent. That humanized antibody, now called JSP191, was first studied for conditioning for transplant in immune-deficient patients in collaboration with Amgen, UCSF and CIRM.

Stem cell transplantation is a potential curative therapy for people with hematologic cancers, autoimmune diseases, and debilitating genetic diseases. However, the pre-transplant conditioning required to prepare patients for transplant involves highly toxic chemotherapy, which can be life-threatening and limits the number of people who are able to benefit, said Dr. Shizuru, co-founder and member of the Board of Directors of Jasper Therapeutics. JSP191 is the only anti-CD117 antibody to demonstrate safety and efficacy in severely ill patients receiving stem cell transplant in the clinic. We plan to expand clinical development to patients receiving transplants for acute myeloid leukemia/ myelodysplastic syndrome or autoimmune diseases and to patients receiving stem cell-directed gene therapies.

Dr. Shizuru added, With an experienced executive team of biotech veterans and a strong syndicate of healthcare-focused investors, Jasper Therapeutics is well positioned to achieve our vision of building a leading biotech company starting with JSP191 and expanding to other novel therapies for immune modulation, graft engineering and cell and gene therapies.

JSP191 is currently being evaluated in an ongoing Phase 1 clinical trial as a conditioning agent to enable stem cell transplantation in patients with severe combined immunodeficiency (SCID) who received a prior stem cell transplant that failed. This severe genetic immune disorder leaves patients without a functioning immune system. Interim results of the study will be presented in an oral presentation (abstract #800) on Monday, December 9, at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Fla. Clinical studies to evaluate the safety and efficacy of JSP191 as a conditioning agent in patients undergoing hematopoietic cell therapy for hematologic cancers are planned for 2020.

Founding Management Team

Dr. Shizuru and Mr. Lis are joined on the Jasper Therapeutics Board of Directors by Kurt von Emster, Managing Partner of Abingworth LLP, and Anna French, Ph.D., Principal at Qiming Venture Partners USA. Dr. Prohaska is a Board observer.

With our investment in this program, were able to realize our mission of fast-tracking stem cell treatments by helping academic researchers rapidly advance the most promising discoveries in the lab into the clinics and to drug development with commercialization partners, said Maria T. Millan, M.D., President and CEO of CIRM. Jaspers two co-founders took a novel antibody with unique properties and moved it from the bench to the bedside relatively quickly, and were thrilled to partner with this talented team to potentially impact a broad group of people who could benefit from stem cell therapy.

About Stem Cell Transplantation

Blood-forming, or hematopoietic, stem cells are cells that reside in the bone marrow and are responsible for the generation and maintenance of all blood and immune cells. These stem cells can harbor inherited or acquired abnormalities that lead to a variety of disease states, including immune deficiencies, blood disorders or hematologic cancers. Successful transplantation of hematopoietic stem cells is the only cure for most of these life-threatening conditions. Replacement of the defective or malignant hematopoietic stem cells in the patients bone marrow is currently achieved by subjecting patients to toxic doses of radiation and/or chemotherapy that cause DNA damage and lead to short- and long-term toxicities, including immune suppression and prolonged hospitalization. As a result, many patients who could benefit from a stem cell transplant are not eligible. New approaches that are effective but have minimal to no toxicity are urgently needed so more patients who could benefit from a curative stem cell transplant could receive the procedure.

Safer and more effective hematopoietic cell transplantation regimens could overcome these limitations and enable the broader application of hematopoietic cell transplants in the cure of many disorders. These disorders include hematologic cancers (e.g., myelodysplastic syndrome [MDS] and acute myeloid leukemia [AML]), autoimmune diseases (e.g., lupus, rheumatoid arthritis, multiple sclerosis and Type 1 diabetes), and genetic diseases that could be cured with genetically-corrected autologous stem cells (e.g., severe combined immunodeficiency syndrome [SCID], sickle cell disease, beta thalassemia, Fanconi anemia and other monogenic diseases).

