Inducing skeletal muscle regeneration may have numerous potential therapeutic applications, including the treatment of sarcopenia, cancer induced cachexia and muscular dystrophies, afflicting up to 17 million, 1.3 million and 50 thousand people in the US, respectively.  Other conditions, such as injury / immobilization-induced muscular atrophy or stress urinary incontinence, may impact only certain isolated muscle groups within the body.  Fate is pursuing FT301 therapeutic forms that can be injected locally for these isolated muscle conditions and is also evaluating systemic delivery of certain FT301 forms to enable treatment of systemic conditions such as Duchenne Muscular Dystrophy, sarcopenia and cachexia.

There are over 650 different striated skeletal muscles in the human body.  These skeletal muscles are responsible for a range of functions, including standing, ambulation, chewing, breathing and controlled urine excretion.

Because of this multitude of functions for skeletal muscle, a product that helps regenerate weakened skeletal muscle could have a host of potential therapeutic applications.  Fate’s FT301 preclinical development program utilizes a recombinant version of a naturally occurring protein to target the body’s endogenous skeletal muscle repair pathway.

FT301 has been shown in published research to selectively expand a pool of muscle stem cells (called satellite cells), and cause the growth of differentiated muscle cells.  This dual mechanism of action results in balanced, productive growth of skeletal muscle: long term skeletal muscle hypertrophy and increased strength is supported by an increased number of satellite stem cells.  Fate is currently optimizing selected protein forms and testing these forms across multiple areas of pharmacology.  The scientific foundation of our FT301 program is based on the ground-breaking research of one of Fate’s scientific founders, Dr. Michael Rudnicki, whose research on this topic is the subject of several high profile publications in journals such as Cell (Seale et al., Cell 2000; Kuang et al., Cell 2007; LeGrand et al., Cell Stem Cell 2009).