The FDA is investigating deaths related to liver failure following administration of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy. The regulator is assessing whether further action is warranted due to safety concerns. Meanwhile, Capricor Therapeutics updated regulatory status for its cell therapy Deramiocel targeting Duchenne cardiomyopathy. Leadership upheaval at the FDA’s cell and gene therapy office, including the departure of key officials, reflects tensions around oversight of advanced therapies. These developments highlight ongoing challenges balancing innovation and patient safety in gene therapy regulation.