Sarepta Therapeutics, Inc.
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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.
Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.
Of all the stories we published this year, these deep dives by BioSpace editors stand out as relevant re-reads going into the New Year.
Sarepta must also run a post-marketing study for Elevidys to better assess the risk of serious liver injury in patients dosed with the gene therapy.
Nearly two dozen life sciences companies that were awarded Massachusetts tax incentives to create and retain about 1,000 combined jobs hit just 13% of that target in 2024. Ten awardees had reported layoffs last year, including Charles River Laboratories and Moderna.
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.
Due largely to CSL, Merck and Novo Nordisk’s reorganizations that could total about 19,350 people, Q3 cuts rose significantly year over year and quarter over quarter, based on BioSpace tallies.
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene therapy: its limb-girdle muscular dystrophy candidate. But the treatment’s path forward, analysts say, is highly uncertain.
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.
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