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Biotech / Medical : Immunomedics (IMMU) - moderated
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From: ghettogoulash10/11/2025 5:31:30 PM
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SRPT: I was unaware of this. Sarepta is co-developing another promising, late stage microdystrophin gene therapy for DMD (GNT0004) with Genethon, a non-profit research organization in France. Sarepta owns worldwide rights, excluding Europe. Data from the first 5 patients in Phase 1/2 is impressive (See link), and a global Phase 3 has already begun enrollment. GNT0004 is a lower-dose, and theoretically, much safer AAV, perhaps best suited for non-ambulatory patients. Ostensibly, it might also be utilized as a follow-on treatment to Elevidys, assuming it does not prove to be a superior therapy altogether.

biospace.com
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