Sarepta Therapeutics

The FDA granted accelerated approval in June 2023 for Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy specifically targeted at Duchenne Muscular Dystrophy for non-ambulatory patients aged four to six.

Sarepta's late-stage study of casimersen and golodirsen for Duchenne muscular dystrophy failed to achieve statistical significance on the primary endpoint after nine years of testing in 225 boys.

Peter Marks, former director of the FDA's Center for Biologics Evaluation and Research, has joined Eli Lilly as senior vice president of molecule discovery and head of infectious disease work.

Sarepta Therapeutics exchanged approximately $700 million of 1.25% convertible notes due 2027 for $602 million in new 4.875% notes due 2030, extending debt maturity by three years.

Kriya Therapeutics has secured $313 million in new funding to advance its pipeline of single-dose gene therapies targeting chronic conditions including geographic atrophy and trigeminal neuralgia.

Schrödinger has terminated development of its CDC7 inhibitor SGR-2921 after two patient deaths in a Phase I trial for relapsed/refractory acute myeloid leukemia and high-risk myelodysplastic syndromes.

Dr. Vinay Prasad resigned from his FDA leadership position after less than three months following criticism from right-wing activists and pressure from the White House.

Arrowhead Pharmaceuticals earned a $100 million milestone payment from Sarepta Therapeutics after reaching enrollment targets for ARO-DM1, an RNAi therapeutic for type 1 myotonic dystrophy.

The FDA has appointed Dr. George Tidmarsh, a former pharmaceutical executive and cancer specialist, to direct the Center for Drug Evaluation and Research, the agency's largest division responsible for reviewing drug safety and effectiveness.

Sarepta Therapeutics announced a $400 million annual cost-savings initiative in July 2025, including a 36% workforce reduction of 500 employees to achieve financial sustainability through 2027.