December 2011

New Research Collaboration Takes Aim at Most Common CF Mutation

As part of its effort to speed the development of therapies targeting the basic defect in CF, the Cystic Fibrosis Foundation has entered into a collaboration with Pfizer, Inc., one of the world’s largest pharmaceutical companies.

The goal of the program is to discover compounds to treat people with the most common CF mutation, Delta F508. In this mutation, the defective CFTR protein does not fold in the correct way to reach its proper place at the cell surface, where it helps maintain the proper flow of salt and fluids into the airways.

Nearly 90 percent of people with CF have at least one copy of the Delta F508 mutation.
The new agreement grows out of an earlier collaboration between the CF Foundation and the biotech company FoldRx Pharmaceuticals, Inc., which Pfizer acquired in 2010.

“This collaboration marks a very exciting step in the fight against CF,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Pfizer’s commitment of resources and scientific capacity, combined with its expertise in the field of rare diseases, will help accelerate the development of more therapies targeting the underlying cause of CF that will benefit the greatest number of people with the disease.”

Pfizer has significantly expanded the team of scientists and technicians working on the CF program, and will also be able to draw on its massive library of about 5 million chemical compounds to screen for potential CF therapies.

The CF Foundation will provide technical and scientific as well as financial support to develop the new program. “The Foundation has assembled a significant number of experts that are regularly helping us, as well as its networks of virtually all the researchers around the world who are working on this disease,” said Ed Mascioli, M.D., vice president of the Orphan and Genetic Diseases Research Unit at Pfizer.

Watch an interview with Ed Mascioli, M.D., vice president of the Orphan and Genetic Diseases Research Unit at Pfizer.

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