Stem Cell Clinic

Vor Biopharma Hires Senior Cell and Gene Therapy Leaders as Chief Technology Officer and Vice President of Research – Business Wire

By Stell Cell Research

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vor Biopharma, an oncology company pioneering engineered hematopoietic stem cells (eHSCs) for the treatment of cancer, today announced senior appointments to its leadership team. Sadik Kassim, PhD, a cell and gene therapy bioprocessing and translational research expert, joins Vor from Kite Pharma as Chief Technology Officer. Tirtha Chakraborty, PhD, a hematological and gene engineering research specialist with experience at Sana Biotechnology and CRISPR Therapeutics, joins as Vice President of Research. These new positions follow Vors recent move into an integrated headquarters in Cambridge, Mass., the appointment of Robert Ang, MBBS, MBA, as President and Chief Executive Officer and a $42 million Series A financing directed at developing Vors platform technology and advancing its pipeline of eHSC-based candidates.

Vor is bringing a fundamentally novel approach to hematopoietic stem cells to empower targeted cancer therapies, and we are rapidly building an industry-leading team to realize the value in this scientific foundation, said Dr. Ang. Dr. Kassim brings his substantial experience with the complex methods and processes that are required for manufacturing genetically-manipulated cell therapies, and Dr. Chakraborty provides deep expertise in hematology and genetic engineering. Their complementary knowledge will aid Vors expansion, platform development and the move towards our first Investigational New Drug filing for VOR33.

I am impressed that compelling in vivo data already supports the potential of Vors cellular engineering platform to protect healthy cells from antigen-directed therapies via antigen removal, said Dr. Kassim. This is especially noteworthy when therapeutic effectiveness is so often highly limited by co-location of target antigens on healthy immune cells, creating a huge opportunity for Vor to significantly broaden the applicability of these and future therapies.

Its exciting to join the Vor team during this period of accelerated expansion, said Dr. Chakraborty. As a geneticist and cell biologist, I look forward to developing this new approach to treat a range of devastating cancers, beginning with VOR33 in acute myeloid leukemia.

Dr. Kassim is a former Executive Director at Kite Pharma where he led the development of manufacturing processes for autologous CAR- and TCR-based gene-modified cell therapies. Prior to Kite, he served as Chief Scientific Officer at Mustang Bio, where he was the first employee and oversaw the foundational build-out of the companys preclinical and manufacturing activities. Prior to Mustang, Dr. Kassim was Head of Early Analytical Development for Novartis Cell and Gene Therapies Unit, where he contributed to the BLA and MAA filings for Kymriah. Earlier in his career, Dr. Kassim was a research biologist at the National Cancer Institute, where he was involved in early research and CMC work that led to the development of several first-in-human TCR and CAR-T products, including Kites Yescarta. Dr. Kassim has also conducted preclinical immunology research at Janssen and was a research fellow in the University of Pennsylvania Gene Therapy Program, where he led the initial discovery and preclinical studies for an AAV8 gene therapy for familial hypercholesterolemia, a program that is now in the clinic. Dr. Kassim earned his BS in Cell and Molecular Biology from Tulane University and received his PhD in Microbiology and Immunology from Louisiana State University.

Dr. Chakraborty joins Vor from Sana Biotechnology, where he served as the Vice President of Cell Therapy Research. Prior to Sana, Dr. Chakraborty was the Head of Hematology at CRISPR Therapeutics, where his teams work on hemoglobin disorders paved the way for the first clinical trial for the CRISPR industry. Before that, at Moderna Therapeutics, Dr. Chakraborty led synthetic mRNA platform technology research. He was trained as an RNA biologist and an immunologist during his postdoctoral research at Harvard Medical School. Dr. Chakraborty received his PhD from the Tata Institute of Fundamental Research in Mumbai, India.

About VOR33Vors lead engineered hematopoietic stem cell (eHSC) product candidate, VOR33, is in development for acute myeloid leukemia (AML). VOR33 is designed to produce healthy cells that lack the receptor CD33, thus enabling the targeting of AML cells through the CD33 antigen, while avoiding toxicity to the bone marrow. Currently, targeted therapies for AML and other liquid tumors can be limited by on-target toxicity. By rendering healthy cells invisible to CD33-targeted therapies, VOR33 aims to significantly improve the therapeutic window, utility and effectiveness of these AML therapies, with the potential to broaden clinical benefit to different patient populations.

About Vor BiopharmaVor Biopharma aims to transform the lives of cancer patients by pioneering engineered hematopoietic stem cell (eHSC) therapies. Vors eHSCs are designed to generate healthy, fully functional cells with specific advantageous modifications, protecting healthy cells from the toxic effects of antigen-targeted therapies, while leaving tumor cells vulnerable.

Vors platform could potentially be used to change the treatment paradigm of both hematopoietic stem cell transplants and antigen-targeted therapies, such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vors lead program has been published in Proceedings of the National Academy of Sciences.

Vor is based in Cambridge, Mass. and has a broad intellectual property base, including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.

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Vor Biopharma Hires Senior Cell and Gene Therapy Leaders as Chief Technology Officer and Vice President of Research - Business Wire

What’s in the cards for this year’s Nobel Prizes? – STAT

By Embryonic Stem Cells

Were not saying that discovering molecular drivers of cancer or cancer-causing genes doesnt deserve the Nobel Prize in medicine or physiology. But for Dr. Brian Druker (whose work led to the targeted leukemia drug Gleevec), Dr. Dennis Slamon (Herceptin), and Mary-Claire King (the BRCA breast- and ovarian-cancer gene), 2019 is probably not their year to be summoned to Stockholm: The 2018 medicine Nobel honored immuno-oncology, and according to STATs Nobel crystal ball, cancer wont win two years in a row.

