Capricor Therapeutics experienced a major setback after the FDA rejected its cell therapy, Deramiocel, for treating heart disease in Duchenne muscular dystrophy (DMD) patients. The decision led to a 30% drop in Capricor’s stock and highlighted the need for more efficacy data. Deramiocel, which uses cardiac-derived stem cells, aims to regenerate heart tissue and slow cardiomyopathy progression in DMD. Capricor plans to resubmit its application with additional clinical data lat

Ultragenyx has received an FDA rejection for its experimental gene therapy targeting Sanfilippo syndrome, a rare and fatal pediatric neurodegenerative disorder. The FDA cited concerns over efficacy data, delaying approval for the treatment. The decision is a major setback for families awaiting a one-time gene therapy option to address the root cause of this rare disease. Ultragenyx plans to work with regulators to resolve the issues and continue advancing its gene therapy pip