VILLANOVA UNIVERSITY

FDA's Cell and Gene Therapy Champion Peter Marks Departs, Leaving Industry at Critical Juncture

2 months ago

• Peter Marks, head of FDA's Center for Biologics Evaluation and Research since 2016, has resigned, leaving cell and gene therapy developers without their biggest regulatory advocate during a challenging investment period. • Under Marks' leadership, the FDA approved dozens of cell and gene therapies including the first gene therapy, first cellular treatment for cancer, and first CRISPR gene editing medicine, establishing flexible regulatory frameworks for these novel treatments. • Despite concerns about regulatory uncertainty following Marks' departure, incoming FDA Commissioner Marty Makary has signaled support for conditional approval pathways for rare disease treatments where randomized controlled trials aren't feasible.