Stem cells on the ballot – Science Magazine

California's ballot measures often reveal much about the broader U.S. policy environment. This is particularly true of the approval by the state's voters in November of Proposition 14, The California Stem Cell Research, Treatments, and Cures Initiative of 2020. Proposition 14 extends the 2004 ballot Proposition 71, which established the California Institute for Regenerative Medicine (CIRM) and authorized $3 billion in state-issued bonds for CIRM to fund stem cell and regenerative research and medicine (restricted to California). Proposition 14, which authorizes $5.5 billion over the next 10 years to continue CIRM's work, succeeded in part by informing voters of CIRM's successes and that its conflict-of-interest provisions are extremely strong. This state-level action is critical because, contrary to opponents' opinions, the overall policy environment for human stem cell research in the United States is in some ways worse now than when Proposition 71 passed.

Since 2004, CIRM has funded groundbreaking work on immune disorders, cancer, spinal cord injury, diabetes, and more. The result has been more than 90 stem cellrelated clinical trials (directly or indirectly supported by CIRM), almost 3000 scientific papers, and contributions to two cancer therapies approved by the U.S. Food and Drug Administration. The lives of many patients have improved because of CIRM. Notably, many CIRM-funded clinical trials rely on human embryonic or fetal stem cells, whereas the federal government currently does not fund any clinical trials using these types of cells.

Proposition 71 was motivated largely in response to restrictions on human embryonic stem cell research in the United States in 2004. However, although research was limited to a small number of human embryonic stem cell lines, there was no formal ban on federal funding of research on such stem cells. In addition, in 2004 there were no restrictions on federal funding of human fetal stem cell and tissue research; however, there is now near-complete blockage of federal funding for such research. And federal funding for human embryonic stem cell research is again at risk. On 4 September 2020, 22 Republican senators and 72 Republican House members wrote to President Trump requesting an end to all federal funding of human embryonic stem cell research. Could President Trump impose a ban that would be difficult to revoke? Or, could Republican senators manufacture a ban by legislative maneuvering on a budget reconciliation vote, which requires 60% support? Such maneuvering created the effectively permanent 1995 Dickey-Wicker amendment, which prohibits federal funding of any research in which human embryos are created or destroyed. Dickey-Wicker has limited research on in vitro fertilization methods and stalled progress on understanding early human development. It has not solved the problem of the many, perhaps 1 million frozen embryos in the United States that will not be used for in vitro fertilization and will be destroyed without benefit if not used for research. Vital long-term research is greatly harmed by the U.S. policy environment, with the likely outcome that many young scientists will avoid research using human embryonic stem cells and human fetal tissue.

Restrictions on valuable, ethical research appear particularly fool-hardy during a deadly pandemic. Research on viruses such as HIV and SARS-CoV-2 (severe acute respiratory syndrome coronavirus 2) can benefit greatly from work using mice that utilize human fetal stem cells and tissues to generate a human-like immune system. These mice allow evaluation of a human immune system in the contexts of infection mechanisms, generation of immunity, and drug response. These studies can be supported with Proposition 14 funds in California, but not with federal funds. It is crucial for the incoming Biden administration to evaluate the need for federal funding in these important areas with high-quality scientific input and evidence.

California's vote on Proposition 14 should also help the rest of the country appreciate the need to increase investments in biomedical research at the U.S. National Institutes of Health and other federal agencies. Current biomedical research expenditures amount to only a tiny fraction of the costs of disease, so an objective evaluation of appropriately increased research funding relative to disease costs is warranted. Once again, California is showing the way.

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Stem cells on the ballot - Science Magazine

2021 Startups to Watch: Ronawk’s T-Blocks stack the future of stem cell research in an Olathe lab – Startland News

Editors note: Startland News selected 10 Kansas City firms to spotlight for its annual Startups to Watch list. The following is one of 2021s companies. Click here to view the full, ranked list of Startups to Watch presented bysponsorsHusch Blackwelland the Ewing Marion Kauffman Foundation.

Born out of a frustration with growing stem cells, scientists A.J. Mellott and Heather Decker found a solution that would make cell production more efficient and consume fewer materials, Mellott shared.

