AAV8 gene therapy

Study Design and Endpoint Selection to Evaluate Safety, Preliminary Efficacy, and Dose Selection of AAV8 Gene Therapy in Patients with Infantile and Late Onset Pompe Disease (IOPD and LOPD)

Sal Rico
VP Clinical Development

July 2018

This is a proposal from Audentes Therapeutics to treat Pompe disease using gene therapy. The proposal aims to evaluate safety, preliminary efficacy, and dose selection in patients with infantile and late onset Pompe disease (IOPD and LOPD). The proposal is based on promising preclinical results which have shown improved muscle pathology and proof of concept.

The committee felt this was a well-planned clinical trial proposal, and made recommendations including those regarding further pre-clinical studies, safety considerations, control groups and end-point selection. Inclusion of patients in the study design was also supported strongly.

17 Dec 2018