Precision BioSciences Receives U.S. Patent Allowance Covering PBGENE-PMM for m.3243-Associated Mitochondrial Diseases
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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
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
About Mitochondria and Primary Mitochondrial Myopathy
Mitochondria comprise multiple copies of a circular DNA referred to as mitochondrial DNA, which encodes for 13 subunits of the oxidative phosphorylation (OXPHOS) system, 2 rRNAs, and 22 tRNAs that are all necessary to support mitochondrial function. It is believed that a shift in mitochondrial DNA heteroplasmy toward wild-type (normal) may provide therapeutic benefit for patients, and not all mutant mitochondrial DNA must be eliminated to achieve improvements in symptoms. Rather, mutant mitochondrial DNA levels only need to be shifted below a disease threshold level.
Mitochondrial diseases that arise from mutations in mitochondrial DNA are the most common hereditary metabolic disorder, affecting 1 in 4,300 people. Primary mitochondrial myopathy is characterized by severe fatigue and can affect skeletal muscle, and other high energy organs such as the brain, eyes, ears and heart. Primary mitochondrial myopathy currently lacks curative treatment and impacts approximately 50% of patients with mitochondrial disease.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. The Company intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the expected safety, efficacy, and benefit of our gene editing approaches including editing efficiency and delivery methods, the suitability of ARCUS nucleases for gene insertion, excision, and elimination, including the elimination of mutant mitochondrial DNA, the clinical development, nomination, and goals of our PBGENE-PMM program, the potential for a shift in heteroplasmy toward wild-type mitochondrial DNA to restore mitochondrial function, and therapeutic potential of an ARCUS gene editing approach for the treatment of m.3243-associated mitochondrial diseases. The words “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “promise,” “pursue,” “should,” “target,” “will,” “would,” and other similar words or expressions, or the negative of these words or similar words or expressions, are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, but involve number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical studies and clinical trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the
All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.
Senior Director of Finance and Corporate Controller