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...

The Economist January 19, 2026 Mila makovec was ten in 2021 when she died from an ultra-rare neurodegenerative disorder. But, though it had not saved her, she was nevertheless in the history books as the first to receive a drug designed for a single patient. And her story did not end there. After her death her mother, Julia Vitarello, set out to change how drugs are made, so that others with obscure genetic faults could have bespoke treatments more quickly and easily. Ultra-rare disorders are...