AXO-AAV-GM1 / Roivant - LARVOL DELTA

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human <=-Galactosidase in Type II GM1 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=45 | Recruiting | Sponsor: National Human Genome Research Institute (NHGRI) | Trial completion date: Apr 2026 ➔ Dec 2027 | Trial primary completion date: Apr 2024 ➔ Dec 2027

Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases • Rare Diseases