On August 20 and 21, 2018, the National Center for Advancing Translational Sciences (NCATS) and the FDA Center for Biologics Evaluation and Research (CBER) will host the jointly-sponsored The Growing Promise of Gene Therapy Approaches to Rare Diseases workshop to promote the efficient development of regulatory science pathways for gene therapies.

Gene-based therapies are a promising and cutting-edge approach for treatment of rare diseases. This joint meeting will facilitate discussion among stakeholders (NIH and FDA staff, academics, researchers, biotech- and pharma-industry and patient group representatives) on overcoming bottlenecks in the development of gene-based therapies. 

The agenda includes sessions on pre-clinical and clinical development; manufacturing issues; transitioning therapies from academia to biotech to pharma; and the challenges in making these therapies a reality for those affected by the approximately 7,000 rare diseases for which there is no effective treatment. 

U.S. Food and Drug Administration