Overview

• Michael Welsh, Jesús (Tito) González, and Paul Negulescu were named winners of the 2025 Lasker-DeBakey Clinical Medical Research Award on Thursday, sharing a $250,000 prize.
• Their complementary work—mechanistic insights, real-time ion-flow screening, and large-scale compound discovery—led to CFTR modulators culminating in the triple therapy Trikafta (Kaftrio), cleared by the FDA in 2019.
• Use of Trikafta has reduced lung transplants and infection-related hospitalizations and is associated with near-normal lifespan when started early, with a 2023 study estimating life expectancy of about 83 years if begun around age 12.
• The World Health Organization designated these modulators essential in 2025, yet access remains constrained by a list price near $300,000 per year and the need for lifelong treatment.
• Vertex says roughly two-thirds of diagnosed patients worldwide are on its therapy, and it has launched a pilot donation program in 14 countries, while a minority with certain mutations still lack effective options.