Core Insights - Sarepta Therapeutics is advancing its investigational siRNA treatment SRP-1003 for Myotonic Dystrophy Type 1, with positive progress reported in the Phase 1/2 clinical study [1][2] Study Progress - Cohorts 1 (1.5 mg/kg) and 2 (3 mg/kg) of the study are complete, while cohort 3 (4.5 mg/kg) is fully enrolled and ongoing [2] - Patients are currently being dosed in cohort 4 (6 mg/kg), with plans to initiate dosing in cohort 5 (12 mg/kg) in early 2026 [3] Financial Milestones - Following a positive review by the drug safety committee, a second milestone payment of USD 200 million will be made to Arrowhead Pharmaceuticals within 60 days [2] siRNA Platform Overview - Sarepta's siRNA platform focuses on therapies for neurodegenerative and pulmonary diseases, with ongoing preclinical programs for Spinocerebellar ataxia types 1 and 3 [3] Company Mission - Sarepta is dedicated to engineering precision genetic medicine for rare diseases, holding leadership positions in Duchenne muscular dystrophy and expanding its portfolio across various disease areas [4]