Gene Therapy Platform for Rare Diseases

National Center For Advancing Translational Sciences

Investigators

Linked publications & trials

Abstract

The TRND Program initiated a suite of collaborations with biotech and academic groups that were strategically selected to serve as inaugural pilot projects. The overall goal is to enable TRND to build a gene therapy translation toolbox. New technologies to scale up gene vector manufacturing and to deliver the transgene to the right tissue at the right time and dosage are among those being developed at TRND. These technologies, along with best practices to achieve regulatory approval of gene therapy, will help improve the speed of development and reduce costs for gene therapy in general. The pilot projects include preclinical development of therapies for Duchenne muscular dystrophy, Pompe disease, and aromatic L-amino acid decarboxylase (AADC) deficiency.