At Vertex we are relentless in our quest to improve the lives of people living with serious diseases. We always say that drug discovery is the ultimate team sport, and our scientists embrace innovation with the ambition to improve the lives of patients through the development of innovative medicines. 

This month, three Vertex researchers — Paul Negulescu, Sabine Hadida and Fred Van Goor — are receiving the Breakthrough Prize for groundbreaking discoveries leading to the first medicines to address the underlying cause of cystic fibrosis (CF). The Breakthrough Prize recognizes the world’s leading global scientists for “transformative advances toward understanding living systems and extending human life.” The citation from the Breakthrough Foundation recognizes the team’s research and development of multiple medicines that greatly improve quality of life — and length of life — for people with CF.  

For over 20 years, Paul, Sabine and Fred have worked with countless others across Vertex to invent and develop a new class of therapeutics. The team’s collaborative contributions are inspiring. Paul conceived of the project and has served as overall program leader since its conception; Fred has been the lead biologist, and Sabine the lead chemist. It is truly an honor to congratulate these three for their leadership that helped get us to this point. I also want to recognize the contributions of Peter Grootenhuis, who served as the project leader for the CF program for 15 years. His contributions to this teams’ success are tremendous; sadly, Peter passed away in 2019 from complications due to amyotrophic lateral sclerosis (ALS).  

Paul, Sabine and Fred’s accomplishments are of lasting significance to people with CF and to therapeutics more broadly. These advances have not only inspired a different class of therapeutics, but they are also having a profound impact on the lives of tens of thousands of people and families with CF.  

This recognition adds to the series of prestigious awards the team has received for their groundbreaking work in CF. In 2023, Paul, Sabine and Fred were honored with the Wiley Foundation Prize in Biomedical Sciences for their discovery of small molecule therapies for the treatment of CF. The team shared the award with Professor Michael Welsh at the University of Iowa, who made key biological insights that informed CF drug discovery efforts.  

Further recognizing these significant advances, Paul Negulescu was awarded the 2022 Shaw Prize in Life Science and Medicine with Professor Michael Welsh and the 2018 Warren Alpert Foundation Prize alongside Michael Welsh, Francis Collins, Lap-Chee Tsui and Bonnie Ramsey. Both prizes honor the group’s pioneering contributions to the discovery of CFTR modulators and the subsequent research that led to the development of these medicines to treat the underlying cause of CF. The Shaw Prize is a prestigious international award that honors individuals who have made outstanding contributions in academic and scientific research and is dedicated to furthering societal progress, enhancing quality of life and enriching humanity’s spiritual civilization. The Alpert Prize, in association with Harvard Medical School, recognizes and honors one or more scientists, physicians and researchers whose scientific achievements have led to the prevention, cure or treatment of human disorders or for seminal research that holds great promise to change our ability to treat disease.  

More broadly, each of these awards recognizes the entire effort at Vertex over more than two decades. This effort required innovation, dedication and commitment spanning the entire company. Paul, Fred and Sabine would be the first to call out the contributions of everyone at Vertex who has worked for so long — and so well — to bring these medicines to patients. 

While this award recognizes the amazing discoveries these Vertexians have made for people living with CF, our work in CF is nowhere near done. I believe the best is yet to come as we pursue investigational therapies to treat all people with CF.  

To learn more about the Breakthrough Prize in Life Sciences, click here.