With the naming of the science Nobels fast approaching the medicine prize will be announced on Oct. 7, physics on Oct. 8, chemistry on Oct. 9 polls, betting pools, and number crunching are in full swing, using approaches from toting up how many predictor prizes a scientist has won to calculating the periodicity of awards, meaning how many years pass before a specific subfield is honored again.

Past laureates, who get to submit nominations every year after their own anointing, have their favorites and, sometimes, their hunches, wrong though they usually are. As 2018 chemistry winner Frances Arnold of the California Institute of Technology said, Its not helpful to second-guess these things!

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Indeed, the Nobels are known for some head-scratching choices over the decades, though less in the science prizes than in peace and literature. Nevertheless, some experts have developed systems that do pretty well. Since 2002, David Pendlebury of Clarivate Analytics has made 50 correct predictions (though usually not in the right year) by analyzing how often a scientists key papers are cited by peers.

Using that strategy, Pendlebury thinks the chemistry Nobel could go toinventors of DNA sequencing techniques: Marvin Caruthers of the University of Colorado, Leroy Hood of the Institute for Systems Biology, and Michael Hunkapiller, CEO of DNA sequencing goliath Pacific Biosciences.Without their [1980s] inventions, Pendlebury said, there would be no map of the human genome.More on that below.

In medicine, Pendlebury likes the chances of Hans Clevers of the Netherlands Utrecht University for research on the Wnt signaling pathway. Wnt controls how stem cells differentiate and how some cancers develop. If Wnt wins, then the private biotech Samumed (valuation: $12 billion) can say its investigational Wnt-targeting drugs are based on Nobel-winning research.

Pendlebury also has his eyes on John Kappler and Philippa Marrack of National Jewish Health in Denver for discovering T-cell tolerance, a mechanism by which the thymus eliminates T cells that would attack the self. That advanced understanding of autoimmune diseases such as rheumatoid arthritis and lupus, and is so basic and important one wonders, Why no Nobel yet?

In 2019, the answer might be, because the 2018 prize honored the hybrid of immunology and cancer, so immunology might have to wait another decade for its next turn. Thats sad news for Jacques Miller, who has never received a Nobel for discovering the function of the thymus and the fact that immune cells include T and B cells in the 1960s. Look how many Nobels were given for immunology based on Millers work, said MITs Phillip Sharp, who shared the 1993 medicine Nobel. There is just a lot of good science that will never get recognized.

If immunology and cancer are off the table, it opens a lane for optogenetics, the revolutionary mashup of genetic engineering and neuroscience. Here, Pendlebury likes Ernst Bamberg of the Max Planck Institute of Biophysics in Germany, Karl Deisseroth of Stanford University, and Gero Miesenbck of the University of Oxford in the U.K. Honoring optogenetics would be wonderful on many levels, starting with the power of the technique to identify neural circuits involved in virtually any function and disease. (The Nobel committees like techniques that enable others to make cool discoveries, Sharp said.)

Even more fun, a prize for optogenetics could open the floodgates of controversy. The key discoveries have so many fathers (alas, optogenetics has no mothers) that picking any three, the maximum for a Nobel, would likely trigger weeks (maybe years!) of carping about who got left out. As it happens, on Thursday the $500,000 Warren Alpert Prize will honor optogenetics, but the winners are Deisseroth, Miesenbck, MITs Edward Boyden (Deisseroths former postdoc), and Peter Hegemann of Germanys Humboldt University. If science had cage matches, we could root for one between Bamberg, Boyden, and Hegemann for the third slot in an optogenetics Nobel.

Records of Nobel committee deliberations remain secret for 50 years, so its anyones guess if theyre influenced by other big prizes. But the latter do seem to have predictive value. Thats one reason David Allis of Rockefeller University and Michael Grunstein of Hebrew University, who in the 1990s discovered one way genes are activated and quieted (through proteins called histones), are favorites (again) for a medicine Nobel. Theyve shared a slew of awards, including a 2018 Lasker and a 2016 Gruber Prize in Genetics, so 2019 could (finally) be their year, especially since there hasnt been a Nobel for gene expression since 2006, and the Allis/Grunstein discovery basically launched the hot field of epigenetics.

Sharp points to another scientist who made seminal contributions to the understanding of genes off/on switches: Yale Universitys Joan Steitz, who in 1979 discovered small noncoding RNAs that control gene expression.

For his crystal ball, biologist Jason Sheltzer of Cold Spring Harbor Laboratory looks at periodicity, a strategy that helped him correctly predict last years medicine win for immune-oncology pioneer James Allison. Fields like infectious disease, immunology, and cancer win every 10 to 20 years, and so are probably off the table for 2019. But the last Nobel for DNA sequencing was way back in 1980, Sheltzer points out, and since then we have seen the complete sequencing of the human genome, one of humanitys towering achievements.

The problem is, hundreds of scientists worked on the Human Genome Project. Last year the Nobels honored the organizers of the project that discovered gravitational waves, so with similar reasoning the medicine or chemistry prize might go to Dr. Francis Collins, now director of the National Institutes of Health and the guy who herded all those cats to get the genome project done, and/or to the Broad Institutes Eric Lander, whose lab churned out much of the sequence. A third might be Craig Venter, the outsider whose private sequencing efforts raced the Collins/Lander government project to a bitter draw.

But if this trio wins, Pendleburys DNA-sequencing three (Caruthers, Hood, and Hunkapiller) wont. To complicate things even further, among the leading vote getters in an online poll for the chemistry Nobel is Shankar Balasubramanian of Cambridge University, who helped developed next-generation DNA sequencing. The Nobel committees grapple with questions of credit all the time: who did it, who did it first, who had the greatest impact, said chemist Peter Dorhout, past president of the American Chemical Society. I wouldnt want to be them.