Elevator pitch: Ronawk develops customized 3D-printed consumables to accelerate cell production in the biotech, healthcare and agriculture industries.

Founders: A.J. Mellott, Heather Decker Founding year: 2019 Amount raised to date: $1.3M Noteworthy investors: Undisclosed Programs completed: NIGMS Sustainable Heartland Accelerator Regional Partnership Hub,Wichita State University Shockers Innovation Corps, UMKC E-Scholars, Digital Sandbox KC, Pipeline Entrepreneurs Current employee count: 3 (2 FTE, and 1 part-time)

We came up with these Tissue Blocks, also referred to as T-Blocks, said Mellott, who co-founded Ronawk with Decker in February 2019 after the duo left their jobs at University of Kansas Medical Center to pursue the idea.

T-Blocks 3D model allows researchers to rapidly expand the growth of cells, eliminate the need to subculture and greatly reduce labor costs, Mellott stated. With all these benefits at hand, the COVID-19 pandemic accelerated Ronawks timeline.

COVID gave us this unique opportunity, he continued, noting the pandemic caused heightened interest in using T-Blocks as a way to research the never-before-studied virus. But even outside of COVID, there are several applications to how our technology can be used.

Click here to read more about Ronawks journey throughout the pandemic.

Already having closed a first round of funding and other additional open funding in 2020, Ronawk is set to launch its Series A funding in early 2021. The funding is designated to allow Ronawk to expand both its team and Olathe lab facility to meet the production needs of customers, Mellott stated.

The biggest goal for 2021: secure a manufacturing partner able to do large-scale production.

That need is there, Mellott shared. We are also already in conversations with a large distributor that is testing our product because they are looking at the potential to include it co-branded with a product that they sell. Its another exciting opportunity.

Ronawk is also on track to receive its Good Manufacturing Practices (GMP) certification this year, Mellott added.

Our product right now is what is considered 510K exempt; its a class one device with the FDA, Mellott explained. What that means is we only have to manufacture under GMP conditions, but it does not require an FDA approval to be used. We will be making this leap from what was a [research and development] product to now a GMP grade version of the product.

Having a GMP-certified product will help close production deals, Mellott said noting that several companies already in trial with the T-Blocks are interested in large purchase orders once the product is certified.

We have 23 product trials going on in four of the seven continents, he said. Some of our customers are testing stem cells, others are testing cancer cells, and even some are actually looking for things that they can procure from the cells.

Were hoping that well have some of that positive data, possibly by the turn of the year, but we expect the bulk of it to come in early to mid-quarter one of 2021.

Click here to read about Ronawks T-Blocks run for the Kansas Chambers Coolest Thing Made in Kansas prize.

As a scientist who added entrepreneur to his title, Mellot said he has learned a lot from the innovative ecosystem and is grateful for the chance to share his expertise.

Its been chaotic and intense, but also fun and rewarding, Mellott said of his journey so far. Its allowed us to meet a number of people, and weve gotten to learn more about the human aspect of community which is a main reason we founded the company. We wanted to be able to help people with our knowledge.

The Kansas City Startups Watch in 2021 list is made possible by presenting sponsorsHusch Blackwell,a value-driven law firm with offices in Kansas City, and the Ewing Marion Kauffman Foundation, though independently produced by Startland News.

Startups to Watch in 2021

1) TripleBlind 2) LaborChart 3) Bar K 4) Ronawk 5) SureShow 6) Daupler 7) PMI Rate Pro 8) Scissors & Scotch 9) Replica 10) The Market Base

Startups to Watch is now in its sixth year, thanks to ongoing support from the Ewing Marion Kauffman Foundation, a private, nonpartisan foundation that works together with communities in education and entrepreneurship to create uncommon solutions and empower people to shape their futures and be successful.

For more information, visit http://www.kauffman.org and connect at http://www.twitter.com/kauffmanfdnandwww.facebook.com/kauffmanfdn

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2021 Startups to Watch: Ronawk's T-Blocks stack the future of stem cell research in an Olathe lab - Startland News

Towards a Cure: Carleton Research Team Working on Stem Cell Therapy to Reverse Type 1 Diabetes – Carleton Newsroom

Tyrone Burke, January 13, 2021

The human pancreas is only about 15 cm long, but within it are about a million tiny islets of hormone-producing cells. Though they are many in number, these islets are tiny they make up only 2-3% of the volume of the pancreas. Cells within these islets secrete hormones that help us regulate our blood sugar. And when they malfunction, it can cause diabetes.

