The Cystic Fibrosis Foundation has announced the official opening of a new, one-of-a kind research facility in Lexington, Mass. Operated by Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), an affiliate of the CF Foundation, the lab is designed to identify and test potential therapies for rare and common mutations of cystic fibrosis, with the ultimate goal of bringing new treatments to people living with the disease.
CF is a complex, fatal disease that affects about 30,000 adults and children in the United States and 70,000 people worldwide. It is caused by more than 1,700 genetic mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Although the majority of individuals with CF have at least one copy of the most common mutation, 5 percent of people carry only nonsense and other types of rare mutations that do not respond to existing drugs – known as CFTR modulators – that treat the underlying cause of the disease.
“Our goal with the new research lab is to speed the development of new treatments for all people with cystic fibrosis,” said William Skach, M.D., senior vice president for research affairs at the CF Foundation. “The lab will provide a unique bridge to fill the gap between academic discovery and the pharmaceutical industry in advancing CF therapies.”
Building on CFFT’s expertise in cell culture, gene editing and stem cell research, the lab’s 25 scientists will spearhead a new initiative to collect and grow cells from people with CF who have nonsense and other rare mutations. These cells will be critical for identifying treatments for rare mutations, and will be made available to academic institutions and pharmaceutical companies to expedite the development of new medications.
The team also will continue to screen promising compounds in order to identify the ones that hold the greatest potential for correcting CFTR mutations.