For that and other messy reasons, when asked if sequencing has a shot at a Nobel, Sharp didnt hesitate: No, he told STAT.

Gene editing, on the other hand, last won in 1993, so it could be time for a new editing prize, Sheltzer tweeted. CRISPR in particular, [Jennifer] Doudna [of the University of California, Berkeley] will win for either chemistry or medicine.

The wisdom of the crowd agrees with him. Sigma Xi, the scientific research honor society, has been asking members to vote for most likely laureates in bracketology-style matchups. Doudna made the final four in chemistry. (Sigma Xi will announce the winner of its contest Thursday.) The other three finalists: Harvards Stuart Schreiber for research on signal transduction and master regulators of cell function such as the gene mTOR, now a hot target for cancer and other drugs; John Goodenough of the University of Texas for inventing lithium-ion batteries, and Stanfords Carolyn Bertozzi for basically developing bio-orthogonal chemistry, in which reporter molecules label biomolecules within cells.

Since STAT covers only life sciences, we have nothing to say about Goodenoughs chances (but thank you for making smartphones, digital cameras, and Teslas possible!), and agree that Schreiber and Bertozzi are stars. But the Doudna pick is a minefield. If she wins it alone, there will be hurt egos galore, starting with her collaborator Emmanuelle Charpentier of the Max Planck Institute for Infection Biology and including Virginijus iknys of Vilnius University. iknys is often called the forgotten man of CRISPR because Doudna and Charpentier had been running the table of prizes for their work turning a bacterial immune system into a DNA editor until he shared last years $1 million Kavli Prize in nanoscience.

If the Nobel committees prefer to keep firestorms of controversy for the peace prize (Greta Thunberg or Donald Trump?), they have many safe but stellar choices. Pierre Chambon and Ronald Evans have never won for discovering nuclear hormone receptors, where molecules as different as steroids and vitamins dock to make a whole suite of physiological reactions happen, Sharp points out. He also thinks Victor Ambros of the University of Massachusetts and Harvards Gary Ruvkun could bounce up this year for discovering microRNAs DNA-regulating molecules that turn out to control embryonic development, cancer, cell differentiation, and more. The pair has also snared a pile of predictor awards, including a Lasker, a Gairdner, a Breakthrough, and a Gruber Genetics Prize.

To all the many scientists whose work deserves a Nobel but who will not get an early-morning call from Stockholm next week, Sharp offers this quasi-consolation: Life is not fair.

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What's in the cards for this year's Nobel Prizes? - STAT

Skin-Cells-Turned-to-Heart-Cells Help Unravel Genetic Underpinnings of Cardiac Function – UC San Diego Health

By Stem Cell Medical Center

Genome-wide association studies have uncovered more than 500 genetic variants linked to heart function, everything from heart rate to irregular rhythms that can lead to stroke, heart failure or other complications. But since most of these variations fall into areas of the genome that dont encode proteins, exactly how they influence heart function has remained unclear.

By examining heart cells derived from the skin samples of seven family members, researchers at University of California San Diego School of Medicine have now discovered that many of these genetic variations influence heart function because they affect the binding of a protein called NKX2-5.

The study is published September 30, 2019 in Nature Genetics.

By examining heart cells derived from the skin samples of seven family members, researchers at UC San Diego School of Medicine discovered that many genetic variations known to influence heart function do so because they affect the binding of a protein called NKX2-5.

NKX2-5 is a transcription factor, meaning it helps turn on and off genes in this case, genes involved in heart development. To do this, NKX2-5 must bind to non-coding regions of the genome. Thats where genetic variation comes in.

NKX2-5 binds to many different places in the genome near heart genes, so it makes sense that variation in the factor itself or the DNA to which it binds would affect that function, said senior author Kelly A. Frazer, PhD, professor of pediatrics and director of the Institute for Genomic Medicine at UC San Diego School of Medicine. As a result, we are finding that multiple heart-related traits can share a common mechanism in this case, differential binding of NKX2-5 due to DNA variants.

The study started with skin samples from seven people from three generations of a single family. The researchers converted the skin cells into induced pluripotent stem cells (iPSCs) as an intermediary. Like all stem cells, iPSCs can both self-renew, making more iPSCs, and differentiate into a specialized cell type. With the right cocktail of molecules and growth factors, the researchers directed iPSCs into becoming heart cells.

These heart cells actually beat in the laboratory dish, and still bear the genetic and molecular features of the individuals from which they were derived.

Frazer and team conducted a genome-wide analysis of these patient-derived heart cells. They determined that NKX2-5 can bind approximately 38,000 sites in the genome. Of those, 1,941 genetic variants affected NKX2-5 binding. The researchers investigated the role of those variants in heart gene function and heart-related traits. One of the genetic variants was associated with the SCN5A gene, which encodes the main channel through which sodium is transported in heart cells.

Since related individuals tend to share similar genetic variants, the team was able to validate their findings by analyzing the same variants in multiple samples.

People typically need a large number of samples to detect the effects of common DNA variants, so we were surprised that we were able to identify with high confidence these effects on NKX2-5 binding at so many sites across the genome with just few people, said first author Paola Benaglio, PhD, a postdoctoral researcher in Frazers lab.

Yet, she said, this finding may just be the tip of the iceberg.

There are probably a lot more genetic variants in the genome involved with NKX2-5 as well as with other important cardiac transcription factors, Frazer said. We identified almost 2,000 in this study, but thats probably only a fraction of what really exists because we were only looking at seven people in a single family and only at one transcription factor. There are probably many more variants in gene regulation sites across the entire population.

