Sarepta Therapeutics, Inc.
NEWS
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.
Amid an unprecedented turnover in leadership at the FDA and mass layoffs of staff, communication has crumbled and uncertainty runs rampant, leaving small and medium biopharma companies without a clear path forward for their therapies.
Massachusetts biopharma workforce growth was fairly flat last year, and R&D and manufacturing employment declined, according to a new MassBio report. BioSpace data further highlight challenges facing the state, showing roughly 2,300 people out of work in 2025 and jobs live on the website falling.
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