PRISM Rare Disease Spotlight: Capricor Therapeutics Advances Treatment for Duchenne Muscular Dystrophy

Rare disease company, Capricor Therapeutics (Nasdaq: CAPR), has been granted an in-person Type-B meeting with the FDA to discuss chemistry, manufacturing and controls (CMC) plans for commercial launch for the potential launch of its treatment for Duchenne Muscular Dystrophy (DMD). The company believes this may enable opportunities to expedite the pathway to a Biologics License Application (BLA) submission.

Linda Marbán, Ph.D., Capricor’s Chief Executive Officer, commented, “At this time, with our pivotal Phase 3, HOPE-3 trial fully enrolled in Cohort A and top-line data expected in the fourth quarter of this year, we believe that discussing our CMC plans for potential commercialization with the FDA will allow us the opportunity to bring CAP-1002 to patients in the most expeditious manner possible for patients in need.”

Capricor’s Type-B meeting is scheduled for late March 2024.


DMD is one of the most severe forms of inherited muscular dystrophies and leads to progressive muscle fiber degeneration and weakness. The disease primarily affects boys and its onset is in early childhood.

DMD is a rare disorder but it is one of the most frequent genetic conditions affecting approximately 1 in 3,500 male births worldwide.

Capricor has been granted Regenerative Medicine Advanced Therapy (RMAT) and orphan drug designations for the use of CAP-1002 in DMD.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, CAP-1002 — an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform which is focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit, and follow Capricor on Facebook, Instagram and Twitter.

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About the Author

PRISM Rare Disease Spotlight: Capricor Therapeutics Advances Treatment for Duchenne Muscular Dystrophy

Catie Corcoran

Biotech Editor