Not only does the team plan to further investigate cardiovascular genetics, they also have their sights set on other organ systems.

We are now expanding this same model system to look at many different transcription factors, across different tissue types, such as pancreas and retina epithelia, and scaling it up to include more families, Benaglio said.

Co-authors include: Agnieszka DAntonio-Chronowska, William W. Young Greenwald, Margaret K. R. Donovan, Christopher DeBoever, He Li, Frauke Drees, Sanghamitra Singhal, Hiroko Matsui, Kyle J. Gaulton, Erin N. Smith, Matteo DAntonio, Michael G. Rosenfeld, UC San Diego; Wubin Ma, Feng Yang, Howard Hughes Medical Institute and UC San Diego; Jessica van Setten, University Medical Center Utrecht; and Nona Sotoodehnia, University of Washington.

This research was funded, in part, by the National Institutes of Health (grants HG008118, HL107442, F31HL142151, T32GM008666, P30CA023100, HL116747, HL141989, R01DK114650, DK018477, DK039949), National Science Foundation (grant 1728497), California Institute for Regenerative Medicine (grants GC1R-06673-B, TG2-01154), Swiss National Science Foundation (postdoc mobility fellowships P2LAP3-155105,P300PA-167612), ADA (grant 1-17-JDF-027) and Howard Hughes Medical Institute.

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Skin-Cells-Turned-to-Heart-Cells Help Unravel Genetic Underpinnings of Cardiac Function - UC San Diego Health

AIVITA Biomedical to Present at Meeting on the Mesa and Other Investor, Oncology and Regenerative Medicine Conferences in October – PRNewswire

By Stem Cell Medical Center

IRVINE, Calif., Oct. 1, 2019 /PRNewswire/ -- AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, today announced that it will be presenting at the following investor, oncology and regenerative medicine conferences in October:

Cell & Gene Meeting on the Mesa Company Presentation Time: October 2, 11:00 AM PST Location: Park Hyatt Aviara Resort Golf Club & Spa, Cognate in the Bioservices Ballroom, Carlsbad, CA

World Immunotherapy Congress Keynote Title: Enhancing patient responses with autologous cancer stem cell vaccine in combination with checkpoint inhibitors Time: October 16, 9:25 AM CET Location: Congress Centre Basel, Basel, Switzerland

BIO Investor Forum Company Presentation Time: October 23, 10:15 AM PST Location: Elizabethan A, The Westin St. Francis,San Francisco, CA

World Vaccine Congress Talk Title:Autologous Cancer Stem Cell Vaccines for Solid Tumor Cancers When: October 29, 3:45 PM CET Location: Palau de Congressos de Catalunya, Barcelona, Spain

About AIVITA Biomedical AIVITA Biomedical is a privately held company engaged in the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. Founded in 2016 by pioneers in the stem cell industry, AIVITA Biomedical utilizes its expertise in stem cell growth and directed, high-purity differentiation to enable safe, efficient and economical manufacturing systems which support its therapeutic pipeline and commercial line of skin care products. All proceeds from the sale of AIVITA's skin care products support the treatment of women with ovarian cancer.

SOURCE AIVITA Biomedical, Inc.

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AIVITA Biomedical to Present at Meeting on the Mesa and Other Investor, Oncology and Regenerative Medicine Conferences in October - PRNewswire

3 Houston innovators to know this week – InnovationMap

By Stem Cell Medical Center

A new Houston-based startup accelerator is planning to advance companies focusing on regenerative medicine and stem cell treatment.

Houston Healthspan Innovation Group was created by founder and CEO Ed Bosarge, a local entrepreneur who's made millions of developing health care and finance technology.

"From day one, Houston Healthspan will play a significant role in shaping Houston's vibrant life sciences scene with its seasoned leadership and state-of-the-art facilities," Bosarge says in a news release. "Houston Healthspan may be a tipping point for the region's life sciences community."

The program will provide its participating startups and joint venture partners with expertise and resources in biology, clinical disease, therapeutic delivery systems, finance, and marketing, per the release.

The accelerator will be housed out of the Houston Healthspan Bio Labs 10,000 square feet of lab space just south of the Texas Medical Center. The labs will provide the scientists and researchers with cutting-edge technologies, large cleanrooms, and cGMP cell culture workstations will be used for cell manufacturing, bioprocessing, and therapeutic protocol development. The lab can even handle small-scale biologics manufacturing.

"Gaining access to lab space is a significant hurdle many start-up life sciences companies must overcome," says Dr. Steven Greco, chief science officer at Houston Healthspan. "Our Bio Labs address this need and offer a compelling and ideal setting for start-ups and joint-venture partners to conduct pre-clinical studies and obtain valuable research services."

Houston Healthspan has already started working with two regenerative medicine companies that have both relocated their operations to Houston. Rejenevie Therapeutics, which moved from New Jersey, develops therapies for immune system restoration as well as age-related illnesses. Formerly based in Hawaii, Tissue Genesis created the Icellator X, a technology that focuses on stem cell isolation.

"With two collaborator companies like Rejenevie and Tissue Genesis working out of our Houston Healthspan Bio Labs, we can offer significant resources and expertise for start-up and joint-venture partners to thrive and succeed," says Eric Schaeffer, chief strategy officer, in the release.

