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Sarepta Therapeutics Inc (NASDAQ: SRPT) is a leader in precision genetic medicine, pioneering RNA-targeted therapies for rare neuromuscular and infectious diseases. This dedicated news hub provides investors and healthcare professionals with essential updates on SRPT's scientific advancements, regulatory milestones, and strategic initiatives.
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Key updates include FDA communications regarding novel therapies, research collaborations advancing genetic medicine, and operational developments impacting SRPT's therapeutic pipeline. All content is verified through primary sources to ensure accuracy and timeliness.
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Sarepta Therapeutics (NASDAQ:SRPT) submitted a clinical trial application to Medsafe for SRP-1005 (INSIGHTT), an investigational subcutaneous siRNA therapy for Huntington’s disease.
Pending approval, the first-in-human Phase 1 INSIGHTT study is expected to start in Q2 2026 and will enroll ~24 participants in a multi-center, dose-escalation safety and tolerability trial. Preclinical data showed protein knockdown in deep brain regions including the putamen and caudate.
Sarepta Therapeutics (NASDAQ:SRPT) said senior management will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 12, 2026 at 12:00 p.m. ET / 9:00 a.m. PT, with a Q&A starting at 12:20 p.m. ET / 9:20 a.m. PT.
The presentation will be webcast live via the Events & Presentations section of Sarepta’s investor website and will be archived for 90 days. Attendees are advised to connect to the webcast several minutes early to allow time for any required software download.
Sarepta describes its mission as engineering precision genetic medicine for rare diseases, with leadership in Duchenne muscular dystrophy and programs across muscle, central nervous system, and cardiac diseases.
Sarepta Therapeutics (NASDAQ:SRPT) granted equity awards on Dec. 31, 2025 as material inducements to employment for 10 individuals hired in Q4 2025. The awards, approved under Sarepta’s 2024 Employment Commencement Incentive Plan and in accordance with Nasdaq Listing Rule 5635(c)(4), total 55,118 restricted stock units (RSUs) granted in the aggregate. The RSUs vest 25% annually on each anniversary of the grant date and will be fully vested on the fourth anniversary, subject to continued employment. No stock options were granted to these employees.
Sarepta Therapeutics (NASDAQ:SRPT) announced privately negotiated exchanges of approximately $291.4 million principal of its 1.25% convertible senior notes due 2027 for $291.4 million principal of new 4.875% convertible senior notes due 2030 plus $31.6 million in cash.
The Exchange is expected to close on or about December 18, 2025, subject to customary conditions. After closing, aggregate principal of the 4.875% notes due 2030 will be $893.4 million, and $158.6 million of the existing 2027 notes will remain outstanding.
Sarepta (NASDAQ:SRPT) said the FDA approved dosing for Cohort 8 of the ENDEAVOR study to test an enhanced immunosuppression regimen with sirolimus in ~25 non-ambulant Duchenne patients, with dosing expected to begin before year-end 2025. The regimen uses 14 days of peri-infusion sirolimus and continues for 12 weeks after ELEVIDYS infusion. Primary endpoints are incidence of acute liver injury (ALI) and ELEVIDYS-dystrophin expression at 12 weeks. Sarepta expects primary endpoint data collection in H2 2026 and will review results with the FDA to inform commercial dosing decisions for this population.
Sarepta (NASDAQ:SRPT) reported progress in the Phase 1/2 multiple ascending dose study of SRP-1003 for myotonic dystrophy type 1. Cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) are complete; cohort 3 (4.5 mg/kg) is fully enrolled and ongoing. Following a positive pre-specified drug safety committee review, the study will continue dose escalation with patients currently being dosed in cohort 4 (6 mg/kg) and planned initiation of cohort 5 (12 mg/kg) in early 2026. Having reached the enrollment target, a second milestone payment of $200 million to Arrowhead will be made within 60 days.
Arrowhead Pharmaceuticals (NASDAQ: ARWR) announced it earned a $200 million milestone payment from Sarepta Therapeutics (NASDAQ: SRPT) after reaching the second development milestone in the Phase 1/2 study of ARO-DM1 (SRP-1003) for type 1 myotonic dystrophy.
The milestone followed a drug safety committee review, authorization to dose escalate, and attainment of a pre-specified patient enrollment target. Arrowhead said it expects to receive the payment within 60 days. The company reported cohort 4 (6 mg/kg) accrual is nearly complete and plans to begin enrollment in cohort 5 (12 mg/kg) in Q1 2026.
Sarepta (NASDAQ:SRPT) announced FDA approval of updated prescribing information for ELEVIDYS on November 14, 2025. Key label changes include a boxed warning for acute serious liver injury (ALI) and acute liver failure (ALF), removal of the non-ambulatory indication, expanded prescriber guidance with modified corticosteroid regimens, and enhanced weekly monitoring for 3 months post-infusion. The company plans to commence a study of an enhanced sirolimus immunosuppressive regimen to address ALI/ALF risk so dosing may resume for non-ambulatory patients with FDA concurrence. ELEVIDYS has been administered to over 1,100 patients globally.
Sarepta Therapeutics (NASDAQ:SRPT) will report third quarter 2025 financial results after the Nasdaq Global Market closes on Monday, Nov. 3, 2025. A conference call to discuss results and recent corporate developments will follow at 4:30 p.m. ET.
The event will be webcast live via the investor relations site at https://investorrelations.sarepta.com/events-presentations and a replay will be archived there for one year. Phone participants must register online to receive dial-in details and a personal PIN by auto-generated email.