Prime Medicine, Inc. reported new preclinical data demonstrating the ability of Prime Editors to efficiently and precisely correct the predominant mutations that cause rhodopsin associated autosomal dominant retinitis pigmentosa (RHO adRP). The data were presented at the International Symposium on Retinal Degeneration 2023 Congress (RD2023) in Costa del Sol, Spain. RHO adRP is a rare inherited retinal disease that causes progressive vision loss in early adolescence, leading to eventual blindness in adulthood due to photoreceptor degeneration.

It results from mutations in the gene RHO, which encourages rhodopsin, the light-sensitive G protein-coupled receptor involved in phototransduction in rods, a type of photoreceptor, and leads to the progressive loss of rods and, subsequently, cones in the retina. Specifically, today's data highlight the ability of two Prime Editors to correct the predominant mutations causing RHO adRP -- one to correct p.P23H, the most common disease-causing mutation in RHO in the U.S., and one to correct 18 different mutations at a mutational hotspot in RHO, including p.V345L and p.P347L, which are the most prevalent mutations in Europe.