Carletons Jenny Bruin is part of a team of researchers that has been awarded a 5-year, $3 million grant from the Canadian Institutes of Health Research (CIHR) and JDRF Canada to develop a novel therapy that transplants insulin-secreting cells derived from stem cells into patients with Type 1 diabetes.

This could reverse the effects of the condition, and help eliminate diabetics need for insulin therapy.

People with Type 1 diabetes lack insulin secreting cells in the pancreas. These are called beta cells, and the immune systems has destroyed them, says Bruin, an Assistant Professor in the Department of Biology and the Institute of Biochemistry.

We want to replace them with beta cells derived from pluripotent stem cells. Right now, we can take stem cells part way down the path to becoming beta cells in vitro. When we transplant these cells into mice, the final stages of maturation occur in the mouse over a period of several months. It works really well, but it is kind of like a black box we dont know what is happening in the time after the cells are transplanted, but before they start producing insulin and responding to glucose.

The project brings five principal applicants and two co-researchers, and will use several different techniques to understand factors that could be influencing the cell maturation process. For example, Francis Lynn of the University of British Columbia is conducting a detailed characterization of stem cell-derived beta cells and human beta cells from organ donors to identify differences between the two different types, and Pat MacDonald of the University of Alberta is studying the electrophysiology of these cells how ions move in and out.

If we can understand what is functionally missing at a genetic level, then it should be possible to target those gaps, and activate the key pathways that are missing, says Bruin.

Transplants of stem cells into human patients are currently being tested for safety, but the ultimate aim is to be able to transform stem cells into fully functional beta cells in a dish.

Some think it could be sufficient to transplant cells that are secreting insulin, but not yet fully functional. And it might be, but the environment that the cells are being transplanted into is hugely variable. There is a lot of room for environmental factors to throw off the process inside a human, says Bruin.

Bruins role in the project is to identify how common contaminants and pollutants could be affecting the maturation process.

When we grow these cells in a dish, it is a very controlled environment. We control every step, and every component of the media that they are in. But when we transplant them into a patient, the environment is completely uncontrolled. We know that people across Canada are exposed to all kinds of environmental pollutants, and we can measure those in their blood and their tissues, says Bruin.

My lab is interested in how these contaminants are influencing beta cell function, not just in transplanted cells, but also in our pancreas. That environmental exposure might potentially influence the function of beta cells, and our ability to successfully transplant stem cells.

This has implications for the new stem cell transplantation technique, but also for our understanding of diabetes more broadly.

We focus mainly on the beta cell because it is critical for both Type 1 and Type 2 diabetes. Any defect in beta cells will affect the regulation of glucose. If the way that beta cells secrete insulin or sense glucose is affected by chemicals in our environment or additives in food or there is beta cell death in response to some of these exposures then you have fewer functional beta cells, says Bruin.

One of the problems for people with Type 2 diabetes is that their beta cells dont work as well as healthy individuals. In early stages of the disease, they secrete too much insulin. At later stages, they dont secrete enough, in part because they lose maturity. We dont understand how beta cells become mature, and what potentially sends them backwards. This research could help us understand why beta cells in people with Type 2 diabetes dont work as well as they should, but our primarily goal is to learn how to generate fully mature beta cells from stem cells for treating patients with Type 1 diabetes.

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Towards a Cure: Carleton Research Team Working on Stem Cell Therapy to Reverse Type 1 Diabetes - Carleton Newsroom

Stem Cell Assay Market | Know the aspects that will serve as game-changers for the market – BioSpace

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. The assay consists of qualitative or quantitative analysis or testing of affected tissues and tumors, wherein their toxicity, impurity, and other aspects are studied.

