BETHESDA, Md. -- Today, the Cystic Fibrosis Foundation announced that it has awarded up to $400,000 to Life Edit Therapeutics Inc. to explore the application of their unique gene editing technology in CF. The funding will enable the company to screen their library of editing tools for options that may correct the six most common nonsense mutations in people with cystic fibrosis.
Life Edit aims to improve base editing, a variation on more traditional gene editing techniques. This approach may make it easier to deliver gene editing components to cells.
“Gene editing holds promise as a way to permanently correct mutations in the cell and -- hopefully, one day -- provide a cure,” said William Skach, MD, executive vice president and chief scientific officer of the CF Foundation. “By funding this award, we are taking an early step along this long journey to developing gene editing as a potential therapy for people with cystic fibrosis.”
Nearly 4,000 people with CF in the U.S. -- or almost 13% -- have at least one nonsense mutation, according to the CF Foundation Patient Registry. Of that number, more than 850 people -- or between 2% and 3% -- cannot benefit from any currently available modulator therapy.
About the Path to a Cure
The CF Foundation launched its $500 million Path to a Cure initiative in October 2019. This initiative centers around 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. 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. Innovators who are interested in pursuing programs in cystic fibrosis can learn about our specific funding opportunities on cff.org.
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 CF 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 CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.