Cystic Fibrosis Foundation Invests up to $15 Million in Prime Medicine

Rare Daily Staff

One of the challenges with developing genetic medicines for the rare lung disease cystic fibrosis is that the condition can be caused by roughly 2,000 different mutations to the CFTR gene.

While there have been major advances in treating cystic fibrosis, thanks in part to past investment the Cystic Fibrosis Foundation has made, about 15 percent of people with the condition cannot take existing CFTR modulators because their mutations aren’t eligible or because they can’t tolerate side effects.

Certain gene editing approaches, though, offer the potential to correct the faulty gene in any patient with cystic fibrosis regardless of the underlying mutations. 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.

The Cystic Fibrosis Foundation said it will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic fibrosis. Prime Medicine is pursuing prime editing, a form of gene editing that enables insertions or deletions of small segments of DNA at precise sites.

Prime Medicine will use prime editing to fix the CFTR gene by inserting a part of the DNA that codes for the CFTR gene.

The CF Foundation funding will enable Prime to advance two platform technologies to address cystic fibrosis. 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.

Prime Medicine is exploring delivering potential treatments using lipid nanoparticles to address challenges of delivering a genetic therapy to the lungs of people with CF.

The investment in Prime Medicine is part of the CF Foundation’s Path to a Cure, a $500 million research initiative to accelerate treatments 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.

While the Cystic Fibrosis Foundation acknowledges it will take some time to develop a genetic medicine for cystic fibrosis, it does show the critical role patient organizations can play in de-risking early-stage research to enable the development of transformational medicines for rare diseases.