With the growing number of successful stem cell therapy treatment cases, the global market for stem cell assays will gain substantial momentum. A number of research and development projects are lending a hand to the growth of the market. For instance, the University of Washingtons Institute for Stem Cell and Regenerative Medicine (ISCRM) has attempted to manipulate stem cells to heal eye, kidney, and heart injuries. A number of diseases such as Alzheimers, spinal cord injury, Parkinsons, diabetes, stroke, retinal disease, cancer, rheumatoid arthritis, and neurological diseases can be successfully treated via stem cell therapy. Therefore, stem cell assays will exhibit growing demand.

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Another key development in the stem cell assay market is the development of innovative stem cell therapies. In April 2017, for instance, the first participant in an innovative clinical trial at the University of Wisconsin School of Medicine and Public Health was successfully treated with stem cell therapy. CardiAMP, the investigational therapy, has been designed to direct a large dose of the patients own bone-marrow cells to the point of cardiac injury, stimulating the natural healing response of the body.

Newer areas of application in medicine are being explored constantly. Consequently, stem cell assays are likely to play a key role in the formulation of treatments of a number of diseases.

Global Stem Cell Assay Market: Overview

The increasing investment in research and development of novel therapeutics owing to the rising incidence of chronic diseases has led to immense growth in the global stem cell assay market. In the next couple of years, the market is expected to spawn into a multi-billion dollar industry as healthcare sector and governments around the world increase their research spending.

The report analyzes the prevalent opportunities for the markets growth and those that companies should capitalize in the near future to strengthen their position in the market. It presents insights into the growth drivers and lists down the major restraints. Additionally, the report gauges the effect of Porters five forces on the overall stem cell assay market.

Global Stem Cell Assay Market: Key Market Segments

For the purpose of the study, the report segments the global stem cell assay market based on various parameters. For instance, in terms of assay type, the market can be segmented into isolation and purification, viability, cell identification, differentiation, proliferation, apoptosis, and function. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits. Based on instruments, flow cytometer, cell imaging systems, automated cell counter, and micro electrode arrays could be the key market segments.

In terms of application, the market can be segmented into drug discovery and development, clinical research, and regenerative medicine and therapy. The growth witnessed across the aforementioned application segments will be influenced by the increasing incidence of chronic ailments which will translate into the rising demand for regenerative medicines. Finally, based on end users, research institutes and industry research constitute the key market segments.

The report includes a detailed assessment of the various factors influencing the markets expansion across its key segments. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length.

Global Stem Cell Assay Market: Regional Analysis

Regionally, the market is expected to witness heightened demand in the developed countries across Europe and North America. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. Besides this, the market is also expected to witness lucrative opportunities in Asia Pacific and Rest of the World.

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Global Stem Cell Assay Market: Vendor Landscape

A major inclusion in the report is the detailed assessment of the markets vendor landscape. For the purpose of the study the report therefore profiles some of the leading players having influence on the overall market dynamics. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the reports forecast period.

Some of the most prominent enterprises operating in the global stem cell assay market are Bio-Rad Laboratories, Inc (U.S.), Thermo Fisher Scientific Inc. (U.S.), GE Healthcare (U.K.), Hemogenix Inc. (U.S.), Promega Corporation (U.S.), Bio-Techne Corporation (U.S.), Merck KGaA (Germany), STEMCELL Technologies Inc. (CA), Cell Biolabs, Inc. (U.S.), and Cellular Dynamics International, Inc. (U.S.).

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Stem Cell Assay Market | Know the aspects that will serve as game-changers for the market - BioSpace

Center for cell and gene therapy to open next year – Harvard Gazette

An innovative public-private partnership took a big step in its plan to open a center next year that aims at boosting advances in cell and gene therapy in the region, signing a 10-year lease for a 40,000-square-foot facility in Watertown.

Project participants refer to the facility, which has not been formally named, as the center for advanced biological innovation and manufacturing (CABIM). The goal is to increase availability of materials like genetically altered cells that are essential to advancing discoveries from the lab to clinics for use in treating patients.

There has been great progress in developing pharmaceuticals small-molecule drugs to treat a wide range of diseases, said Harvard Provost Alan Garber, who has led the effort. But many conditions resist treatment with conventional pharmaceuticals. Cell-based therapies offer biological approaches that are complementary to and sometimes far more effective than chemistry-based treatments.

