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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that it has entered into separate, privately negotiated exchange agreements with a limited number of holders of its 1.25% convertible senior notes due 2027 (the “Existing Convertible Notes”). Pursuant to the exchange agreements, the Company wil...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to evaluate the use of an enhanced immunosuppressive regimen as part of treatment with ELEVIDYS (delandist...
PASADENA, Calif.--(BUSINESS WIRE)---- $arwr--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has earned a $200 million milestone payment from Sarepta Therapeutics (NASDAQ: SRPT). The milestone was earned when Arrowhead achieved the second development milestone event in a Phase 1/2 clinical study of ARO-DM1, also called SRP-1003, an investigational RNA interference (RNAi) ...
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared progress in the Phase 1/2 multiple ascending dose (MAD) clinical study of SRP-1003 (formerly ARO-DM1), an investigational small interfering RNA (siRNA) therapeutic for the treatment of type 1 myotonic dystrophy (DM1). Cohorts 1 (1.5 mg/kg) and 2 (...
Intellia Therapeutics and Sarepta Therapeutics have hit serious setbacks potentially linked to their treatments. Both biotechs have lost market value as a result, but both still seem too risky to consider investing in right now.
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an update to the prescribing information for ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD). As previously disclosed, the ELEVIDYS label now includes several key updates, inclu...
The U.S. Food and Drug Administration said on Friday it approved new labeling for Sarepta Therapeutics' gene therapy Elevidys that includes its most serious safety warning and restricts use of the treatment to walking patients with Duchenne muscular dystrophy.
Sarepta Therapeutics, Inc. faces uncertainty after Elevidys' revenue decline and ESSENCE trial failure, leading to a 25% SRPT stock drop. SRPT's PMO franchise remains its cash engine, but regulatory reliance on real-world evidence and FDA unpredictability add risk to the investment thesis. Label contraction for Elevidys and margin pressures weigh on near-term outlook, with upside catalysts most...
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