Linda Marbán, PhD, on the Future of Capricor’s Cardiomyopathy Cell Therapy Deramiocel

“Many people think ‘okay, this is a mutation and so we just have to fix the mutation and then we fix the disease—gene therapy, yay, done, out the door.’ Not so. Until this disease can be fixed intrauterine where every single cell that the person is born with is normal, they will always have the sequelae associated with the disease.”

Capricor Therapeutics’ Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy, is being developed as a potential adjunctive therapy for patients with Duchenne muscular dystrophy (DMD). It has previously been evaluated in the HOPE-2 clinical trial (NCT03406780), and it is continuing to be evaluated in an ongoing open-label extension (OLE) study for HOPE-2 (HOPE-2-OLE; NCT04428476). Data from these trials support a biologics license application that is currently under review by the FDA. At the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, held March 16-19, in Dallas, Texas, data from HOPE-2-OLE were presented by the company.

Following the conference, CGTLive® spoke with Linda Marbán, PhD, the CEO of Capricor Therapeutics, to ask about the big picture implications of the new data and future plans for the therapy. The discussion also touched upon whether any challenges came up in the trial so far.

Marbán pointed out that although currently approved and investigational gene therapy approaches aim to correct the root mutation causing DMD, these approaches can't fully eliminate the disease's long-term effects like inflammation and fibrosis. In fact, patients with DMD will always experience sequelae until a method is developed that can correct the mutation in every cell in utero. As such, Deramiocel can serve as an adjunctive therapy to help address these sequelae in patients who have received genetic treatments for DMD. Furthermore, although Deramiocel is currently being developed for DMD cardiomyopathy, Marbán noted that it may eventually be evaluated for other cardiomyopathies such as Becker cardiomyopathy. She also touched on the safety profile of Deramiocel, emphasizing its strong track record in 700 infusions across 8+ years.

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