Press release
Vertex Researchers Awarded 2024 Breakthrough Prize in Life Sciences
- Paul Negulescu, Fredrick Van Goor and Sabine Hadida receive Breakthrough Prize for developing transformative medicines for people with cystic fibrosis –
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- Paul Negulescu, Fredrick Van Goor and Sabine Hadida receive Breakthrough Prize for developing transformative medicines for people with cystic fibrosis –\n\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that three of its researchers have been awarded the 2024 Breakthrough Prize in Life Sciences “for developing life-transforming drug combinations that repair the defective chloride channel protein in patients with cystic fibrosis.” Specifically, the award celebrates the work of Paul Negulescu, Ph.D., Fredrick Van Goor, Ph.D., and Sabine Hadida, Ph.D., who have worked together to lead cystic fibrosis (CF) discovery for over 20 years.\n\n\n“The research teams led by Paul, Fred and Sabine discovered the first and only medicines that address the underlying cause of cystic fibrosis. This remarkable effort required discovery of three novel mechanisms of action, resulting in the first disease modifying therapies that act by restoring function to a misfolded protein,” said David Altshuler, M.D., Ph.D. Executive Vice President, Global Research, and Chief Scientific Officer. “This prize recognizes the dedication and creativity of thousands of people at Vertex spanning research, development, regulatory, manufacturing and patient access, as well as our partners in the CF community, who have worked tirelessly to bring these medicines to people with CF around the world.”\n\n\nThe gene responsible for cystic fibrosis was discovered in 1989, but until the work of Vertex scientists, treatment of CF addressed only its symptoms rather than the underlying cause of the disease. Today, Vertex’s four approved oral medicines treat CF by improving the function of the defective CFTR protein. The first of these medicines, KALYDECO® (ivacaftor), was approved in 2012, and today is approved for people with CF ages 1 month and older carrying responsive CFTR mutations. The most recently developed medicine, TRIKAFTA®, is a triple combination therapy (elexacaftor/tezacaftor/ivacaftor and ivacaftor) first approved in 2019 that today can treat ~90% of people with CF who have responsive mutations.\n\n\nVertex is currently developing a next-in-class investigational CF triple combination therapy, now in Phase 3 trials, that has the potential for enhanced clinical benefit. The company is also in early clinical trials...