In  the  MDX  mouse  model  of  muscular  dystrophy,  intramuscular  injection  of  a  novel  WNT7a  analog resulted in significant dose dependent muscle hypertrophy and several-fold expansion of the satellite stem  cell  population.  Moreover,  three  weeks  after  a single  intramuscular  injection,  functional assessment  revealed  a  significant  increase  in  strength  of  the  targeted  tibialis  anterior muscle  (+18%, p<0.001).  These  functional  improvements  were  accompanied  by  a  significant  reduction  in  markers  of fiber necrosis and inflammation typically seen in dystrophic muscle. Significant muscle hypertrophy and satellite  stem  cell  expansion  were  also  observed  in wild-type  mice.  The  WNT7a  analogs  assessed  in these studies were engineered to overcome several key challenges of Wnt-family protein manufacture and formulation while retaining full biological activity and specificity. These and other data suggestthe potential  of  WNT7a-based  protein  therapeutics  across  a  range  of  neuromuscular  diseases  and conditions.

The findings obtained with Fate’s optimized WNT7a analogs expand upon those previously reported with non-modified WNT7a protein. In November 2012, muscle biology expert and Fate Therapeutics scientific founder Dr. Michael Rudnicki published data demonstrating the potential of WNT7a to ameliorate muscle degeneration in the MDX mouse model of muscular dystrophy (Von Maltzahn et. al.,
PNAS 2012). In previous studies, Dr. Rudnicki elucidated theunique biology of WNT7a and its dual mechanism of action of driving the expansion of themuscle satellite stem cell population and muscle hypertrophy.

About Muscular Dystrophy
Muscular  dystrophies  encompass  a  group  of  disorders with  diverse  pathophysiological  manifestations resulting from genetic aberrations which include mutations or deletions to over 30 distinct genes. The most  prevalent  and  well  characterized  is  Duchenne  muscular  dystrophy  (DMD),  an  X-linked  form  of muscular dystrophy which is seen in 1/3500 live male births. DMD typically manifests in early childhood and  progresses  to  an  advanced  stage  of  severe  muscular  degeneration  resulting  in  impairment  of ambulation and premature mortality. A core pathophysiologic phenomenon seen in muscular dystrophy is  a  cycle  of  muscle  degeneration  leading  to  continuous  compensatory  satellite  cell  activation  and differentiation  to  affect  a  regenerative  response,  but  resulting  in  the  eventual  exhaustion  of  the regenerative capacity and significant loss of muscle function. Enhancing the underlying molecular and cellular mechanisms to restore the regenerative capacity of muscle satellite stem cells thus represents a promising and unique approach for therapeutically intervention in various forms of muscular dystrophy as well as other neuromuscular diseases.

About Fate Therapeutics, Inc.
Uniquely  positioned  at  the  intersection  of  stem  cell  science  and  orphan  disease,  Fate  Therapeutics  is pioneering  the  discovery  and  development of  innovative  adult  stem  cell  modulator  therapeutics  with the  potential  to  cure  or  transform  the  lives  of  patients  with  rare  life-threatening  disorders.  The Company’s  lead  program,  ProHema,  an  innovative  cord blood-derived  cell  therapy  containing  ex-vivo pharmacologically-modulated hematopoietic stem cells (HSCs), is currently in Phase 2 testing in patients with  leukemia  undergoing  hematopoietic  transplantation.  The  Company  plans  to  pursue  clinical evaluation  of  pharmacologically  modulated  HSCs  in  patients  with  rare  genetic  disorders,  an  area  of tremendous unmet medical need in which the curativepotential of cord blood transplantation is well recognized. In addition, Fate Therapeutics is developing proprietary WNT7a-based protein therapeutics that have shown efficacy in preclinical models of muscular dystrophy. To advance its discovery efforts, the Company applies its award-winning, proprietary,induced pluripotent stem cell (iPSC) technology to generate rare cell populations and model disease. Fate Therapeutics is headquartered in San Diego, CA, with a subsidiary in Ottawa, Canada. For more information, please visit www.fatetherapeutics.com.

Contact:
Paul Cox, Stern Investor Relations, Inc.
212.362.1200, paul@sternir.com