Overview
- Michael J. Welsh, Jesús (Tito) González, and Paul A. Negulescu will share the $250,000 clinical prize, with awards to be presented in New York on September 19.
- Their work led to CFTR modulators including Trikafta (Kaftrio), approved in 2019 and now benefiting about 90% of people with cystic fibrosis.
- Post-approval analyses report large survival gains, including estimates near 83 years if therapy begins around age 12 and projections of rising longevity with earlier use.
- Key advances combined Welsh’s foundational CFTR discoveries with González’s fluorescence-based high‑throughput assays and Negulescu’s screening of potentiators and correctors.
- Cost and access remain barriers, with list prices near $300,000 per year, a Vertex donation pilot active in 14 countries, and roughly 10% of patients still lacking effective modulator options.