BETHESDA, Md.--(BUSINESS WIRE)--The Cystic Fibrosis Foundation has agreed to invest up to $15 million in ReCode Therapeutics to support their gene editing collaboration with Intellia Therapeutics. The funding is intended to be used to develop a gene editing therapy that could be delivered into the lung cells of people with cystic fibrosis.
ReCode is focused on developing a version of its lipid nanoparticles that is optimized for delivery to lung stem cells, the ideal target for a permanent gene editing therapy. Similar to a pill capsule that protects medicines, lipid nanoparticles shield and direct genetic material to cells without damaging the material along the way. Lung stem cells are important because they create new lung cells to replace damaged or dying ones. Delivering genetic-based therapies to specific cells in the lungs remains a significant challenge in medicine.
While ReCode concentrates on the delivery system, Intellia is working to develop the gene editing therapy being delivered. Intellia’s therapy is designed to target permanent or near-permanent correction of a specific nonsense mutation (also called a “stop” mutation) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Other rare mutations that are not eligible for CFTR modulators will be explored as the editing technology matures.
“Gene editing is a promising, complex technology, which will take many years to develop into a therapy for people with CF,” said Steven Rowe, MD, executive vice president and chief scientific officer of the Cystic Fibrosis Foundation. “By investing in this research now, we are laying the foundation to develop what we hope will be a transformative treatment for people with CF, especially those who can’t take CFTR modulators.”
The companies are initially targeting the lungs for gene correction because CF severely impacts the lungs and can cause respiratory failure — a leading cause of death in people with the disease. Eventually, a systemic treatment will need to be developed to correct CF in other organs impacted by the disease, such as the pancreas.
The funding for the gene editing program is in addition to a separate $15 million investment in ReCode that the Foundation made to develop a messenger RNA therapy, which is being tested in Phase 1 and 2 clinical trials. Messenger RNA therapy is another potential treatment that could help all people with CF, especially those who aren’t eligible for or cannot tolerate CFTR modulators.
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.
