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SAR446268

Development stage: Phase 2
Lead developer: Sanofi
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Intravenous

Overview

SAR446268 is an investigational adeno-associated viral vector-mediated gene therapy being developed by Sanofi for the treatment of myotonic dystrophy type 1 (DM1). The therapy is designed to knock down dystrophia myotonica protein kinase (DMPK) messenger RNA (mRNA) levels, aiming to improve neuromuscular function in affected individuals. It is administered as a single intravenous dose and is currently being evaluated in a Phase 1/Phase 2 open-label, multicenter clinical trial involving participants aged 10 to 50 years with non-congenital DM1.

Other names: AAV-amiRDMPK

Targets

CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)

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