Scientists say that a bottleneck in manufacturing such biological materials is slowing the development of cutting-edge advances in gene therapy, stem cell science, regenerative medicine, CRISPR/Cas9 gene editing, and cancer immunotherapy. An array of treatments based on those and similar technologies such as those involving RNA, peptides, and oligonucleotides are in development, in clinical trials, and in some cases already in the clinic.

This facility will help turn scientific findings into approved therapies by making these resources available to early-stage companies and labs.

Alan Garber, Harvard provost

The center, whose creation was announced in late 2019, is led by institutions from both academia and industry. It will contain both manufacturing and innovation space to boost the supply of materials for late-stage research and early clinical trials and provide space to develop ideas that have left the lab but are not yet ripe for corporate investment. It will also emphasize training in the operation of advanced equipment used in cell manufacturing as a way to increase the pool of workers with such critical skills in the region.

The promise of cell-based therapies has been proven, Garber said, pointing to recent gene-therapy trials to treat sickle cell anemia, which showed significant improvement. He also cited stem-cell-based work to treat diabetes by implanting insulin-producing beta cells, developed in the Harvard lab of Xander University Professor Douglas Melton.

The development of tools like CRISPR and progress in stem-cell science are among the advances that have given us hope that we will soon be able to treat cancer, immunological diseases, neurological conditions, and many other inherited conditions far better, Garber said. This facility will help turn scientific findings into approved therapies by making these resources available to early-stage companies and labs.

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Center for cell and gene therapy to open next year - Harvard Gazette

Covid-19 Impact On Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020 Booming So Rapidly by 2027 | Fibrocell Science, Inc., Genzyme…

A new market study on the 2020-2027Autologous Stem Cell and Non-Stem Cell Based Therapies Marketwith 100+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. At present, the market is developing its presence. The Research report presents a complete assessment of the Market and contains a future trend, current growth factors, attentive opinions, facts, and industry-validated market data. Report offering you more creative solutions that combine our deep geographic experience, intimate sector knowledge and clear insights into how to create value in your business. The research study provides estimates for the 2020-2027 Autologous Stem Cell and Non-Stem Cell Based Therapies Market Forecast till2027*.

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Major Market Top Key Players: Autologous Stem Cell and Non-Stem Cell Based Therapies market

Caladrius Biosciences, Vericel Corporation, Fibrocell Science, Inc., Genzyme Corporation, BrainStorm Cell Therapeutics, Regeneus Ltd., and Dendreon Corporation.

Regional Analysis:

North America: United States, Canada, Mexico

Europe: Germany, France, UK, Russia, Italy, Rest of Europe

Middle East Africa: Turkey, Egypt, South Africa, GCC Countries, Rest of Middle East & Africa

Asia-Pacific: India, Australia, Japan, China, South Korea, Indonesia, Malaysia, Philippines, Thailand, Vietnam

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Further, in the research report, the following points are included along with an in-depth study of each point:

Production Analysis Production is analyzed with respect to different regions, types, and applications. Here, the price analysis of various Market key players is also covered.

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Innovation Techniques enlisted for the development in the market.

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Some of the key questions answered in this report:

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What was the size of the emerging Autologous Stem Cell and Non-Stem Cell Based Therapies market by value in 2020?

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What trends, challenges and barriers will impact the development and sizing of the Global Autologous Stem Cell and Non-Stem Cell Based Therapies market?

What is sales volume, revenue, and price analysis of top manufacturers of Autologous Stem Cell and Non-Stem Cell Based Therapies market?

What are the Autologous Stem Cell and Non-Stem Cell Based Therapies market opportunities and threats faced by the vendors in the global Autologous Stem Cell and Non-Stem Cell Based Therapies Industry?

The reports conclusion leads into the overall scope of the Global market with respect to feasibility of investments in various segments of the market, along with a descriptive passage that outlines the feasibility of new projects that might succeed in the Global Autologous Stem Cell and Non-Stem Cell Based Therapies market in the near future.

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Covid-19 Impact On Autologous Stem Cell and Non-Stem Cell Based Therapies Market 2020 Booming So Rapidly by 2027 | Fibrocell Science, Inc., Genzyme...

