BETHESDA, Md. — Today, the Cystic Fibrosis Foundation announced that it will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic fibrosis. Prime Medicine is using a gene editing technology called prime editing — a technology that enables insertions or deletions of small segments of DNA at precise sites. Prime Medicine — founded by researchers who pioneered the development of this unique editing technology — is investigating whether prime editing could treat several diseases, including CF. This technology has the potential to enable many types of CF mutations to be corrected with a single type of genetic therapy.
Gene editing is an approach that corrects the CFTR mutation in the cell’s DNA, resulting in a potentially permanent fix to the CFTR gene. Prime Medicine intends to use prime editing to fix the CFTR gene by inserting a part of the DNA that codes for the CFTR gene.
The funding provided by the CF Foundation to Prime Medicine will go toward research into two of Prime’s platform technologies. The first, called “hotspot,” uses prime editing to make smaller corrections to specific CFTR mutations. Prime Medicine has already begun to use hotspot to correct the G542X nonsense CFTR mutation in the lab and will extend this work to develop therapies for other clusters of CFTR mutations. The second, called PASSIGETM, uses prime editing to make large gene insertions, which could potentially work in nearly any person with CF, regardless of their mutations. For both technologies, Prime Medicine is investigating delivering potential treatments using lipid nanoparticles, a technology that could address challenges in delivering a genetic therapy to the lungs of people with CF.
“This is an exciting time for genetic technologies. Last month marked the first FDA approval of a gene editing therapy for sickle cell disease, showing the potential of gene editing outside the lab. We want to bring that success to cystic fibrosis, so that all people with CF may benefit from a transformative treatment,” said Steven M. Rowe, MD, executive vice president and chief scientific officer at the CF Foundation.
The CF Foundation’s Path to a Cure
The investment in Prime Medicine is part of the CF Foundation’s Path to a Cure, a $500 million research initiative to accelerate treatments — and ultimately a cure — for cystic fibrosis. Funding genetic therapies is critical in cystic fibrosis research, and in the CF Foundation’s mission to help all people with CF live longer, healthier lives. There are nearly 15% of people with CF who cannot take existing CFTR modulators because their mutations aren’t eligible or because they can’t tolerate side effects.
Dr. Rowe continued, “A genetic therapy for cystic fibrosis will take time to develop. Our investment in Prime Medicine represents one of the Foundation’s key strategies to achieving a genetic therapy in cystic fibrosis: de-risking early-stage science to pave the way for the ultimate cure.”
For more on how the CF Foundation is pursuing treatments for all people with cystic fibrosis, visit 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.