Precision gets NOA from USPTO covering PBGENE-PMM for m.3243

Precision BioSciences announced it has received a Notice of Allowance from the U.S. Patent and Trademark Office USPTO for U.S. Patent Application No. 18/161,560, titled “Engineered Meganucleases That Target Human Mitochondrial Genomes.” Once issued, the patent arising from this application will have a standard expiration date in April 2042. The allowed composition of matter claims in this U.S. application encompass a mitochondria-targeted ARCUS nuclease or mitoARCUS, that is designed to specifically target, cleave, and eliminate mutant mitochondrial DNA comprising an m.3243Agreater thanG mutation. The m.3243A greater thanG mutation is one of the most common pathogenic mitochondrial DNA mutations, differing from wild-type mitochondrial DNA by a single base change, and is associated with the development of a number of disorders, including primary mitochondrial myopathies that primarily affect skeletal muscle, and mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes…”The high specificity and single component nature of Precision’s mitoARCUS nucleases are designed to enable specific elimination of mutant mitochondrial DNA while allowing the normal mitochondrial DNA to repopulate in the mitochondria and reestablish normal function,” said Jeff Smith, PhD, Co-Founder and Chief Research Officer at Precision BioSciences. “PBGENE-PMM holds the potential to deliver a one-time, transformative treatment for patients with primary mitochondrial myopathy.”