Rare Daily Staff

The Cystic Fibrosis Foundation unveiled five-year, $500 million research agenda to drive the discovery and development of therapies and cures for cystic fibrosis patients who have not benefited from new therapies that have emerged.

Cystic Fibrosis (CF) is a rare, life-shortening genetic disease. It is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from mutations in the CFTR gene. There are approximately 2,000 known mutations in the CFTR gene. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

The foundation is calling on potential collaborators to submit proposals for its so-called Path to a Cure initiative with the aim of accelerating the pace of progress.

The Foundation is challenging potential collaborators to submit proposals that will accelerate the pace of progress in CF drug discovery and development.

“The CF Foundation has a long track record of success in facilitating the development of new cystic fibrosis therapies. Our bold investments in innovative research sparked the development of CFTR modulators, medicines which are now transforming the lives of people with CF,” said Michael Boyle, senior vice president of therapeutics development at the CF Foundation, who will assume the role of CEO effective January 1, 2020. “We see an unprecedented opportunity to identify and advance treatments for the underlying cause of CF and deliver a cure for every person with the disease and will use every tool available to advance the research that will get us there.”

The Path to a Cure initiative is intended to build on the foundation’s venture philanthropy strategy to stimulate industry investment in CF. In addition to funding, the foundation offers partners a range of resources to de-risk CF drug discovery and development—including the world’s largest network of cystic fibrosis clinical trial sites and a dedicated laboratory to support translational research for potential therapeutic programs.

Under the effort, the Foundation has prioritized identifying innovative approaches for individuals who do not respond to currently available treatments.

It said its initiative centers on three core strategies to address the underlying cause of CF: repairing broken CFTR protein, restoring CFTR protein when none exists, and fixing or replacing the underlying genetic mutation to address the root cause of CF. The organization said each approach requires a different set of scientific tools and knowledge, leading the foundation to bring together researchers and industry leaders from a range of disciplines to advance multiple areas of research in parallel.

Photo: Michael Boyle, senior vice president of therapeutics development at the CF Foundation

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