Prime Editor for Cystic Fibrosis

Prime Editor for Cystic Fibrosis is a gene-editing therapeutic approach under development to correct mutations in the CFTR gene that cause cystic fibrosis. This technology uses "prime editing," a next-generation CRISPR-derived genome editing method capable of introducing precise nucleotide substitutions, insertions, and deletions at targeted locations in DNA. The therapy aims to restore normal function of the CFTR protein by directly correcting disease-causing mutations—including those not addressable by current modulator drugs—potentially offering a durable, one-time treatment. Laboratory studies have demonstrated successful correction of several key CFTR mutations (such as F508del, L227R, N1303K) in human airway epithelial cells and organoids derived from patients with cystic fibrosis. Functional assays show restoration of over 50% wild-type channel activity after correction[1][5][6]. The program is sponsored by Prime Medicine and partially funded by the Cystic Fibrosis Foundation[1][7][8].

Other names

prime editing for cystic fibrosisprime editing CFTR therapy