Rare endpoints: Delivering on unmet patient needs

By Jamie Pierson, Director, Cell and Gene Therapy
Kim MacDonnell, Director, Rare and Genetic Diseases

Published on: May 20, 2024

The latest episode of Preparing for a Cell and Gene Future features a discussion between Jamie Pierson (Program Lead within Parexel’s Cell and Gene Center of Excellence) and Kim MacDonnell (Associate Director for Rare Diseases). They consider uses of surrogate markers as endpoints in rare disease development and look ahead to positive directions established by FDA's current initiatives.

Contributing Expert