Sarepta Therapeutics @Sarepta
Commercial-stage biopharma company focused on the discovery & development of precision genetic medicine to treat rare neuromuscular diseases. https://t.co/HFP4txOCxe sarepta.com Cambridge, MA Joined November 2013-
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Read the statement at Sarepta.com.
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 Sarepta.com.
Attending #ASGCT2025? Explore our posters and oral presentations. Learn more here: annualmeeting.asgct.org
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: annualmeeting.asgct.org
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: bit.ly/3G9VEUV
Today we issued a safety update for our approved gene therapy. Read here: bit.ly/4iqEekG
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
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: bit.ly/4khBJTs
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:
Today at #JPM25, CEO Doug Ingram discussed our preliminary Q4 and full-year 2024 performance and shared an update on corporate developments.
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.
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.
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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