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3 Houston innovators to know this week - InnovationMap

R3 Stem Cell Launches Master Class Series on Stem Cell Therapy and Regenerative Medicine – PRNewswire

By Stem Cell Treatment

PHOENIX, Oct. 1, 2019 /PRNewswire/ --R3 Stem Cell, a national leader in stem cell therapy marketing and education, today announced that they are launching a new Master Class centered around the topic of stem cells and regenerative procedures. To commemorate their launch, they will be giving away one out of the eight episodes of their Master Class series for free and the rest will be available for only $49. Purchase the R3 Stem Cell Master Class series and receive Stem Cell Therapy with an R3 Center for $500 off. Register for the Master Class at https://stemcellmasterclass.org/.

The Master Class offers those considering regenerative procedures with stem cells vital information such as discovering how the process works, understanding the options that are given to them, learning the research behind it and gaining realistic expectations.

"Experiencing chronic pain is tiring and can lead to anxiety, depression, obesity, and addiction of medication," said David Greene, MD, MBA, Founder and CEO of R3 Stem Cell. "For those who are considering a stem cell procedure, our Master Class will provide them with the knowledge needed in order to make the decision. Questions regarding common misconceptions in the ever-growing field will be uncovered, giving the patient a realistic understanding of what a stem cell is, where they come from and what they can do."

The Master Class series explains what to expect with a regenerative procedure investment, what the different ways are to optimize stem cell therapy outcomes, what to look for in a stem cell clinic, and a patient's real-life experience with a stem cell procedure. The series is divided into eight different episodes, offering the patient a four-hour long learning opportunity with information on discovering a procedure that is right for them.

"It is important for patients to fully understand what type of stem cell therapies are being offered to them and know the full effect it can have on their everyday lives," continues Dr. Greene. "Regenerative therapies may give patients the opportunity to return to pain-free personal everyday activities. Our purpose is to make people educated consumers in a time when so much misinformation is being disseminated."

About R3 Stem Cell

R3 Stem Cell offers regenerative stem cell therapies to those who suffer from chronic pain. Their sole purpose is to repair, regenerate, and restore damaged tissue from the body. Giving hope and options for patients of relieving chronic pain and avoiding surgery. Stem cell procedures are done using bone marrow, adipose, amniotic, PRP or umbilical cord tissue containing platelets, cytokines, growth factors and exosomes that work to start a healing process within the body and repair damaged tissue. R3 Stem Cell has over 35 nationwide Centers and there is most likely a clinic near you. To learn more about R3 Stem Cell, visit their website at http://www.r3stemcell.comor call (844) GET-STEM.

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R3 Stem Cell Launches Master Class Series on Stem Cell Therapy and Regenerative Medicine - PRNewswire

Michael Schumachers surgeon denies hes performing experiments on F1 legend with stem cell treatment and sl – The Sun

By Stem Cell Treatment

MICHAEL Schumacher's doctor has warned fans that he "does not work miracles" following stem cell therapy he performed on the F1 star.

Dr Philippe Menasch has also slammed claims he was carrying out "experiments" on the legendary racing driver when he treated him in Paris last month.

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Little was known about the stem-cell operation, but seven-time world champion Schumacher is believed to have received transfusions of inflammation-reducing stem cells.

Amongst the media flurry surrounding the top-secret op, local media reported on it as "experimental."

Menasche - known as a pioneer in stem-cell research - has slammed these claims, telling Italian newspaper La Republica: "I do not perform miracles.

"My team and I are not doing an experiment, an abominable term that is not in line with a serious medical view."

Schumacher's condition is not public knowledge, but after the procedure last month, a nurse toldLe Parisien: "Yes he is in my service ... And I can assure you that he is conscious."

The French paper also said Schumacher has been treated at least twice previously at the Georges-Pompidou hospital in Paris, admitted each time under a false name and treated by a small medical team.

It was also reported that the F1 driver was accompanied by security staff.

Schumacher's health has been shrouded in secrecy ever since a near-fatal brain injury he suffered in 2013.

The seven time F1 championsuffered severe head injuries on a family skiing holiday in the French Alps and has not been seen in public since.

What is stem cell therapy and how does it work?

STEM cell therapy is one of the most promising new medical treatments.

Stem cells are the body's raw materials - the cells from which all cells in the body are generated.

In the lab, scientists can take stem cells and help them divide to create daughter cells.

These daughter cells can either become new stem cells or turn into specialised cells - blood, brain, heart muscle, bone cells for example.

Scientists and doctors across the world hope stem cells could prove the breakthrough for treating a range of conditions, from heart disease to cancer.

How does it work?

One of the key ways stem cell treatment can work is to repair or regenerate damaged and diseased tissues.

By taking the cells and creating specialised heart muscle cells for example, doctors can help repair damaged heart muscle and use them to treat heart failure.

Who can benefit?

According to the Mayo Clinic in the US, patients with a wide range of illnesses and disease could benefit from the treatment.

They include those suffering:

Stem cells can also be used to grow new tissue that can then be used for transplants and regenerative medicine.

It means in future, stem cells could be used to grow new organs rather than relying on organ donation.

He was skiing with his son Mick when he fell and cracked his head on a boulder on the Combe de Saulire above Mribel.

The devastating injury left him paralysed and unable to speak.

Schumacher spent three months in a medically-induced coma after the accident and has had years of intensive care at his house in Gland, a Swiss town on the shore of Lake Geneva.

His condition now appears to have stabilised and in January this year he was taken by helicopter to the family's holiday home in Mallorca for his 50th birthday.

In a celebratory post on Instagram, his family wrote: "Please understand if we are following Michael's wishes and keeping such a sensitive subject as health, as it has always been, in privacy."

They confirmed that Schumacher was in "the very best of hands."

The wall of secrecy is reportedly enforced by his wife, Corinna.

Forumla One's head of motorsport, Ross Brawn has spoken publicly about Corinna's decision to keep her husband's health private.