Jasper Therapeutics and Graphite Bio Announce Collaboration to Evaluate JSP191 as Conditioning Regimen for Novel Gene Replacement Therapy in Patients…

Details Category: DNA RNA and Cells Published on Wednesday, 13 January 2021 17:53 Hits: 293

REDWOOD CITY, CA & SOUTH SAN FRANCISCO, CA, USA I January 13, 2021 I Jasper Therapeutics, Inc., a biotechnology company focused on hematopoietic cell transplant therapies, and Graphite Bio, Inc., a next-generation gene editing company focused on therapies that harness targeted gene integration to treat or cure serious diseases, today announced a research and clinical collaboration agreement to evaluate JSP191, Jaspers first-in-class anti-CD117 monoclonal antibody, as a targeted, non-toxic conditioning regimen for Graphite Bios investigational GPH201 gene replacement therapy for severe combined immune deficiency (SCID) in patients with IL2RG deficiency, known as x-linked SCID (XSCID).

XSCID is a severe, inherited disorder of the immune system with symptoms often presenting in early infancy, including persistent infections and failure to thrive. Without treatment, XSCID is typically fatal to patients in the first two years of life.

Graphite Bio is focused on the development of potentially curative therapies for patients suffering from serious diseases, using its targeted gene integration platform to harness the natural cellular process of homology directed repair (HDR) in order to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Jasper Therapeutics JSP191 is a first-in-class humanized monoclonal antibody that depletes hematopoietic stem cells from bone marrow and acts as a conditioning agent in patients prior to receiving a hematopoietic stem cell transplant. JSP191 is currently being evaluated in multiple trials as a stem cell depleting conditioning agent, including a Phase 1/2 trial to achieve donor stem cell engraftment in SCID patients undergoing hematopoietic cell transplant and a separate Phase 1/2 trial in AML/MDS patients undergoing hematopoietic cell transplant.

This collaboration with Jasper demonstrates our shared commitment to pioneering novel therapeutic approaches with the potential to significantly improve the treatment experiences of individuals with devastating conditions who stand to benefit from gene replacement therapies, initially for patients with XSCID, said Josh Lehrer, M.Phil., M.D., chief executive officer at Graphite Bio. GPH201 harnesses our targeted gene integration platform to precisely target the defective gene that causes XSCID and replace it with a normal copy. We are impressed by the initial positive clinical results demonstrated by JSP191 when used as a conditioning regimen, and look forward to collaborating with the Jasper team to explore how our novel technologies can be brought to more patients with XSCID and other indications.

Our collaboration with Graphite Bio is an exciting opportunity to further advance the field of curative gene correction by combining a targeted gene integration platform with our first-in-class targeted CD117 antibody, JSP191, that has already demonstrated preliminary clinical efficacy and safety as a conditioning agent in XSCID patients and those with blood cancers undergoing allogeneic hematopoietic stem cell transplant, said Bill Lis, executive chairman and CEO, Jasper Therapeutics.

Graphite Bio and Jasper will collaborate on research, and potentially a clinical study, evaluating JSP191 as a conditioning agent for GPH201. Each company will retain commercial rights to their respective technologies.

About JSP191

JSP191 (formerly AMG 191) 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 stem cells to engraft.

Preclinical studies have shown that JSP191 as a single agent safely depletes normal and diseased hematopoietic stem cells, including in animal models of SCID, myelodysplastic syndromes (MDS) and sickle cell disease (SCD). Treatment with JSP191 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 90 healthy volunteers and patients.

JSP191 is currently being evaluated in two separate clinical studies in hematopoietic cell transplant. The first clinical study is evaluating JSP191 as a sole conditioning agent in a Phase 1/2 dose-escalation and expansion trial to achieve donor stem cell engraftment in patients undergoing hematopoietic cell transplant for severe combined immunodeficiency (SCID), which is potentially curable only by this type of treatment. JSP191 is also being evaluated in combination with another conditioning regimen in a Phase 1 study in patients with MDS or acute myeloid leukemia (AML) who are receiving hematopoietic cell transplant. For more information about the design of these clinical trials, visit http://www.clinicaltrials.gov (NCT02963064 and NCT04429191).

Additional studies are planned to advance JSP191 as a conditioning agent for patients with other rare and ultra-rare monogenic disorders and autoimmune diseases.

