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

Biogen’s investigational drug, Salanersen, shows strong potential as a once-yearly treatment for spinal muscular atrophy (SMA), even after gene therapies like Zolgensma. Early trial results reveal slowed neurodegeneration and improved motor milestones in children. With reduced neurofilament levels and notable functional gains, Salanersen is heading directly into Phase 3 trials, signaling a possible breakthrough in SMA care and a promising alternative to existing therapies lik

Sarepta Therapeutics has paused its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, after a second patient death due to acute liver failure. The pause affects ongoing trials and therapy shipments, raising safety concerns—especially for non-ambulatory patients. Approved under the FDA’s accelerated pathway, Elevidys aimed to slow DMD progression, but recent events have sparked debate over gene therapy risks, regulatory oversight, and treatment decisions for affected f

In a historic first, doctors used base editor gene therapy to treat a baby with CPS1 deficiency, a rare genetic liver disorder. The personalized treatment—delivered using lipid nanoparticles—led to improved ammonia levels and reduced medication needs. Unlike traditional CRISPR, base editing made precise DNA changes without cutting, offering a safer, more targeted option. This breakthrough marks a major step forward in treating rare diseases with gene-editing technology.