ASP2016 / Astellas - LARVOL DELTA

Pharmacokinetic, Pharmacodynamic, and Toxicokinetic Analysis of an rAAV8 Gene Therapy for Friedreich Ataxia-Associated Cardiomyopathy (ASGCT 2025) - "A 20-fold therapeutic window for ASP2016 was established based on the observed efficacy at the lowest dose of 1E12 vg/kg in FA-MCK mice, and the lowest observed adverse effect level without unacceptable toxicities at 2E13 vg/kg in NHPs. These results identify ASP2016 as a promising clinical candidate for treating cardiac manifestations in Friedreich ataxia patients. Disease Focus of Abstract:Neuromuscular Disorders"

Gene therapy • PK/PD data • Ataxia • Cardiomyopathy • Cardiovascular • CNS Disorders • Friedreich ataxia • Gene Therapies • Movement Disorders • FXN

A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia (clinicaltrials.gov) - P1 | N=14 | Suspended | Sponsor: Astellas Gene Therapies | Recruiting ➔ Suspended

Trial suspension • Ataxia • Cardiomyopathy • Cardiovascular • Friedreich ataxia • Heart Failure • Movement Disorders

A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia (clinicaltrials.gov) - P1 | N=14 | Recruiting | Sponsor: Astellas Gene Therapies | Not yet recruiting ➔ Recruiting

Enrollment open • Ataxia • Cardiomyopathy • Cardiovascular • Friedreich ataxia • Heart Failure • Movement Disorders

A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia (clinicaltrials.gov) - P1 | N=14 | Not yet recruiting | Sponsor: Astellas Gene Therapies

New P1 trial • Ataxia • Cardiomyopathy • Cardiovascular • Friedreich ataxia • Heart Failure • Movement Disorders