About GPH201

GPH201 is a first-in-human investigational hematopoietic stem cell treatment that will be evaluated as a potentially curative therapy for patients suffering from XSCID. GPH201 is generated using Graphite Bios precise and efficient targeted gene integration platform technology to directly replace the defective IL2RG gene, maintain normal IL2RG regulation and expression, and ultimately lead to the production of fully functional adaptive immune cells.

About Jasper Therapeutics

Jasper Therapeutics is a biotechnology company focused on the development of novel curative therapies based on the biology of the hematopoietic stem cell. The companys lead compound, JSP191, is in clinical development as a conditioning antibody that clears hematopoietic stem cells from bone marrow in patients undergoing a hematopoietic cell transplant. This first-in-class conditioning antibody is designed to enable safer and more effective curative hematopoietic cell transplants and gene therapies. For more information, please visit us at jaspertherapeutics.com.

About Graphite Bio, Inc.

Graphite Bio is a next-generation gene editing company focused on the development of potentially curative therapies for patients suffering from serious diseases. The companys targeted gene integration platform harnesses the natural cellular process of homology directed repair (HDR) to efficiently repair genetic defects at their source, deliver genetic cargo with precision and engineer new cellular effector functions. Graphite Bio is leveraging its differentiated platform, initially focused on ex vivo engineering of hematopoietic stem cells, to advance a portfolio of transformative treatments with potential for saving and dramatically improving patients lives. The company was co-founded by academic pioneers in the fields of gene editing and gene therapy, including Maria Grazia Roncarolo, MD, and Matthew Porteus, MD, PhD, and is backed by Versant Ventures and Samsara BioCapital. For more information, please visit graphitebio.com.

SOURCE: Jasper Therapeutics

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Jasper Therapeutics and Graphite Bio Announce Collaboration to Evaluate JSP191 as Conditioning Regimen for Novel Gene Replacement Therapy in Patients...

PureTech Founded Entity Vor Announces FDA Clearance of IND Application for VOR33 – Business Wire

BOSTON--(BUSINESS WIRE)--PureTech Health plc (LSE: PRTC, NASDAQ: PRTC) (PureTech or the Company) is pleased to note that its Founded Entity, Vor Biopharma, a clinical-stage cell therapy company pioneering engineered hematopoietic stem cell (eHSC) therapies combined with targeted therapies for the treatment of cancer, today announced that the U.S. Food and Drug Administration (FDA) has cleared the companys Investigational New Drug (IND) application for VOR33, an eHSC therapy candidate being developed for the treatment of acute myeloid leukemia (AML). Vor plans to initiate a Phase 1/2a clinical trial for VOR33 in the first half of this year.

VOR33, consisting of hematopoietic stem cells that are engineered to lack the CD33 protein, is a cell therapy candidate intended to replace the standard of care in hematopoietic stem cell transplant settings for patients with AML who are at high-risk for relapse.

The full text of the announcement from Vor Biopharma is as follows:

Vor Announces FDA Clearance of IND Application for VOR33

Phase 1/2a clinical trial expected to begin in first half of 2021

CAMBRIDGE, MA January 14, 2021 Vor Biopharma, a clinical-stage cell therapy company pioneering engineered hematopoietic stem cell (eHSC) therapies combined with targeted therapies for the treatment of cancer, today announced that the U.S. Food and Drug Administration (FDA) has cleared the companys Investigational New Drug (IND) application for VOR33, an eHSC therapy candidate being developed for the treatment of acute myeloid leukemia (AML). The company plans to initiate a Phase 1/2a clinical trial for VOR33 in the first half of this year.

VOR33, consisting of hematopoietic stem cells that are engineered to lack the CD33 protein, is a cell therapy candidate intended to replace the standard of care in hematopoietic stem cell transplant settings for patients with AML who are at high-risk for relapse.

Though advances have been made in the treatment of AML and other myeloid malignancies, the median overall five-year survival rate for patients diagnosed with AML remains under 30 percent, said Christopher Slapak, MD, Vors Chief Medical Officer. With the development of VOR33, we are seeking to change the treatment paradigm for AML and potentially other hematologic malignancies. We engineered VOR33 to provide patients with a hematopoietic stem cell transplant that we believe, upon hematopoietic reconstitution, will be treatment resistant to CD33 targeted therapies, potentially resulting in new treatment options and improved post-transplant outcomes.