He said: "I am constantly in touch with Corinna, and I totally agree with their decision."

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"Michael has always been a very private person and that's been a guiding principle in his career, his life and his family always agreed with that choice.

"It's completely understandable that Corinna has wanted to maintain the same approach, even after the tragic event, and it's a decision we must all respect.

"I'm sure the millions of people who are still Michael fans will understand it, too."

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Michael Schumachers surgeon denies hes performing experiments on F1 legend with stem cell treatment and sl - The Sun

Regenerative Stem Cell Therapy Pioneer, Celavie Biosciences, LLC, to Attend Cell & Gene Meeting on the Mesa on October 2 4 in Carlsbad,…

By Stem Cell Treatment

OXNARD, Calif.--(BUSINESS WIRE)--Celavie Biosciences, LLC, a company working to improve lives and restore hope by advancing innovations in CNS diseases with regenerative stem cell-based therapies, announced today their attendance at the Alliance of Regenerative Medicines (ARM) Cell & Gene Meeting on the Mesa to be held October 2nd 4th in Carlsbad, California.

Sandy Solmon, CEO and President, and Oleg Kopyov, MD, PhD, Executive Vice President and Chief Scientific Officer, will attend the conference and take meetings to explore collaboration and funding opportunities for further development of Celavies human and veterinary clinical programs.

At partnering meetings, Celavie will discuss their lead clinical program for the application of the companys human undifferentiated allogeneic pluripotent stem cells in Parkinsons disease (PD). Celavie recently published exploratory clinical data in 7 PD patients with four-year follow-up in Cell Transplantation. Celavie anticipates filing an IND with the FDA for a Phase I clinical trial in patients with moderate to advanced PD.

Celavie will also discuss their pre-clinical program in cerebellar ataxia and plans for taking their programs into further clinical development. Additionally, Celavie is actively seeking opportunities to out-license subsidiary Celavets veterinary applications in equine tendonitis and other indications.

To schedule a meeting with Celavie Biosciences at the Cell & Gene Meeting on the Mesa, please contact Sandy Solmon or Dr. Oleg Kopyov via the Cell & Gene Meeting on the Mesa partnering portal: https://www.meetingonthemesa.com/partnering/.

About the Cell & Gene Meeting on the Mesa

The Cell & Gene Meeting on the Mesa is the sector's foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures. Tackling the commercialization hurdles facing the cell and gene therapy sector today, this meeting covers a wide range of topics from clinical trial design to alternative payment models to scale-up and supply chain platforms for advanced therapies. The program features expert-led panels, extensive partnering capabilities, exclusive networking opportunities, and 70+ dedicated presentations by the leading publicly traded and privately held companies in the space. Attracting over 1,150 attendees over 20% of which are C-level executives this conference enables key partnerships through more than 2,200 one-on-one meetings while highlighting the significant clinical and commercial progress in the field. For more information, visit the website: https://www.meetingonthemesa.com/.

About Celavie Biosciences

Celavie Biosciences is a privately-held company whose mission is to improve lives and restore hope by advancing regenerative stem cell therapies for the treatment of Parkinsons disease and other disorders of the central nervous system (CNS). The company develops undifferentiated, unmodified allogeneic pluripotent stem cell-based therapies, holds a strong IP portfolio, including 18 issued patents, and has an experienced management team blending expertise in concept and cell technology, product scalability and entrepreneurship. Celavet, a subsidiary, applies the same proprietary technologies for the treatment and prevention of serious veterinary diseases. More information is available at https://www.celavie.com/.

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Regenerative Stem Cell Therapy Pioneer, Celavie Biosciences, LLC, to Attend Cell & Gene Meeting on the Mesa on October 2 4 in Carlsbad,...

ElevateBio Launches HighPassBio to Advance Novel Targeted T Cell Immunotherapies with Technology from Fred Hutchinson Cancer Research Center -…

By Stem Cell Treatment

CAMBRIDGE, Mass.--(BUSINESS WIRE)--ElevateBio, a Cambridge-based biotechnology holding company, today announced it has established and launched HighPassBio, a company dedicated to advancing novel targeted T cell immunotherapies. The companys lead product is an engineered T cell receptor (TCR) T cell therapy for HA-1 expressing tumors, which is designed to treat and potentially prevent relapse of leukemia following hematopoietic stem cell transplant (HSCT). The product and approach were developed by researchers at Fred Hutchinson Cancer Research Center.

At ElevateBio we are committed to building therapeutics companies with the worlds leading innovators in cell and gene therapy to advance novel treatments that have strong potential to dramatically improve patient lives, said David Hallal, Chairman and CEO of ElevateBio. We look forward to leveraging our centralized industry-leading cell and gene therapy process development and manufacturing capabilities while working closely with Dr. Marie Bleakley and her team, to accelerate their impressive work through clinical development with the goal of serving patients who have no other treatment options. Additionally, we will explore this approach as a potential treatment for other diseases that are treated by stem cell transplants.

HighPassBios scientific founder is Marie Bleakley, M.D., Ph.D., M.ClinEpi, pediatric oncologist and stem cell transplant physician at Fred Hutchinson Cancer Research Center and chair of the scientific advisory board (SAB) of HighPassBio. As the chair of HighPassBios SAB, Dr. Bleakley will continue working with ElevateBio to advance and accelerate this innovative program through clinical development toward patients.

A Phase 1 clinical trial has treated initial patients and is recruiting adult and pediatric patients who have relapsed with leukemia or related conditions following blood and marrow transplantation (also known as stem cell transplantation). More details on http://www.clinicaltrials.gov under the study ID number NCT03326921.

