Precision BioSciences Receives U.S. Patent Allowance Covering PBGENE-PMM for m.3243-Associated Mitochondrial Diseases

Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion, excision, and elimination, today 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 (mitoARCUS) that is designed to specifically target, cleave, and eliminate mutant mitochondrial DNA comprising an m.3243A>G mutation. The m.3243A>G mutation is one of the most common pathogenic mitochondrial DNA mutations, differing from wild-type (normal) 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 (MELAS).

Precision recently announced PBGENE-PMM, the Company’s clinical candidate targeting mutant mitochondrial DNA, as a potentially first-in-class opportunity for treatment of m.3243 associated primary mitochondrial myopathy. Utilizing the claimed mitoARCUS nuclease, PBGENE-PMM is designed to target and eliminate mutant mitochondrial DNA, allowing for repopulation by wild-type mitochondrial DNA and restoration of mitochondrial function.

“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.”

Unlike CRISPR-based gene editing tools, mitoARCUS nucleases are able to gain access to mitochondria because they are small, single-component proteins that integrate DNA-binding and DNA-cleavage and do not require a nucleic acid, such as a guide RNA, for targeting.

“The fact that mitoARCUS can be delivered directly to mitochondria, and has the specificity to distinguish a single base pair difference in the m.3243 A>G mutation, makes PBGENE-PMM a very important potential treatment candidate for patients suffering from m.3243 associated primary mitochondrial myopathy,” said Carlos Moraes, PhD, Esther Lichtenstein Professor of Neurology, and Cell Biology and Anatomy at the University of Miami Miller School of Medicine, and co-inventor of the allowed application.