Sarepta Therapeutics, Inc.
NEWS
A lawsuit and FDA warning ensued after Hims & Hers launched a compounded version of Novo Nordisk’s new obesity pill, more Big Pharma report earnings—including from weight loss rivals Novo and Eli Lilly—and the gene therapy space sees another rejection.
With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.
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.
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.
JOBS
IN THE PRESS