LION-101 / Bayer - LARVOL DELTA

AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort (Bayer Press Release) - "AskBio...announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with dosing of the first participant in the second cohort. The recommendation to advance to the second cohort followed the completion of a Data Safety Monitoring Board (DSMB) review of trial recruitment activity, and safety reporting from the first cohort that determined it was safe to proceed to cohort two....The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation clinical trial to evaluate the safety of AB-1003 gene therapy in adult participants (18–65 years) who have genetic confirmation of LGMD2I/R9."

DSMB • Trial status • Genetic Disorders • Muscular Dystrophy

LION-CS101: A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: Asklepios Biopharmaceutical, Inc.

Gene therapy • Trial completion date • Trial primary completion date • Gene Therapies • Muscular Dystrophy

LION-CS101: A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) (clinicaltrials.gov) - P1/2 | N=10 | Recruiting | Sponsor: Asklepios Biopharmaceutical, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Gene Therapies • Muscular Dystrophy

AskBio Receives European Commission Orphan Drug Designation through its EU-Based subsidiary BrainVectis for AB-1003, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy (LGMD) (PRNewswire) - "Asklepios BioPharmaceutical...announced today that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101)* for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is a novel investigational recombinant adeno-associated virus (AAV) based gene therapy currently being developed as a one-time intravenous (IV) infusion for the treatment of patients with LGMD type 2I/R9 (LGMD2I/R9), a disease subtype affecting 4.5 people per million worldwide, including more than 5,000 people in the EU and US. The EC decision, dated February 15, 2023, follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) and was received through AskBio's EU-based subsidiary BrainVectis."

European regulatory • Genetic Disorders • Muscular Dystrophy