Duchenne muscular dystrophy
After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments around the long-time Wall Street darling, and patients may soon begin looking elsewhere.
Roche’s obesity candidate achieves 22.5% weight loss in Phase II; Moderna pulls the plug on late-stage vaccine trials as Health Secretary Robert F. Kennedy’s anti-vaccine policies and rhetoric continues; and embattled gene therapy maker Sarepta announces new data in Duchenne muscular dystrophy.
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for this treatment class, Stifel analysts said.
Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial.
Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging trends” in efficacy.
The takeover of its competitor, announced Sunday, could also bring some attention to Dyne Therapeutics, which has a similar RNA-based pipeline in rare muscle diseases.
A new analyst survey suggests that doctors are still prescribing Sarepta’s Elevidys, even after a series of deaths in certain populations marred the gene therapy’s record.
Analysts at BMO Capital Markets expect the lack of other exon-44-skiping therapies to facilitate a “smooth” approval process for Avidity’s del-zota.
As AAV9 and CRISPR programs navigate safety, delivery and scalability hurdles, small molecules offer a deployable, scalable bridge, complementing genetic approaches and accelerating meaningful impact for patients with Duchenne muscular dystrophy.
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