Agenda Details

The Accelerating Medicines Partnership Program - Bespoke Gene Therapy Consortium (BGTC), managed by the Foundation for the National Institutes of Health (FNIH), is a public-private partnership consisting of multiple NIH ICs, the FDA, and numerous bio/pharma companies and patient advocacy organizations. The goal of the BGTC is to accelerate the development of gene therapies to treat genetic diseases of no commercial interest by developing: (1) a mechanistic understanding of AAV biology and factors that influence AAV gene expression and vector production; and (2) a gene therapy playbook, which will be a public resource that leverages best-practices templates, master regulatory files, and aligned manufacturing and testing processes. This playbook approach will be stress-tested through multiple clinical trial test cases, providing insights which will streamline the regulatory process for future gene therapy development for genetic diseases of no commercial interest. This workshop at ASGCT will serve as BGTC's first annual meeting, during which we will update on our progress so far, including AAV biology, the clinical program, and playbook development, engage the community on next steps, and provide member and patient perspectives on overcoming the challenges faced in the gene therapy field.