Sarepta just shared first test results for two new medicines aimed at rare muscle diseases FSHD1 and DM1. Early data show the drugs reach high levels in muscle safely, with no serious side effects. Shares jumped.
Guggenheim starts SLDB coverage with Buy rating and $26 price target. SGT-003 gene therapy shows safer profile vs Sarepta's Elevidys, eyes accelerated FDA approval.
Dr. Vinay Prasad, head of the FDA's vaccines and biologics division, is stepping down at the end of April after a controversial year overseeing approvals for vaccines, gene therapies, and biotech products.
Major analysts like Citi, Needham, and Bank of America maintain Sell ratings on Sarepta after recent updates, citing risks to key products like Elevidys, lower 2026 outlook, and regulatory concerns in Duchenne muscular dystrophy treatments.
Sarepta Therapeutics reports positive three-year results for Elevidys gene therapy in Duchenne patients, showing 70% slowing of disease progression and sustained motor function improvements, boosting investor confidence.
Nuvation Bio and Sarepta shares tumble on disappointing preliminary results while Adaptive Biotechnologies surges on strong revenue beat.
