Prime Medicine receives $24 million to develop gene-editing therapy for cystic fibrosis

BETHESDA, Md. (July 16, 2025) – The Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue developing gene-editing therapies.

Prime Medicine uses prime editing technology, a highly precise DNA editing process. The new investment is focused on developing a prime-editing therapy for the G542X mutation, one of the cystic fibrosis mutations for which there is no cure.

“We believe gene editing offers the best hope for a cure for cystic fibrosis because it can permanently correct mutations,” said Foundation President Michael P. Boyle.

The main challenge is delivering the treatment to the lungs through thick mucus and the body’s immune response. If successful, the therapy would correct a DNA mutation in lung cells that normally produce the CFTR protein, potentially leading to a permanent correction of the CFTR gene.

The Foundation has previously invested up to $15 million in Prime Medicine to develop this technology and demonstrate the versatility of prime editing to correct multiple cystic fibrosis-causing mutations in the laboratory.

As advances in prime editing and delivery to the lung continue, this technology could eventually be used to correct many types of cystic fibrosis mutations. Prime Medicine is advancing multiple approaches in cystic fibrosis, including “hotspots,” which use prime editing to make smaller corrections to specific CFTR mutations, and PASSIGE, which uses prime editing for larger gene insertions. These combined strategies eliminate the need to develop new prime editing therapies for each individual mutation.