Issues, Ideas, And Initiatives: Proposals From The DIA/Harvard-MIT CRS Cell And Gene Therapy Executive Roundtable

By Fouad Atouf, USP; Tamei Elliott, DIA; Susan Awad, Sanofi; Min Lin, Chenghong Wei, and Yao-Yao Zhu, AstraZeneca; Ryan Soderquist, Bristol Myers Squibb; Megan Canniere, Spark Therapeutics, Peter Marks and Nicole Verdun, FDA; James Wabby, AbbVie, Daniel Cushing, Carisma Therapeutics; and Janice Watch, UCB

As of May 2024, the U.S. has approved 36 cellular and gene therapy products, offering unprecedented solutions for diseases where traditional methods fall short. However, these innovations bring unique challenges in development and commercialization. Director Peter Marks of the FDA Center for Biologics Evaluation and Research (CBER) highlighted manufacturing and regulatory challenges during the DIA/Harvard-MIT CRS Executive Roundtable in January 2024.

This collaborative effort aims to address key hurdles, especially for rare disorders. Recent FDA guidance documents underscore the importance of these therapies, providing insights for their development. The roundtable, convened by DIA with input from Harvard-MIT CRS, assessed regulatory, clinical, and manufacturing challenges, seeking solutions to streamline the pathway for cell and gene therapies.