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Capricor Eyes RMAT Designation Based On Interim Phase II Data

 
• By Mandy Jackson

Capricor reported positive interim Phase I/II results for its cell therapy designed to treat cardiomyopathy in DMD patients and said it plans to seek regenerative medicine advanced technology, breakthrough or both designations from the US FDA.

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Capricor Therapeutics Inc. is considering whether to seek the US FDA's Regenerative Medicine Advanced Technology (RMAT) designation, a breakthrough therapy designation or both for CAP-1002 (allogeneic cardiosphere-derived cells) in the treatment of Duchenne muscular dystrophy (DMD) based on positive interim results from the ongoing 12-month Phase I/II HOPE clinical trial.

Los Angeles-based Capricor designed CAP-1002 to improve cardiomyopathy, which increases as DMD progresses, but in addition to reducing cardiac scarring, a single treatment with the cardiac progenitor cell therapy improved...

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