Apertura Gene Therapy Supports the Broad Institute in Development of Gene Therapy for Prion Disease Using Engineered AAV Capsid Targeting TfR1 for CNS Delivery

The prion program utilizes Apertura’s preclinical development data with an AAV capsid targeting human Transferrin Receptor 1 (TfR1) to provide a roadmap for rapid advancement to the clinic Unique gene therapy approach will utilize innovative technologies of the CHARM epigenetic editing…