About TCR-Engineered T Cell Therapy

A key role of the immune system is to detect tumor antigens, engage T cells and eradicate the tumor. However, the immune response to tumor antigens varies and is often insufficient to prevent tumor growth and relapse. An approach known as adoptive T cell therapy, using T cell receptors, or TCRs, can overcome some of the obstacles to establishing an effective immune response to fight off the target tumor. TCRs are molecules found on surface of T cells that can recognize tumor antigens that are degraded to small protein fragments inside tumor cells. Unlike CAR T cells that recognize only surface antigens, TCRs can recognize small protein fragments derived from intracellular and surface antigens offering a more diverse way to attack tumors. These small protein fragments show up on the tumor cell surface with another protein called major histocompatibility complex (MHC), get recognized by the TCRs and consequently signal the bodys immune system to respond to fight off and kill the tumor cells.

Tumor-specific TCRs can be identified and then engineered into T cells that recognize and attack various types of cancers, representing a novel approach to treating and potentially preventing disease.

Adoptive T cell therapy can be applied to tackling relapse of leukemia post hematopoietic stem cell transplant (HSCT) by targeting the antigens expressed only by the patient and not by the stem cell transplant donor, known as minor histocompatibility antigens, or HA1. HA1 is expressed predominantly or exclusively on hematopoietic cells and leukemic cells. There is evidence that T cells specific for HA1 can induce a potent and selective antileukemic effect. HA1 TCR T cell therapy is a new investigational immunotherapy for the management of post transplantation leukemia relapse.

About Leukemia post HSCT Treatment and the Risk of Relapse

Leukemia, a cancer of the blood or bone marrow characterized by an abnormal proliferation of blood cells, is the tenth most common type of cancer in the U.S. with an estimated 60,140 new cases and 24,400 deaths in 2016. Leukemia arises from uncontrolled proliferation of a specific type of hematopoietic (blood) cell that is critical for a functional immune system. As a result, when patients are given very high doses of chemotherapy to eradicate leukemic cells most normal cells are killed as well, necessitating a transplant of hematopoietic stem cells from a donor to reconstitute the patients bone marrow and circulating hematopoietic cells. In some cases, the transplanted T cells from the donor can also recognize and eliminate the hematopoietic cells, including leukemia, from the recipient, thus preventing relapse.

While the majority of HSCT recipients are cured, 25-50 percent of HSCT recipients relapse, leukemia relapse remains the major cause of allogeneic HSCT failure, and the prognosis for patients with post-HCT relapse is poor. Relapse occurs following allogeneic HSCT in approximately one-third of patients with acute leukemia who undergo the procedure, and most patients subsequently die of their disease.

About HighPassBio

HighPassBio, an ElevateBio portfolio company, is working to advance a novel approach to treating hematological malignancies by leveraging T cell receptor (TCR)-engineered T cells, known as TCR T cells. The companys lead program is designed to treat or potentially prevent relapse of leukemia in patients who have undergone hematopoietic stem cell transplant (HSCT). The technology was born out of Fred Hutchinson Cancer Research Center by world renowned expert, Dr. Marie Bleakley.

About ElevateBio

ElevateBio, LLC, is a Cambridge-based cell and gene therapy holding company, established to create and operate a broad portfolio of cell and gene therapy companies with leading academic researchers, medical centers and entrepreneurs. ElevateBio builds single- and multi-product companies by providing scientific founders with fully integrated bench-to-bedside capabilities including world-class scientists, manufacturing facilities, drug developers and commercial expertise. ElevateBio BaseCamp, a company-owned Cell and Gene Therapy Center of Innovation, will serve as the R&D, process development and manufacturing hub across the entire ElevateBio portfolio while also supporting selected strategic partners.

ElevateBios lead investors are the UBS Oncology Impact Fund (OIF) managed by MPM Capital, as well as F2 Ventures. Investors also include EcoR1 Capital, Redmile Group, and Samsara BioCapital.

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ElevateBio Launches HighPassBio to Advance Novel Targeted T Cell Immunotherapies with Technology from Fred Hutchinson Cancer Research Center -...

Positive Study Results of Phase IIa Clinical Trial Using Intravenous Administration of Mesenchymal Stem Cells for Ischemic Stroke Published in…

By Stem Cell Treatment

SAN DIEGO, Sept. 30, 2019 /PRNewswire/ -- Results from a study sponsored by Stemedica Cell Technologies, Inc., a global biotechnology company that uses allogeneic stem cells for ischemic conditions, form the basis for a peer-reviewed paper published inStrokeentitled "Phase I/II Study of Safety and Preliminary Efficacy of Intravenous Allogeneic Mesenchymal Stem Cells in Chronic Stroke." Co-authors include Michael L. Levy, MD, PhD, John R. Crawford, MD, Nabil Dib, MD, Lev Verkh, PhD, Nikolai Tankovich, MD, PhD and Steven C. Cramer, MD.

As indicated in theStrokepublication, "Stroke is perennially among the leading causes of human disability and the leading neurological cause of lost disability-adjusted life years. The mean survival after stroke is 6-7 years, with more than 85% of patients living past the first-year post-stroke, many with years of enduring disability. Many restorative therapies are under study to improve outcomes after stroke." However, restorative therapies often have a short time window for improvement usually measured in days-months.

"Based on Stemedica's preclinical data that supported the safety and efficacy of its MSCs as a restorative therapy to improve outcomes after stroke, the company was granted approval by the FDA to conduct a Phase I/IIa dose escalation trial that examined the effects of a single IV infusion of Stemedica's cGMP manufactured allogeneic ischemia-tolerant MSCs," said Dr. Lev Verkh, Chief Regulatory and Clinical Development Officer of Stemedica.

