Capricor Granted FDA Orphan Drug Designation for Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

Industry News

Capricor Therapeutics, a biotech company based in Beverly Hills, CA, announced that the FDA has granted Orphan Drug Designation for its cell therapeutic product candidate, CAP-1002, for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD).  DMD is a rare genetic disease caused by mutations in the dystrophin gene.  It is characterized by progressive muscle degeneration and weakness.  Development of cardiomyopathy is common and is the leading cause of death in patients.  Currently, there is no cure for DMD, nor is there any treatment for its associated cardiomyopathy complication.  CAP-1002 is a cardiac cell therapy composed of cardiosphere-derived cells.  Preclinical studies have shown that CAP-1002 promotes cardiomyogenesis and angiogenesis, while inhibiting oxidative stress, inflammation and fibrosis.  The company is planning to file an IND (Investigational New Drug Application) with the FDA in the near future.


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