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Start-Up To Develop Stem Cell Therapies For Blood Cancers, Related Disorders: Company Licenses Technology Developed At Mount Sinai.

NEW YORK, N.Y., January 9, 2020 -- In a collaboration with the Mount Sinai Health System, clinical-stage start-up Tacitus Therapeutics will develop stem cell therapies initially targeting blood cancers and related clotting disorders.

The first therapy, HSC100, is being investigated in a Phase 1 clinical trial.

The technology developed and licensed by Mount Sinai is based on research by Tacitus co-founders Ronald Hoffman and Camelia Iancu-Rubin.

The technology includes proprietary cell expansion, differentiation and engineering methods used to manufacture healthy cells that overcome the limitations of traditional allogeneic, or donor, cell transplantations.

Blood cancers comprise about 10 percent of new cancer cases in the U.S. each year, and almost 60,000 people die from blood cancer complications annually. Most blood cancers start in the bone marrow, where blood is produced.

A common therapy for such blood cancers is a hematopoietic stem cell (HSC) treatment (bone marrow transplantation), in which doctors infuse healthy HSCs into the patient's bloodstream, where they migrate to the bone marrow to grow or engraft.

HSCs for this process can be collected from bone marrow, circulating blood, or umbilical cord blood (CB) of healthy donors.

While HSC transplants are common, significant barriers to success exist, including high levels of graft-versus-host disease, low numbers of healthy cells obtained from CB, and increased risk of bleeding due to delayed megakaryocyte, or platelet, engraftment.

Hoffman and Iancu-Rubin are pioneers of bone marrow cell therapy treatments, and development of this technology was enabled by the New York State Stem Cell Science program, NYSTEM.

As a New York State Department of Health initiative, NYSTEM awarded a $1 million grant to Hoffman in 2010 that supported the original research underpinning this platform technology.

In 2015, NYSTEM awarded Hoffman and Iancu-Rubin an $8 million grant to translate the technology from the laboratory into the clinic, where it is currently in clinical trial.

"Tacitus is committed in its mission to advance next-generation cell therapies with curative potential," said CEO Carter Cliff. "Based on our founders' solid foundation of research, we are translating these discoveries into broad clinical practice as we look to dramatically improve the standard of care for patients with life-threatening conditions."

HSC100 is an investigational therapy based on allogeneic hematopoietic stem cells (HSC) expanded from umbilical cord blood. HSC100 is being investigated in an open-label Phase 1 clinical trial in the U.S. for treatment of hematological malignancies.

The success of unmanipulated cord blood as a source of stem cells has been hampered by the small number of stem cells present in a single cord, leading to delayed engraftment and frequent graft failure.

The proprietary technology includes the use of an epigenetic modifier, valproic acid, to expand the number and the quality of HSCs found in cord blood collections.



Contact (Mount Sinai Innovation Partners):

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Title Annotation:Research Partnerships
Publication:Stem Cell Research News
Geographic Code:1U2NY
Date:Jan 27, 2020
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