NATIONAL INSTITUTE OF HEALTH (NIH), BESPOKE GENE THERAPY CONSORTIUM (BGTC)P3 BCC TO# 009 NCATS/ORDR

Black Canyon Consulting Llc, Fairfax VA

Investigators

Abstract

The Accelerating Medicines Partnership® (AMP®) program is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and multiple public and private organizations. Managed through the Foundation for the NIH (FNIH), the AMP program aims to improve our understanding of therapeutically relevant biological pathways and validate information that could be relevant for the development of multiple therapeutics. The AMP Bespoke Gene Therapy Consortium (BGTC) aims to develop platforms and standards that will speed the development and delivery of customized or ‘bespoke’ gene therapies that could treat the millions of people affected by rare diseases. Launched in October 2021, BGTC is the first AMP initiative focused on rare diseases and the sixth AMP initiative overall. The effort aims to overcome major obstacles related to developing gene therapies and will create a gene therapy protocol book that the research community can use to make the process of developing gene therapies for rare conditions much more efficient.