The Cystic Fibrosis Foundation announced it will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic fibrosis. Prime Medicine utilizes 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 a tool that corrects the CFTR mutation in the cell’s DNA, resulting in a potentially permanent fix to the CFTR gene. And Prime Medicine plans 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 researching 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. And 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.
KEY QUOTES:
“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.”
— Steven M. Rowe, MD, executive vice president and chief scientific officer at the CF Foundation
