CF Foundation Officially Opens One-of-a-Kind Research Lab

The lab will use the latest scientific advances -- including stem cell research and gene editing -- to accelerate new treatments for cystic fibrosis.

Sept. 16, 2016 | 2 min read

The Cystic Fibrosis Foundation today announced the official opening of its one-of-a kind cystic fibrosis research facility, based in Lexington, Mass. The lab is designed to identify and test potential therapies for CF, readying them for further development with the ultimate goal of bringing new drugs to people living with the disease.

Operated by Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), an affiliate of the CF Foundation, the lab has a team of 25 employees who focus on finding promising drugs for both common and rare mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. To accomplish this, the lab capitalizes on emerging scientific discoveries related to CFTR modulators, nonsense mutations, gene editing and stem cell research.

“Our goal 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 provides a unique bridge to fill the gap between academic discovery and the pharmaceutical industry in advancing CF therapies.”

CF is a complex disease that is caused by more than 1,700 genetic mutations in the 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 CFTR modulators.

Building on its expertise in cell culture, as well as its gene editing and stem cell research capabilities, the lab is spearheading 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 drugs.

Meanwhile, the lab will continue its work screening promising compounds in order to identify the ones that hold the greatest potential for correcting CFTR mutations. 

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