A recent gene therapy death shines a light on AAV safety [Yahoo! Finance]
Sarepta Therapeutics, Inc. (SRPT)
Last sarepta therapeutics, inc. earnings: 2/26 04:05 pm
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Source: Yahoo! Finance
The death of a 16-year-old boy taking a gene therapy this month dealt a fresh blow to the Duchenne muscular dystrophy community. The patient died from acute liver failure months after starting Elevidys , the only FDA-approved gene therapy for DMD. Its maker, Sarepta Therapeutics, hasn't ruled out the possibility that the treatment, which uses an adeno-associated virus vector to deliver its therapeutic gene payload, may have contributed to the fatality. “The Elevidys patient death was due to liver failure, and liver injury is a known side effect of AAV gene therapy. But the investigation is still underway. We should know more in a few months,” Sharon Hesterlee, chief research officer at the Muscular Dystrophy Association, said in an email to PharmaVoice. The teen also had a cytomegalovirus infection, which may have played a role in the outcome, according to Sarepta. Regardless of what the investigation ultimately reveals, the tragedy raises questions about the safety of gene t
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News
- Sarepta Therapeutics, Inc. (SRPT): A Bull Case Theory [Yahoo! Finance]Yahoo! Finance
- A mom was overjoyed when she learned a drug could save her baby's life. Then she learned it cost $2 million. [CBS News]CBS News
- Breakthrough genetic therapies can save lives, but many cost millions for single dose [CBS News]CBS News
- Sarepta Therapeutics Announces Refinancing of Approximately $291 Million of 1.25% Convertible Senior Notes due 2027Business Wire
- Sarepta Therapeutics (SRPT): Reassessing Valuation After a Sharp One-Month Share Price Rebound [Yahoo! Finance]Yahoo! Finance
SRPT
Earnings
- 11/3/25 - Beat
SRPT
Sec Filings
- 12/19/25 - Form 4
- 12/19/25 - Form 8-K
- 12/18/25 - Form 4
- SRPT's page on the SEC website