April 9, 2026 at 10:00 am EDT You're invited to this quarterly open forum designed for those with experience or interest in N-of-1 clinical trials to engage in discussions about the institutional and logistical hurdles we face in translating these cutting-edge therapies. This forum will serve as a space to share insights, identify barriers, and explore strategies for ensuring the safe, efficient, and equitable delivery of individualized treatments to patients in need. If you have any...

February 23, 2026 Individualized medicines for patients with ultra-rare, life-threatening genetic conditions have historically been developed through singular, case by case efforts focused on one or few unique genetic variants. While this approach has enabled lifesaving treatments for individual patients, it has not generated the systematic evidence needed to support scalability, regulatory learning, or broader access to individualized therapies. This session will describe a collaborative...

Timothy W. Yu, Julia Vitarello, Kiran Musunuru, Rebecca C. Ahrens-Nicklas, David R. Liu, Michelle L. Mellion, Fyodor Urnov, Winston Yan, Srinivas Chunduru, David Barrett, Terence R. Flotte, Janet Woodcock January 7, 2026 Main text The Food and Drug Administration (FDA) recently proposed a dedicated regulatory pathway for “personalized” therapies 1 targeting genetic variation found in small numbers of individuals. This pathway is important, timely, and urgently needed. A number of such...