Sarepta Therapeutics Inc. (SRPT), a pioneer in precision genetic medicine targeting rare diseases, has experienced a surge in after-hours trading, signaling a wave of optimism within the biopharmaceutical industry.
The company’s shares ended the day at a commendable $119.00, climbing by $3.36 per share. This jump reflects a 2.91% increase, suggesting that investors are resonating positively with Sarepta’s latest strides and its outlook.
The company witnessed a trading volume of 504.47K, which emphasizes significant investor activity and could presage the company’s market performance when the bell rings on the next trading day. This development is expected to draw considerable attention from biotech market analysts.
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In the latest news, on January 16, 2024, Sarepta announced the commencement of patient screening in its Phase 3 study, SRP-9003-301, also referred to as EMERGENE. This international, open-label study is evaluating the efficacy of SRP-9003 (bidridistrogene xeboparvovec) in treating limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), or beta sarcoglycanopathy. EMERGENE aims to include 15 participants of various mobility levels, aged four years and above, and utilizes a commercial-grade SRP-9003 treatment.
Furthermore, a webinar intended for the limb-girdle muscular dystrophy community is in the pipeline, with more information to be announced later. SRP-9003, the investigational gene therapy in question, utilizes the AAVrh74 vector for systemic delivery to skeletal, diaphragm, and cardiac muscles. Given the critical nature of cardiac expression for patients with LGMD2E—who often face life-threatening pulmonary or cardiac issues—SRP-9003 is engineered to transmit a full-length beta-sarcoglycan transgene, with the MHCK7 promoter enhancing heart muscle expression.
The EMERGENE study, officially named SRP-9003-301, is a landmark Phase 3 trial involving participants across the globe. This study features a six-month natural history lead-in, with the main goal being the expression of beta-sarcoglycan 60 days post-dosing. It will also evaluate various functional measures over a 60-month period, alongside monitoring safety parameters.
