Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, was granted Fast Track Designation from the FDA for the LION-101 gene therapy program. LION-101 is a novel recombinant adeno-associated virus (rAAV) based vector being developed as a one-time intravenous infusion for the treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).
The FDA recently cleared an Investigational New Drug (IND) application for LION-101, authorizing the start of a Phase 1/2 multicenter study which will evaluate the safety, tolerability, and efficacy of LION-101 in adult and adolescents with genotypically confirmed LGMD2I/R9. AskBio plans to initiate dosing in the first half of 2022.
