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In observance of Duchenne Action Month and World Duchenne Day, held annually in September, we took a look back at recent news in gene and cell therapy for DMD.

Review top news and interview highlights from the week ending September 12, 2025.

Capsida has made the move to pause the trial voluntarily.

The company specifically highlighted that one patient who was treated in the trial’s first cohort is still alive at 28 months.

According to REGENXBIO, HS D2S6 is “a key biomarker of MPS II brain disease that is reasonably likely to predict clinical benefit.”

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The findings come from patients with DMD44 treated in the phase 1/2 EXPLORE44 clinical trial and the phase 2 EXPLORE44OLE clinical trial.

Capricor stated that it was not notified in advance by the FDA that the latter would be publishing the CRL publicly on its website.

Robert Alexander Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, expressed optimism for RNA therapy's future.

Mind Moments, a podcast from our sister site Neurology Live, held an exclusive interview with Barry Byrne, MD, PhD.

Alex Wesselhoeft, PhD, the director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute, discussed the Institute's work in exploring the new modality.

Review top news and interview highlights from the week ending September 5, 2025.

The director of RNA Therapeutics at MGB’s Gene and Cell Therapy Institute discussed the Institute's work in exploring the new modality.