AXO-AAV-GM2 / Roivant - LARVOL DELTA

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease (clinicaltrials.gov) - P1 | N=9 | Terminated | Sponsor: Terence Flotte | Completed ➔ Terminated; Sponsor withdrew from the study and there was a lack of funding to complete stage 2 of the designed study.

Trial termination • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease (clinicaltrials.gov) - P1 | N=9 | Completed | Sponsor: Terence Flotte | Active, not recruiting ➔ Completed | Trial completion date: Jun 2028 ➔ Dec 2024 | Trial primary completion date: Nov 2024 ➔ May 2024

Trial completion • Trial completion date • Trial primary completion date • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease (clinicaltrials.gov) - P1 | N=11 | Active, not recruiting | Sponsor: Terence Flotte | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders