One-year KYSA-8 and KYSA-6 data showed durable miv-cel benefit in SPS and gMG, supporting Kyverna's rolling BLA submission targeted for Q4 2026.
News|Articles|September 14, 2025
Duchenne Action Month 2025: Looking Back at News and Expert Insights
Author(s)Noah Stansfield
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.
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According to Parent Project Muscular Dystrophy, Duchenne muscular dystrophy, which causes progressive muscle weakness, constitutes the most common form of muscular dystrophy in children. The condition primarily affects boys, with around 1 in 5,000 boys currently living with DMD. Although no cure for DMD is available, a number of new transformative treatment options are currently under development or have recently reached commercialization.
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FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, for pediatric patients with Sanfilippo syndrome type A, based on cognitive data from an open-label trial in children 2 to 5 years old.
Novartis suspended several autoimmune and neurology trials of rap-cel after 3 patient deaths linked to immune effector cell-associated hemophagocytic syndrome, and BMS separately paused zola-cel studies after observing inflammatory safety events.
The FDA placed a new clinical hold on REGENXBIO's RGX-121 for Hunter syndrome after asymptomatic spine MRI findings were identified in 5 trial participants, the gene therapy's second regulatory setback this year.
The FDA extended its review of deramiocel for Duchenne muscular dystrophy to November 22, 2026, after accepting additional HOPE-3 data as a major BLA amendment.
PTC Therapeutics wins Sangamo's BLA-stage Fabry disease gene therapy ST-920 for $111 million in a bankruptcy auction, with STAAR data supporting accelerated approval.
FDA grants Kyverna's miv-cel RMAT designation in non-active secondary progressive MS, based on Phase 1 data showing CAR T expansion and B-cell reconstitution.
FDA advisory committee votes 9-3 against deramiocel's effectiveness for Duchenne cardiomyopathy; PDUFA decision due August 22, 2026.
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