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 editing technology, is investigating whether prime editing could treat several diseases, including cystic fibrosis. 

This technology has the potential to enable many types of cystic fibrosis mutations to be corrected with a single type of genetic therapy.

Gene editing is an approach that corrects the cystic fibrosis transmembrane conductance regulator (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 Cystic Fibrosis 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 cystic fibrosis, 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 cystic fibrosis.

“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 cystic fibrosis may benefit from a transformative treatment,” says Steven M. Rowe, MD, executive vice president and chief scientific officer at the Cystic Fibrosis Foundation, in a release.

The Cystic Fibrosis Foundation’s Path to a Cure

The investment in Prime Medicine is part of the Cystic Fibrosis Foundation’s Path to a Cure, a $500 million research initiative to accelerate treatments—and ultimately a cure—for cystic fibrosis. There are nearly 15% of people with cystic fibrosis who cannot take existing CFTR modulators because their mutations aren’t eligible or because they can’t tolerate side effects.

Rowe continues in a release, “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.”

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