Abstract
The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.
The main objective of this trial is to evaluate the safety, tolerability, and pharmacodynamic activity of BBP-812, an investigational AAV9-based gene therapy, in pediatric participants with Canavan disease.
Stanley Manne Children's Research Institute
Louis A. Simpson and Kimberly K. Querrey Biomedical Research Center
303 East Superior Street
Chicago, IL 60611
