Scribe Therapeutics Inc., another CRISPR-based genome editing firm out of the Jennifer Doudna stable, launched with $20 million in series A funding and a deal with Biogen Inc. in amyotrophic lateral sclerosis (ALS), which brings in another $15 million up front and up to $400 million in development and commercial milestones.
Both Solid Biosciences Inc. and Pfizer Inc. got lifts to their Duchenne muscular dystrophy (DMD) programs from the FDA as the agency released a clinical hold and awarded a fast track designation to their respective adeno-associated viral (AAV) programs.
PERTH, Australia – Antisense Therapeutics Ltd. saw its stock bounce 26% following the news that the FDA has granted rare pediatric disease designation for ATL-1102 for the treatment of Duchenne muscular dystrophy (DMD), following submission of phase II data that showed its immunomodulatory therapy met primary disease progression endpoints.
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