PRME - Prime Medicine Receives Up to $15 Million from Cystic Fibrosis Foundation to Advance Hotspot and PASSIGE™ Prime Editors for Cystic Fibrosis | Benzinga
CAMBRIDGE, Mass., Jan. 25, 2024 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (NASDAQ:PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced that the Cystic Fibrosis Foundation (CF Foundation) has agreed to provide the Company with up to $15 million to support the development of Prime Editors for the treatment of Cystic Fibrosis (CF).
CF is an inherited genetic disease, which can be caused by more than 1,700 different mutations in the gene that produces the CF transmembrane conductance regulator (CFTR) protein. These mutations, including nonsense and rare mutations, cause the CFTR protein to malfunction or not be made at all; this leads to a buildup of thick mucus, which in turn leads to persistent lung infections, destruction of the pancreas, and complications in other organs. CF affects close to 40,000 people in the United States and, while significant progress in the last decade has created therapeutic options for many patients, there is no cure and approximately 15 percent of patients cannot clinically benefit from or tolerate existing treatments.
Prime Medicine is uniquely positioned to develop potentially curative therapies for CF by using Prime Editing to correct CFTR mutations at the natural genetic locus. Funding from the CF Foundation will allow Prime Medicine to progress two distinct strategies for applying Prime Editing to treat CF: hotspot editing and PASSIGE™ (Prime Assisted Site Specific Integrase Gene Editing). Through hotspot editing, the Company aims to address multiple mutations at mutational hotspots with a small number of Prime Editors. Preclinical data generated by Prime Medicine suggest that using only eight hotspot Prime Editors could benefit more than 98 percent of all people with CF, including those living with nonsense and rare mutations whose disease is not amenable to treatment with currently approved therapies, as well as those who do not tolerate existing therapies. Prime Medicine has begun preclinical research to use hotspot Prime Editors to correct the G542X nonsense CFTR mutation, and plans to extend this work to develop hotspot Prime Editors for other clusters of CFTR mutations.
In parallel, using PASSIGE, Prime Medicine aims to address nearly all people with CF with a single superexon insertion strategy. This ...