AskBio’s Gene Therapy Gets Fast Track

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.

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill an unmet medical need. This designation includes more frequent meetings with the FDA to discuss development and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.