Vertex Scientists Honored with Prestigious Lasker~DeBakey Award for Transformational Advances in Cystic Fibrosis
Vertex Pharmaceuticals Incorporated announced today that Dr. Paul Negulescu, Senior Vice President, has been named a recipient of the 2025 Lasker~DeBakey Clinical Medical Research Award. Dr. Negulescu is being recognized “for the development of a novel, life-saving treatment for cystic fibrosis (CF) — namely, a triple-drug combination therapy, TRIKAFTA®, that has helped countless people with this genetic disease.”
He shares the honor with two other pioneers: Jesús (Tito) González, a former Vertex scientist, and Dr. Michael Welsh, Professor of Internal Medicine specializing in Pulmonary, Critical Care, and Occupational Medicine at the University of Iowa. Together, their discoveries have reshaped the scientific and medical landscape for CF, offering new hope to patients and families worldwide.
Recognizing Decades of Breakthrough Research
The award highlights the groundbreaking work of Dr. Negulescu, Dr. González, and the broader Vertex research team, who were instrumental in discovering and developing the first approved medicines that directly target the root cause of CF. Until recently, treatment for CF largely focused on managing symptoms. Vertex’s approach shifted the paradigm by developing therapies that correct the malfunction of the CFTR protein, which is defective in people with CF.
To date, Vertex has successfully delivered five transformational therapies to patients. Among them, TRIKAFTA®, approved in 2019, has had the most far-reaching impact, extending treatment to approximately 90% of the CF population. More recently, the approval of ALYFTREK® has further expanded access, underscoring the company’s sustained commitment to innovation in CF care.
A Collective Achievement
Reflecting on the recognition, Dr. Negulescu emphasized that the award represents the collective effort of countless researchers over decades:
“I’m honored to represent my co-leaders on the CF program, Fred Van Goor and Sabine Hadida, and the more than a thousand people at Vertex who have worked over two decades to make the impossible possible. Together, we’ve brought five medicines including TRIKAFTA and now ALYFTREK® to tens of thousands of people with CF around the world — medicines that have fundamentally changed the course of this disease. This award recognizes that collective effort and serves as a reminder of the work ahead in reaching all people with CF.”
Scientific Milestones in CF
This year’s awardees embody the trajectory of modern CF research — from uncovering the cellular and genetic mechanisms that drive the disease, to translating those insights into first-in-class, FDA-approved medicines that alter its natural history. The journey from bench to bedside has not only delivered life-saving therapies but also laid the foundation for future advances aimed at addressing the unmet needs of those who remain without effective treatment.
About the Lasker Awards
The Lasker Awards, founded in 1945 by biomedical research advocates Mary and Albert Lasker, are among the most prestigious recognitions in medicine and science. Often called “America’s Nobel,” the Lasker~DeBakey Clinical Medical Research Award honors contributions that have improved the clinical treatment of disease and elevated patient care.
Over the past 80 years, the Lasker Foundation has recognized more than 400 scientific leaders. Remarkably, 101 Lasker Laureates have subsequently gone on to receive the Nobel Prize, including 13 since 2018 — a testament to the awards’ role in spotlighting transformative achievements in biomedical research.
This year’s recognition of Vertex scientists underscores the profound global impact of their discoveries, which continue to extend and improve the lives of people living with cystic fibrosis.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.
Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface.
The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.
Today Vertex CF medicines are treating over 75,000 people with CF in more than 60 countries on six continents. This represents approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.
ABOUT ALYFTREK AND TRIKAFTA IN THE U.S.
ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. *
TRIKAFTA is indicated for the treatment of CF in patients aged 2 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on clinical and/or in vitro data. *
*If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of the indicated mutation(s) for the respective product.
