LGMD is a Rare Form of Muscular Dystrophy with No Approved Therapy RESEARCH TRIANGLE PARK, N.C., Feb. 27, 2023 /PRNewswire/ Asklepios BioPharmaceutical, Inc. (AskBio), a wholly
AskBio Receives FDA Fast Track Designation for LION-101, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) prnewswire.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from prnewswire.com Daily Mail and Mail on Sunday newspapers.
Share this article
Share this article
RESEARCH TRIANGLE PARK, N.C., May 25, 2021 /PRNewswire/ Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced that the U.S. Food & Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LION-101 to proceed in a Phase 1/2 clinical study. LION-101 is a novel recombinant adeno-associated virus (rAAV) based vector being developed as a one-time intravenous infusion for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).
LION-101 will be evaluated in a Phase 1/2 multicenter study to evaluate a single intravenous (IV) infusion in adult and adolescent subjects with genotypically confirmed LGMD2I/R9. AskBio plans to initiate dosing for the LION-101 Phase 1/2 clinical study in the first half of 2022.