Sarepta Therapeutics Faces Challenges Amid Safety Concerns

Sarepta Therapeutics, trading under the symbol NASDAQ:SRPT, is a biotechnology company focused on developing innovative genetic medicines to treat rare diseases. The company is well-known for its work on therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder. However, Sarepta faces competition from other biotech firms also working on gene therapies for similar conditions.

On June 15, 2025, BMO Capital downgraded Sarepta from an "Outperform" to a "Market Perform" rating, with the stock priced at $36.18. This downgrade coincided with significant challenges for the company. Sarepta's Elevidys gene therapy, aimed at treating DMD, has been linked to the death of a second patient, raising serious safety concerns. The tragic event has led to a suspension of Sarepta's full-year financial guidance, as reported by Angelica Peebles on CNBC Television.

The company's CEO has halted dosing and paused ongoing trials, highlighting the inherent risks in developing advanced therapies for complex conditions like DMD. This has cast doubt on the future of Sarepta's gene therapy program. The stock has seen a sharp decline, now trading at $20.29, a 43.92% drop from its previous value. The stock's fluctuation between $18.30 and $21.55 today reflects investor uncertainty. Sarepta's market capitalization is approximately $1.99 billion, with a high trading volume of 26.34 million shares, indicating significant market activity.

The recent patient death, attributed to liver failure, has overshadowed the potential of Elevidys, impacting its development and approval prospects. Sarepta's stock, which once reached a high of $173.25 over the past year, now faces a challenging path forward as it navigates these safety concerns and their implications for its gene therapy pipeline.