Sarepta Therapeutics
@Sarepta
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Commercial-stage biopharma company focused on the discovery & development of precision genetic medicine to treat rare neuromuscular diseases. https://t.co/HFP4txOCxe
Cambridge, MA
Joined November 2013
Today we announced a licensing and collaboration agreement with Arrowhead Pharmaceuticals that will provide Sarepta with exclusive global rights to multiple clinical, pre-clinical and discovery stage programs for rare, genetic diseases.
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News: The U.S. FDA granted Platform Technology Designation to SRP-9003, Sarepta’s investigational gene therapy for the treatment of LGMD type 2E/R4. This is one of the first programs to receive the technology designation. Read more at
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Are you in New Orleans for #ASGCT2025? Come find us at booth 1513, explore our posters and oral presentations and learn about our commitment to advancing precision genetic medicine for rare disease. Learn more here:
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Today we shared updates from our clinical programs focused on limb-girdle muscular dystrophy (LGMD) subtypes 2C/R5, 2D/R3 and 2E/R4. Read more here:
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Today we issued a safety update for our approved gene therapy. Read here:
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Excited to be here in Dallas at the 2025 @MDAorg Clinical & Scientific Conference sharing updates and connecting with the community. Come find us! #MDAconference #MDA #MuscularDystrophy
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Excited to be a partner of the 2025 @MDAorg Clinical & Scientific Conference. We’ll be there celebrating 75 years of #MDA impact & sharing updates on how Sarepta is advancing science for Duchenne and LGMD patients. #MDAconference
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On this #RareDiseaseDay, we’re excited to announce the launch of Route 79, The Duchenne Scholarship Program. We’re awarding up to 20 scholarships to individuals living with Duchenne and up to 5 scholarships to siblings. Learn more:
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Today, we released our financial results for Q4 and the full year of 2024, showing a 75% increase in net product revenue over the same quarter of the prior year. Our president and CEO on the results:
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Today we announced that enrollment & dosing is complete in Study SRP-9003-301, a Phase 3, multi-national, open-label study of investigational candidate SRP-9003 for the treatment of LGMD2E/R4. Data are expected in the first half of 2025.
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CEO Doug Ingram on Sarepta’s global licensing and collaboration agreement with Arrowhead Pharmaceuticals and how the agreement will help solidify Sarepta’s position as a genetic medicine leader.
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On our #Q3Results call, Chief Customer Officer Dallan Murray shared details on an impressive third quarter, led by the continued strength of our gene therapy launch.
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On our #Q3Results call, CEO Doug Ingram discussed our third-quarter performance and shared an update on recent corporate developments.
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We're excited to share our new therapy for Duchenne has been recognized as one of TIME’s Best Inventions of 2024! To learn more about Sarepta and our contributions to the rare disease community, visit: #TIMEBestInventions
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Are you familiar with the North Star Ambulatory Assessment (NSAA)? Watch to see how this tool is used to evaluate #Duchenne progression, the typical trajectory of the NSAA score for kids with Duchenne and what holding steady on an NSAA score may look like in real life.
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