oa Pharmacological modulators of stem cells as novel therapeutics

Fate Therapeutics (Fate) is pursuing the pharmacological modulation of adult stem cells to enhance tissue regeneration. Fate's lead clinical program, ProHema, consists of pharmacologically-enhanced hematopoietic stem cells (HSCs), designed to improve HSC engraftment and hematopoietic reconstitution during the normal course of a stem cell transplant for the treatment of patients with hematologic malignancies. Fate is currently conducting three clinical phase II trials of ProHema (FT1050‐enhanced umbilical cord blood). In addition, Fate is advancing a human recombinant protein, FT301, engineered for improved pharmacologic properties, and for driving the regeneration of skeletal muscle cells for use in atrophy and dystrophy. FT301 has a novel mode of action that results in the expansion of satellite stem cells. Fate has applied its proprietary induced pluripotent stem cell (iPSC) technology to test the efficacy of FT301 in a human model of Duchenne muscular dystrophy (DMD). We established iPSC lines from patients with DMD, and explored the regenerative capacity of FT301 on myoblasts generated from the DMD-specific iPSCs. Treatment of the iPSC-derived muscle fibers with FT301 induced significant hypertrophy confirming our in vitro and in vivo observed effects of FT301 in inducing productive muscular hypertrophy, and highlighting its possible application in muscular degenerative disorders.