Rare Trials Summit

Beyond “One Disease At a Time”: Platform Approaches to Accelerate Gene Therapy Trials

• Philip J. Brooks, Ph.D., Deputy Director, Division of Rare Diseases Research Innovation, NCATS NIH

• Mathew Pletcher, PhD, Chief Scientific Officer, Weaver Biosciences

• Kiran Musunuru, MD PhD, Professor, University of Pennsylvania, and Scientific Director, Center for Inherited Cardiovascular Disease

• Sadik Kassim, PhD, Chief Technology Officer, Genomic Medicines, Danaher Corporation