Programmed hematopoietic cells show potential

Fate Therapeutics demonstrates treatment option that could be life-saver for people with hematologic malignancies

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SAN DIEGO—Fate Therapeutics Inc. is a clinical-stage biopharmaceutical company engaged in the discovery and development of pharmacologic modulators of adult stem cells to treat severe, life-threatening orphan diseases, including hematologic malignancies, lysosomal storage disorders (LSDs) and muscular dystrophies. Incorporated on April 27, 2007, the company’s approaches use pharmacologic modalities, including small molecules and therapeutic proteins, to enhance the therapeutic potential of adult stem cells.
Adult stem cells play a key role in the growth, maintenance and repair of many tissues and organ systems in the body. Because they can self-renew and regenerate and repair diseased or damaged tissue, adult stem cells hold considerable therapeutic promise according to the company.
At the 56th Annual Meeting and Exposition of the American Society of Hematology (ASH) in December, Fate Therapeutics released preclinical data highlighting the pharmacological properties of ex-vivo programmed hematopoietic cells sourced from mobilized peripheral blood. Using a newly identified combination of two small-molecule modulators, scientists from the company demonstrated that both T cells and CD34+ cells from mobilized peripheral blood can be modulated ex vivo, with preclinical evidence pointing to the programmed hematopoietic cells having improved therapeutic potential.
The company’s hematopoietic stem cell (HSC) modulation platform focuses on the ex-vivo pharmacologic optimization of HSCs, which are adult stem cells that regenerate all types of blood cells throughout a person’s lifespan. HSCs have been used for the curative procedure called hematopoietic stem cell transplant (HSCT).
Hematopoietic stem cell transplantation represents a potentially life-saving treatment option for children and adults afflicted with hematologic malignancies. The number of procedures performed has increased steadily over the past two decades. Currently, about 60,000 HSCT procedures are performed on a worldwide annual basis.
The company’s product candidate, PROHEMA, is a pharmacologically modulated HSC therapeutic derived from umbilical cord blood. It has human proof of concept for PROHEMA in the clinical setting by demonstrating enhanced and durable engraftment of HSCs within the bone marrow. The company is advancing PROHEMA in Phase 2 clinical development for hematologic malignancies. It is also engaged in the development of pharmacologically optimized HSC therapeutics for the treatment of LSDs, where HSCs have the ability to migrate to and engraft within the central nervous system. PROHEMA is produced through a proprietary, two-hour, ex-vivo programming process using a small-molecule modulator (FT1050) that promotes rapid and supra-physiologic activation of genes involved in the homing, proliferation and survival of HSCs, which are key biological properties necessary for durable engraftment and hematopoietic reconstitution.
Fate Herapeutics’ satellite stem cell (SSC) modulation platform focuses on the in-vivo pharmacologic activation of SSCs, which are adult stem cells that regenerate muscle throughout a person’s lifespan. The company has identified Wnt7a as a natural promoter of SSCs to drive muscle regeneration, and it is focused on developing Wnt7a analogs for the treatment of muscular dystrophies. Wnts comprise a range of 19 secreted proteins known to play a key physiological role in developmental and regenerative processes.
“While hematopoietic stem cell transplantation has proven curative potential, a significant need remains to reduce the morbidity and mortality associated with the procedure, including the risk of T cell-mediated complications such as viral infections, graft vs. host disease and delayed immune reconstitution,” explains Dr. Christian Weyer, president and CEO of Fate Therapeutics. Weyer joined Fate Therapeutics after a 12-year tenure with Amylin Pharmaceuticals Inc., where he most recently served as senior vice president of research and development until the completion of Amylin’s acquisition by Bristol-Myers Squibb Co. in August 2012. 
“We are excited that our ex-vivo programming platform has identified a combination of small-molecule modulators that promote the supra-physiologic activation of genes implicated in the cell cycle, immune tolerance and antiviral properties of T cells, as well as in the survival, proliferation and engraftment potential of CD34+ cells,” Weyer adds. “We believe these findings form a compelling scientific basis to support the clinical evaluation of ex-vivo programmed mobilized peripheral blood in patients undergoing hematopoietic stem cell transplantation for the treatment of hematologic malignancies.”

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