Sarepta Shares Rise As Company Begins Enrollment In Gene Therapy Safety Enhancement Study

• Sarepta is looking to enroll about 25 Duchenne muscular dystrophy patients who are non-ambulatory in the cohort.
• Elevidys came under scrutiny last year over concerns of acute liver failure and associated patient deaths.
• While the company resumed shipments to ambulatory patients after a temporary pause in July 2025, it is now working to address the safety concerns in patients who cannot walk.

Shares of Sarepta Therapeutics, Inc. (SRPT) edged up nearly 1.5% on Monday after the company said that screening and enrollment are underway in a cohort of its study, aimed at developing an enhanced safety protocol for its gene therapy, Elevidys, in non-ambulant individuals with Duchenne muscular dystrophy.

Data from the cohort is aimed at determining whether administering sirolimus prior to and after Elevidys infusion can help reduce risks of acute liver injury and acute liver failure associated with Elevidys in DMD patients who cannot walk.

Gene Therapy Study Details

Sarepta is looking to enroll about 25 Duchenne muscular dystrophy patients who are non-ambulatory in the cohort.

Patients enrolled in the cohort will be administered peri-infusion sirolimus 14 days before Elevidys administration and will continue to receive it for 12 weeks after Elevidys, the company said.

Elevidys is the only approved gene therapy for Duchenne, a severe, progressive genetic muscle-wasting disease, primarily affecting young boys.

Elevidys came under scrutiny last year over concerns of acute liver failure and associated patient deaths. While the company resumed shipments to ambulatory patients after a temporary pause in July 2025, it is now working to address the safety concerns in patients who cannot walk.

Street Consensus

Last week, Mizuho raised the firm's price target on Sarepta to $31 from $26 and kept an ‘Outperform’ rating on the shares, while Citi raised the its price target on Sarepta to $9 from $8 and kept a ‘Sell’ rating on the shares.

According to data from Koyfin, the average 12-month price target on the stock is $20.61, representing a potential upside of about 24% from current trading levels, as of the time of writing.

How Did Stocktwits Users React?

On Stocktwits, retail sentiment around SRPT stock rose from ‘bearish’ to ‘neutral’ territory over the past 24 hours, while message volume remained at ‘low’ levels.

SRPT stock has fallen nearly 84% over the past 12 months. 

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