About JSP191

JSP191 (formerly AMG191) is a first-in-class humanized monoclonal antibody in clinical development as a conditioning agent that clears hematopoietic stem cells from bone marrow. JSP191 binds to human CD117, a receptor for stem cell factor (SCF) that is expressed on the surface of hematopoietic stem and progenitor cells. The interaction of SCF and CD117 is required for stem cells to survive. JSP191 blocks SCF from binding to CD117 and disrupts critical survival signals, causing the stem cells to undergo cell death and creating an empty space in the bone marrow for donor or gene-corrected transplanted cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in an animal model of MDS. This creates the space needed for transplanted normal donor or gene-corrected hematopoietic stem cells to successfully engraft in the host bone marrow. To date, JSP191 has been evaluated in more than 80 healthy volunteers and patients. It is currently being evaluated as a sole conditioning agent in a Phase 1 dose-escalation trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for SCID, which is curable only by this type of treatment. For more information about the design of the clinical trial, visit http://www.clinicaltrials.gov (NCT02963064). Clinical development of JSP191 will be expanded to also study patients with AML or MDS who are receiving hematopoietic cell transplant.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on enabling safer conditioning and therapeutic agents that expand the application of curative hematopoietic stem cell transplants and gene therapies. Jasper Therapeutics lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a stem cell transplant. For more information, please visit us at http://www.jaspertherapeutics.com.

Read the rest here:
Jasper Therapeutics Launches with $35 Million Series A Financing to Develop and Commercialize Innovative Conditioning Agents and Therapies to...

Bone Marrow Stem Cells Comments Off on Jasper Therapeutics Launches with $35 Million Series A Financing to Develop and Commercialize Innovative Conditioning Agents and Therapies to… | December 9th, 2019

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced that new results from its CD117-ADC patient preparation program were presented at the 61st Annual Meeting of the American Society of Hematology (ASH). These results, which were highlighted in an oral presentation at ASH by John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, showed the first-ever successful transplant of gene-modified cells in non-human primates using a targeted, single-agent antibody-drug conjugate (ADC), without the use of chemotherapy or radiation.

Todays conditioning regimens involve high doses of chemotherapy, often paired with radiation, to remove the disease-causing cells. As a result, patients undergoing gene therapy or stem cell transplant are all faced with a difficult choice: whether to endure severe toxicity and risk infertility and cancer for the chance for a cure. Magentas portfolio of targeted ADCs represents an extremely promising new option to prepare patients for gene therapy or transplant with no need for toxic chemotherapy or radiation, said Dr. Tisdale. The results presented today show that a single dose of single agent CD117-ADC achieves the same level of depletion as four doses of busulfan chemotherapy to enable successful engraftment and persistence of stem cells modified with the -globin gene, the gene that causes sickle cell disease and -thalassemia when mutated. Importantly, the animals undergoing preparation with CD117-ADC showed none of the damaging toxicities associated with busulfan conditioning.

Magenta is the only company with the people, platforms and a product engine committed to comprehensively transforming immune and blood system reset, which includes revolutionizing the toxic methods that are used to prepare patients for gene therapy and transplant today. said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. The gene therapy field has learned that higher levels of stem cell depletion, which meant higher doses of busulfan, were needed to ensure long-term engraftment of the gene-modified cells and persistence of gene therapy. Across all the modalities we have tested, we have seen that ADCs are most effective at achieving these high levels of stem cell depletion without chemotherapy to enable engraftment and long-term durability of the transplant. Todays impressive results provide important validation of the ADC approach as well as the CD117 target for patient preparation and underscore Magentas leadership in the field of conditioning.

Results from the CD117-ADC Patient Preparation Program

Title: A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates (Abstract #610)Presenter: John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, Bethesda, Md.

Magentas most advanced patient preparation program, CD117-ADC, targets CD117, a protein expressed on hematopoietic stem cells. CD117-ADC is designed to remove the genetically mutated cells in the bone marrow that cause certain genetic diseases, such as sickle cell disease, enabling curative stem cell transplant or gene therapy.

Results presented by Dr. Tisdale showed:

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Registration Statement on Form S-1, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191209005253/en/

Go here to read the rest:
Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug...

Bone Marrow Stem Cells Comments Off on Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug… | December 9th, 2019