Sarepta Therapeutics is a biotechnology company focused on pioneering innovative gene therapies for the treatment of rare genetic diseases, particularly those affecting muscle disorders
The company is dedicated to developing therapies that can enable patients with conditions like Duchenne Muscular Dystrophy to achieve improved outcomes and quality of life. With a strong emphasis on research and development, Sarepta utilizes advanced techniques such as RNA-targeted therapies and gene editing to combat genetic disorders at their source. Through its work, the company aims to transform the landscape of genetic medicine and provide hope for individuals and families affected by these challenging diseases.
Shares of biotech company Sarepta Therapeutics (NASDAQSRPT)
fell 24.9% in the pre-market session after the company reported the death of a Duchenne muscular dystrophy patient following treatment with its gene therapy, ELEVIDYS. The patient reportedly suffered acute liver failure, a known risk of AAV-mediated gene therapies. However, Sarepta suggested that a recent cytomegalovirus (CMV) infection might have worsened the condition. Despite this, the company maintained that ELEVIDYS still had a favorable benefit-risk profile. It planned to update the prescribing information and inform regulators, clinical investigators, and prescribing physicians of the incident.
Sarepta shares fell after reporting a patient death linked to Elevidys. The case involved acute liver failure, with a CMV infection as a potential factor.
Arrowhead's ARO-C3 cut C3 levels by up to 89% and proteinuria by 41% in a Phase 1/2 trial. Additional results will be presented at a 2025 medical meeting.
As the Q4 earnings season wraps, let’s dig into this quarter’s best and worst performers in the therapeutics industry, including Sarepta Therapeutics (NASDAQSRPT) and its peers.
Sarepta Therapeutics, Inc. (NASDAQSRPT), the leader in precision genetic medicine for rare diseases, today announced the official opening of Route 79, The Duchenne Scholarship Program for the 2025-2026 academic year. Academic scholarships of up to $5,000 will be awarded to as many as 20 individuals living with Duchenne muscular dystrophy and 5 siblings of individuals living with Duchenne.
Biotech company Sarepta Therapeutics (NASDAQSRPT) announced better-than-expected revenue in Q4 CY2024, with sales up 65.9% year on year to $658.4 million. Its non-GAAP profit of $1.90 per share was 7.8% below analysts’ consensus estimates.
Sarepta Therapeutics, Inc. (NASDAQSRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2024.
Solid Biosciences just saw a 32% move to the upside after its trial results impressed. What's next for this firm that analysts still see massive potential in?
Sarepta Therapeutics, Inc. (NASDAQSRPT), the leader in precision genetic medicine for rare diseases, announced today that it has closed on a $600 million senior secured revolving credit facility with a bank syndicate.
Sarepta Therapeutics, Inc. (NASDAQSRPT), the leader in precision genetic medicine for rare diseases, will report fourth quarter and full-year 2024 financial results after the Nasdaq Global Market closes on Wednesday, Feb. 26, 2025. Subsequently, at 4:30 p.m. E.T., the Company will host a conference call to discuss its fourth quarter and full-year 2024 financial results.
