Vertex Researchers Awarded “Breakthrough Prize in Life Sciences”

Three Vertex 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.” 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.

“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.

The 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, 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, Trikaftka, is a triple combination therapy first approved in 2019 that today can treat about 90% of people with CF who have responsive mutations.

Vertex 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 in collaboration with Moderna to develop an mRNA therapeutic designed to treat the approximately 5,000 people with CF who do not produce any CFTR protein. If this mRNA therapy is successful, it will be possible for all people with CF to have medicines to treat the underlying cause of their disease. (Source: Vertex Pharmaceuticals Website, 14 September, 2023)