Clearance of this IND is the culmination of an incredible team effort at Vor and represents a key milestone for us, added Robert Ang, MBBS, MBA, Vors President and Chief Executive Officer. This brings us an important step closer to treating patients with our potentially transformative therapy.

The Phase 1/2a trial is expected to enroll patients with CD33-positive AML who are at high risk of relapse. The primary goals of the trial are to evaluate tolerability and feasibility of the VOR33 stem cell transplant, with a focus on confirming that VOR33 can engraft normally. Following engraftment, patients will be eligible to be treated with Mylotarg, an FDA approved CD33-directed antibody drug conjugate (ADC) therapy owned by Pfizer, in order to potentially prolong leukemia-free survival and provide evidence that VOR33 protects against the myelosuppression that typically accompanies treatment with Mylotarg.

About VOR33

VOR33 is Vors lead product candidate, consisting of eHSCs that we have engineered to lack the protein CD33, and is designed to replace the standard of care in transplant settings for patients suffering from AML and potentially other hematologic malignancies. Once the VOR33 cells have engrafted, we believe that patients can be treated with anti-CD33 therapies, such as Mylotarg or, if approved by the FDA, Vors in-licensed CD33 chimeric antigen receptor T-cell (CAR-T) therapy candidate, with limited on-target toxicity, leading to durable anti-tumor activity and potential cures. In preclinical studies, we have observed that the removal of CD33 provided robust protection of VOR33 eHSCs from the cytotoxic effects of CD33-directed therapies, yet had no deleterious effects on the differentiation or function of hematopoietic cells.

About Vor Biopharma

Vor Biopharma is a clinical-stage cell therapy company that aims to transform the lives of cancer patients by pioneering eHSC therapies to create next-generation, treatment-resistant transplants that unlock the potential of targeted therapies. By removing biologically redundant proteins from eHSCs, we design these cells and their progeny to be treatment-resistant to complementary targeted therapies, thereby enabling these therapies to selectively destroy cancerous cells while sparing healthy cells.

Our platform could be used to potentially change the treatment paradigm of both hematopoietic stem cell transplants and targeted therapies, such as ADCs, bispecific antibodies and CAR-T cell treatments, including Vors in-licensed CD33 CAR-T.

About PureTech Health

PureTech is a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, including intractable cancers, lymphatic and gastrointestinal diseases, central nervous system disorders and inflammatory and immunological diseases, among others. The Company has created a broad and deep pipeline through the expertise of its experienced research and development team and its extensive network of scientists, clinicians and industry leaders. This pipeline, which is being advanced both internally and through PureTechs Founded Entities, as of the date of PureTechs most recently filed Registration Statement on Form 20-F, was comprised of 24 products and product candidates, including two that have received FDA clearance and European marketing authorization. All of the underlying programs and platforms that resulted in this pipeline of product candidates were initially identified or discovered and then advanced by the PureTech team through key validation points based on the Companys unique insights into the biology of the brain, immune and gut, or BIG, systems and the interface between those systems, referred to as the BIG Axis.

For more information, visit http://www.puretechhealth.com or connect with us on Twitter @puretechh.

Cautionary Note Regarding Forward-Looking Statements

This press release contains statements that are or may be forward-looking statements, including statements that relate to our product candidates and approach towards addressing major diseases, future prospects, developments, and strategies. The forward-looking statements are based on current expectations and are subject to known and unknown risks and uncertainties that could cause actual results, performance and achievements to differ materially from current expectations, including, but not limited to, expectations regarding the initiation of a Phase 1/2a clinical trial for VOR33 in the first half of this year, the potential therapeutic benefits of VOR33 and those risks and uncertainties described in the risk factors included in the regulatory filings for PureTech Health plc. These forward-looking statements are based on assumptions regarding the present and future business strategies of the company and the environment in which it will operate in the future. Each forward-looking statement speaks only as at the date of this press release. Except as required by law and regulatory requirements, neither the company nor any other party intends to update or revise these forward-looking statements, whether as a result of new information, future events or otherwise.

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