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Precision BioSciences Announces Publication in Nature Metabolism Supporting ARCUS® In Vivo Gene Editing as a Potentially Curative Treatment for Mitochondrial Diseases
- Publication validates unique ability of ARCUS genome editing to preferentially target and eliminate mutant m.3243G mitochondrial DNA (mtDNA) with high
About this update from Precision Biosciences, Inc.
[{"type":"text","content":"\n- Publication validates unique ability of ARCUS genome editing to preferentially target and eliminate mutant m.3243G mitochondrial DNA (mtDNA) with high specificity\n\n\n- mitoARCUS efficiently shifted m.3243A>G heteroplasmy without off-target activity, leading to an overall improvement in mitochondrial function\n\n\n DURHAM, N.C.--(BUSINESS WIRE)--\nPrecision 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 publication in the journal Nature Metabolism of a peer reviewed manuscript titled “Efficient elimination of MELAS-associated m.3243G mutant mitochondrial DNA by an engineered mitoARCUS nuclease.” This publication includes preclinical research supporting continued development of the PBGENE-PMM in vivo gene editing program as a potentially curative therapeutic approach for patients with m.3243-associated primary mitochondrial myopathy (PMM).\n\n\n“As a result of attributes that differentiate ARCUS, especially its cut, size, and simplicity as a single-component protein, our strategy is to expand the use of gene editing beyond simple gene knockouts in the liver into more sophisticated edits where ARCUS is uniquely applicable. For mitochondrial diseases, what makes ARCUS such an elegant and simple tool is that it is a single protein that both recognizes and eliminates the mutant mitochondrial DNA,” said Jeff Smith, Ph.D., Chief Research Officer of Precision BioSciences. “Today’s publication further validates the ability of ARCUS to overcome the limitations of CRISPR-based gene editing technologies for treating mitochondrial disease and eliminating mutated mitochondrial DNA with high specificity to improve overall mitochondrial function. Furthermore, these data highlight our ability to design ARCUS nucleases to discriminate a single nucleotide change between the mutant and wild-type DNA sequence, even at very high dose levels. These data strengthen our conviction in PBGENE-PMM as a novel therapy for primary mitochondrial myopathies and we look forward to advancing this program towards an anticipated clinical trial application (CTA) and/or investigational new drug (IND) filing in 2025.”\n\n\nThe Nature Metabolism publication h...