Genzyme and Cystic Fibrosis (CF) Foundation Therapeutics entered into a research agreement to support the discovery of drugs to treat patients with the most common mutation, Delta F508. The program’s focus is to identify compounds known as “correctors,” which may aid in the ability of the malfunctioning CFTR protein found in CF patients to operate correctly.
In the Delta F508 mutation, the CFTR protein does not move to its proper place at the cell surface, impeding the flow of fluids into the airways. Nearly 90% of people with CF have at least one copy of the Delta F508 mutation, according to Genzyme and CF Foundation.
In this collaboration researchers will evaluate different compound libraries for correctors for Delta F508, including those at Genzyme and its parent company, Sanofi. The research will take place at multiple Genzyme and Sanofi R&D facilities around the world.
Genzyme says that its experience in CF includes development of molecular diagnostics, clinical trials with a gene therapy, and past drug discovery collaborations with the CF Foundation. “We are delighted to enter into a research collaboration with Genzyme, a company that has long dedicated itself to improving the lives of people with rare diseases,” says Robert J. Beall, Ph.D., president and CEO of the CF Foundation.