Cystic Fibrosis Foundation Therapeutics, Inc. Announces $14 Million Research Expansion With Genzyme

Program aimed at advancing search for therapies targeting most common mutation of CF

BETHESDA, Md.--(BUSINESS WIRE)--Cystic Fibrosis Foundation Therapeutics Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, has announced a $14 million expansion of its research agreement with Genzyme, a Sanofi company, to promote development of new treatments for people with the most common CF mutation, F508del.

“The Foundation is focused on supporting the discovery and development of powerful new therapies that attack the underlying cause of this deadly disease”

The agreement will support research and development programs aimed at identifying compounds, known as correctors, that could potentially fix the defective CFTR protein in people with CF. The F508del mutation causes a folding problem in the defective protein that disrupts its movement to the surface of the cell, where it is needed to regulate the flow of salt and fluids in and out of the cell.

Nearly 90 percent of people with CF have at least one copy of the F508del mutation.

CFFT’s program with Genzyme began in 2011 with the goal of identifying new correctors to address the misfolded CFTR protein in those with the F508del mutation. This initial effort yielded several compounds that were shown through extensive testing in the laboratory to help the defective CFTR protein move to the cell surface.

During the expansion phase, Genzyme will focus on further developing select compounds to advance them into CF clinical trials.

“The Foundation is focused on supporting the discovery and development of powerful new therapies that attack the underlying cause of this deadly disease,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “We are pleased to continue CFFT’s agreement with Genzyme and are excited by the possibilities of what our pooled knowledge, expertise and resources can bring.”

CFFT has research programs underway with many leading biotechnology and pharmaceutical companies to discover and develop drugs to treat the basic genetic defect in CF.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis. The Foundation funds more cystic fibrosis research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The Cystic Fibrosis Foundation is a donor-supported nonprofit organization. For more information, go to www.cff.org.