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With Sarepta’s gene therapy Elevidys now available to a majority of Duchenne muscular dystrophy patients, experts express cautious optimism while emphasizing the need for further data.

Not exactly known for its dealmaking, Sarepta Therapeutics has thrown down a massive wad of cash to work with Arrowhead Pharmaceuticals on RNAi-based medicines.

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for 2026—potentially posing a threat to Sarepta’s Elevidys.

The investigational therapy, vesleteplirsen, had been positioned as an updated version of Sarepta’s original exon 51-skipping Duchenne muscular dystrophy drug Exondys 51.

With Elevidys expansion in hand, Sarepta commits up to $10 billion to develop short interfering RNA–based drugs to build out its pipeline.

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, granted equity awards on June 28, 2024 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta’s 2024 Employment Commencement Incentive Plan, as a material inducement to employment to 23 individuals hired by Sarepta in June 2024.

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, granted equity awards on May 31, 2024 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta’s 2024 Employment Commencement Incentive Plan, as a material inducement to employment to 9 individuals hired by Sarepta in May 2024.

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, granted equity awards on April 30, 2024 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta’s 2024 Employment Commencement Incentive Plan, as a material inducement to employment to 24 individuals hired by Sarepta in April 2024.

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the first quarter 2024.