Linda Marbán, PhD, on Evaluating Capricor’s Cardiomyopathy Cell Therapy Deramiocel for DMD

“Most of the time people don't realize that while these boys are losing skeletal muscle function, which is visible to the world, what is really taking their lives eventually is the cardiac disease associated with Duchenne... If we can slow the progression of the aggregation of the scar, we can slow the decline in cardiac function, and we can help preserve their lives. That's exactly what Deramiocel has done.”

Although there is currently an FDA-approved gene therapy product available for Duchenne muscular dystrophy (DMD), and several other gene therapy products are in clinical development, unmet needs continue to exist for patients in the DMD community. Notably, even if gene therapy products are able to slow or stop disease progression by targeting the root genetic cause of the disease, sequelae from DMD will continue to afflict patients even after treatment.

Capricor Therapeutics is currently developing a potentially-adjuvant allogeneic cardiosphere-derived cell therapy referred to as Deramiocel (also known as CAP-1002), which is intended to address gaps in treatment for DMD. Capricor currently has a biologics license application (BLA) under review by the FDA for Deramiocel, with a Prescription Drug User Fee Act action date set for August 31, 2025. The BLA is supported by data from the completed HOPE-2 clinical trial (NCT03406780) and HOPE-2’s ongoing open-label extension (OLE) study (HOPE-2-OLE; NCT04428476). Notably, data from HOPE-2-OLE were recently presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 16-19, in Dallas, Texas.

Shortly after the conference, CGTLive sat down with Linda Marbán, PhD, the CEO of Capricor Therapeutics, to discuss the findings that were presented. Marbán explained the mechanism behind Deramiocel and how it is intended to address unmet needs in DMD, and then went over the key findings that were reported at the conference. She emphasized the demonstrated ability of Deramiocel to address cardiac symptoms of DMD, which are ultimately the main cause of death from the disease.

Read more coverage of the 2025 MDA Conference here.

REFERENCE
1. Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy. News release. Capricor Therapeutics. March 4, 2025. Accessed April 24, 2025. https://www.capricor.com/investors/news-events/press-releases/detail/305/capricor-therapeutics-announces-fda-acceptance-and-priority