Cell and Gene Therapy Summit

Approximately 10,000 diseases are classified as “rare,” each affecting fewer than 200,000 people in the United States. Yet, while each rare disease affect a small number of individuals, collectively, they impact millions in the U.S., and can pose significant health and financial challenges.

The Bespoke Gene Therapy Consortium (BGTC), part of the Accelerating Medicines Partnership (AMP), was founded in 2021 to advance gene therapy development for rare diseases that currently lack treatment options. The aim of this public-private collaboration, led by the NIH, FDA, and multiple stakeholders, is to streamline the development, manufacturing, and regulatory pathways for customized gene therapies. This session will provide an update on BGTC's progress to improve the efficiency and accessibility of gene therapy development and delivery for rare diseases, and attendees will learn the strategies being developed to support FDA approval of gene therapies for rare and ultra-rare diseases. Updates will include financial incentives driving gene therapy advancements, such as how new FDA designations for NCATS rare disease therapies may encourage treatment development, and how automation and standardization can reduce cell and gene therapy production costs.