Genzyme, a Sanofi company, and Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, today announced a research agreement to support the discovery of new drugs to treat people with the most common mutation found in patients with CF, Delta F508.
People with cystic fibrosis, a genetic disease, experience a cascade of symptoms that can lead to life-threatening lung infections and premature death.
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 percent of people with CF have at least one copy of the Delta F508 mutation.
In this collaboration, researchers will evaluate different compound libraries for correctors for Delta F508, and will take advantage of the vast compound libraries of both Genzyme and Sanofi. The research will take place throughout several Genzyme and Sanofi R&D facilities globally. Genzyme brings to the collaboration more than 20 years' experience exploring treatments for people living with CF. The company's efforts have ranged from improved molecular diagnostics to clinical trials with a gene therapy, and have included past collaboration with the CF Foundation in the area of drug discovery.
"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," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. "Genzyme's capabilities and resources will help the CF Foundation accelerate its effort to find drugs to treat the most common mutation in CF and have the greatest impact on those with this disease." "While there has been great momentum recently in cystic fibrosis research, there is still great unmet need," said Genzyme's president and CEO David Meeker, MD. "Together with the CF Foundation, we look forward to working to accelerate the pace of discovery on behalf of CF patients around the world."