Study:The target population included patients with chronic ischemic stroke and substantial functional deficits; a group for whom treatment options remained limited. The primary outcome of the study was safety, based on serial measures of behavior, CT scans, and laboratory testing. Four secondary endpoints were scored serially to derive estimates of behavioral changes relatively to the baseline over a period of 12 months: NIH Stroke Scale (NIHSS) for neurological assessment, Barthel Index (BI) for ability to perform daily tasks, Mini-Mental Status Exam (MMSE) for mental status, and Geriatric Depression Scale (GDS) for degree of depression. The study was conducted at three centers: University of California, San Diego (UCSD); Mercy Gilbert Medical Center, Gilbert, Arizona; and University of California, Irvine (UCI).

Entry criteria included ischemic stroke >6 months prior to administration, substantial functional deficits (subject confined to a wheelchair, had home-nursingcare, orneeded assistance withactivities ofdailyliving), no substantial improvement in neurologic orfunctional deficits for the2 months prior to enrollment in thestudypermedical history, and NIHSS score=6-20.

Enrollees received a single intravenous dose of allogeneic mesenchymal bone marrow cells. Phase I used a dose escalation design (3 tiers, n=5 each). Phase IIa (n=21) was an expanded safety cohort. The primary endpoint was safety over 1-year. Secondary endpoints examined behavior, with a pre-specified focus at 6-months.

Subject status at enrollment prior to treatment:At baseline, subjects (n=36) averaged 4.24.6 years post-stroke, age 61.110.8 years, NIHSS score 8 [6.5-10], and Barthel Index 6529.

Safety:Study testing disclosed no safety concerns. No subject showed a positive reaction to intradermal testing. In Phase I, each dose (0.5, 1.0, and 1.5 million cells/kg body weight) was found safe, as a result Phase IIa subjects received 1.5 million cells/kg. Two subjects were lost to follow-up, one was withdrawn, and two died (unrelated to study treatment). There were 15 serious adverse events, none possibly or probably related to study treatment. Two mild adverse events were possibly related to study treatment, a urinary tract infection and IV site irritation. Treatment was determined to be safe based on serial exams, EKGs, laboratory tests, and pan-CT scans.

Behavioral Effects:Improvements across all subjects post-transfusion and for all four secondary endpoints were achieved. Improvements in each index were: Barthel Index (6.811.4 points, p=0.002); in NIHSS (-1.251.7 points, p<0.001); Mini Mental Status Exam (1.82.8 points, p<0.001); and Geriatric Depression Scale (-1.63.8 points, p=0.015). At baseline 11.4% (4/35 subjects) had Barthel Index=95-100 (favorable outcome); at 6-months, 27.3% (9/33); by 12-months, 35.5% (11/31).

Conclusions:The current study is the largest trial of intravenous MSCs in patients with chronic stroke and the first to evaluate allogeneic MSC therapy in this population. It is also the first study to evaluate MSCs grown under hypoxic conditions favorable to cell proliferation, gene expression, cytokine production and migration. While patients with stroke in the chronic stage generally show significant functional decline, enrollees in the current study showed 12 months of continued functional improvements across all secondary endpoints.

Intravenous transfusion of allogeneic ischemia tolerant MSCs in patients with chronic stroke and substantial functional deficits was safe and suggested behavioral gains. These data support proceeding to a randomized, placebo-controlled study of this therapy in this population.

Dr. Nikolai Tankovich, President and Chief Medical Officer added, "Stemedica is encouraged by the results of the study which demonstrated safety and preliminary efficacy of its cell therapy product for the treatment of chronic ischemic stroke patients. It is a significant milestone for Stemedica to bring this new cellular medication to patients with debilitating conditions caused by a stroke. Stemedica plans to move forward to a Phase-IIb discussion with the FDA."

Michael Levy, MD, PhD, FACS, FAANS, Professor of Neurosurgery at UCSD and the Principal Investigator of this study commented: "Based on my clinical trial work in Stemedica's Ischemic Stroke trial, my experience to date with Stemedica's allogeneic ischemic tolerant mesenchymal stem cell product suggests that the product is first and foremost safe and secondarilyhas the potential to produce unparalleled medical benefits."

About Stemedica Cell Technologies, Inc.Stemedica Cell Technologies, Inc. is a global biopharmaceutical company that manufactures best-in-class allogeneic adult stem cells. The company is a government licensed manufacturer of cGMP, clinical-grade stem cells currently used in US-based clinical trials for ischemic stroke, and Alzheimer's Disease. Stemedica's cell are also used on a worldwide basis by research institutions and hospitals for pre-clinical and clinical (human) trials. Stemedica is currently developing additional clinical trials for other medical indications using adult, allogeneic stems cell under the auspices of the FDA and other international regulatory institutions. The company is headquartered in San Diego, California and can be found online atwww.stemedica.com.

Forward Looking StatementsThis press release may contain forward-looking statements. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance and you are cautioned not to place undue reliance on these forward-looking statements. These statements reflect the views of Stemedica as of the date of this press release with respect to future events and, except as required by law, it undertakes no obligation to update or revise publicly any forward looking statements, whether as a result of new information, future events or otherwise after the date of this press release.

Media ContactStemedica Cell Technologies, Inc.Dave McGuiganEVP, Marketing & Business Developmentdmcguigan@stemedica.com+1 858-658-0910 x7203

SOURCE Stemedica Cell Technologies, Inc.

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Positive Study Results of Phase IIa Clinical Trial Using Intravenous Administration of Mesenchymal Stem Cells for Ischemic Stroke Published in...

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