– Data presented at the Mitochondrial Medical Therapeutic Development (MMTD) Annual Conference demonstrated ARCUS’ ability to efficiently remove mutated mitochondrial DNA without nuclear off-target editing.
– PBGENE-PMM shifted heteroplasmy by removing mutant m.3243 mitochondrial DNA (mtDNA) while repopulating wild-type mtDNA, leading to improved mitochondrial function in cells
– Precision plans to file CTA and/or IND for PBGENE-PMM in 2025
Durham, North Carolina, March 19, 2024–(BUSINESS WIRE)–Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company powered by proprietary new ARCUS® The company, a platform for developing in vivo gene editing therapies for advanced gene editing, including gene deletion, insertion, and excision, today announced that its PBGENE, which evaluates ARCUS nuclease as a potential treatment for m.3243, -Published a poster presentation introducing new data from the PMM program. – Associated primary mitochondrial myopathy (PMM). These data will be presented at the Annual Mitochondrial Medicine and Therapeutic Development Conference in Hinxton, UK, March 18-20, 2024.
“Today’s data from our PBGENE-PMM program further validates our work with ARCUS and continues to demonstrate our ability to make precise edits while avoiding off-target activity,” said Chief Research Officer, Precision BioSciences. said Dr. Jeff Smith. “We believe ARCUS is uniquely suited for editing mitochondrial DNA because it can identify single nucleotide differences, making it ideal for editing point mutations such as m.3243. It is designed to remove mutated mitochondrial DNA while leaving a functional wild-type mitochondrial DNA that repopulates the cell intact, resulting in heteroplasmy changes and improved mitochondrial function.”
Smith continued, “Furthermore, ARCUS can cross the mitochondrial membrane to access mitochondrial DNA. This is possible because ARCUS does not require a guide RNA and has both recognition and catalytic activity. This is because it is a protein-only editor contained within a single protein.” This is not possible with his CRISPR-derived editors such as CRISPR-Cas, Base, and Prime editors. We look forward to advancing PBGENE-PMM towards clinical readiness this year and expects him to submit a CTA and/or IND application in 2025. ”
Details of the presentation are below.
title: Shifting m.3243A>G heteroplasmy by PBGENE-PMM: gene editing therapy for primary mitochondrial myopathies
Poster: P22
Presenter: Dr. Wendy Shoop, Precision Bioscience Research Director
Date and time: Tuesday, March 19, 2024, 6:00 PM – 7:00 GMT
position: Hinxton Hall Conference Centre, Wellcome Genome Campus, UK
In preclinical studies announced today, ARCUS demonstrated highly selective removal of mutant m.3423G mtDNA. PBGENE-PMM, which contains both mitochondrial targeting sequences and nuclear export signals, was found to localize exclusively to mitochondria, with no detectable off-target editing within the nuclear genome. Because the m.3243A>G mutation differs from the wild-type sequence by only one nucleotide, PBGENE-PMM was optimized to prevent cleavage of wild-type mtDNA while maintaining activity against mutant mtDNA. When evaluated in cells containing heteroplasmic m.3243A>G mtDNA, PBGENE-PMM-treated cells had 0.3% mutant mtDNA 3 days after transfection compared to control cells containing 95% mutant mtDNA. It was found to contain mtDNA. This strong change in heteroplasmy resulted in an almost two-fold increase in both basal and maximal respiration. Together, these data support his development of PBGENE-PMM as a single-therapy in vivo gene editing therapy for m.3243-associated primary mitochondrial myopathies.
About PBGENE-PMM
PBGENE-PMM is the company’s first wholly owned treatment for m.3243-related primary mitochondrial myopathy (PMM). Mitochondrial disease is the most common inherited metabolic disease, affecting 1 in 4,300 people. There is currently no curative treatment for PMM, which affects approximately 50% of patients with mitochondrial disease. In our 2023 publication of his natural metabolism, Precision takes advantage of the high specificity and single-component nature of PBGENE-PMM and its ability to specifically target mutant mitochondrial DNA while repopulating mitochondria with wild-type (normal) mitochondrial DNA, restoring normal function. We announced new data highlighting the ability to edit and remove. Precision plans to file his CTA and/or IND for this program in 2025.
About Arcus
ARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, designed to insert (knock-in), excise (knock-out), remove, or repair DNA in living cells and organisms. ARCUS is based on the natural genome editing enzyme I-CreI, which evolved in algae. Chlamydomonas reinhardtii It makes highly specific cuts in cellular DNA and stimulates gene insertion at the cut site by homologous recombination. Precision’s platform and products are protected by a comprehensive portfolio of over 130 patents to date.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a leading gene editing company specializing in life improvement (DTIL) with the novel and proprietary ARCUS.® Genome editing platforms differ from other technologies in terms of cutting methods, small size, and structural simplicity. ARCUS is a highly accurate and versatile genome editing platform designed with therapeutic safety, delivery, and control in mind. The company’s pipeline with ARCUS consists of in vivo gene editing candidates designed to provide durable treatments for the widest range of genetic and infectious diseases for which no suitable treatments exist. . For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
Forward-looking statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to historical facts are deemed forward-looking statements. A statement on the therapeutic potential of the ARCUS gene editing approach in the treatment of m.3243-associated PMM includes the following: capabilities and expected timing of CTA and/or IND filings, the ability of mitoARCUS to shift heteroplasmy, and the expected safety, efficacy and benefits of our gene editing approach. In some cases, forward-looking statements can be identified by terminology such as “objective” or the like. “anticipate”, “approach”, “believe”, “contemplate”, “could”, “designed”, “estimate”, “expect”, “goal”, “intend”, “see” , “may,” “mission,” “plan,” “might,” “could,” “anticipate,” “project,” “seek,” “should,” “aim at,” ” “will”, “will” or their negatives and similar word expressions.
Forward-looking statements are based on management’s current expectations, beliefs and assumptions and information currently available to us. These statements are not promises or warranties and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may vary depending on various important factors, including but not limited to: The forward-looking statements may differ materially from those expressed or implied. Ability to increase profitability. our ability to raise sufficient funds to advance our programs; risks related to the impact of our capital requirements, anticipated cash runway, requirements and limitations under our current debt instruments, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progress and success of programs and product candidates in which we expend resources; our limited ability or inability to evaluate the safety and effectiveness of our product candidates; the risk that other genome editing technologies may offer greater advantages than our ARCUS technology; Reliance on ARCUS Technology. the initiation, cost, timing, progress, milestone achievements and results of research and development activities and preclinical and clinical studies, including clinical trials and investigational new drug applications; Public awareness of genome editing technology and its applications. Competition in genome editing, biopharmaceuticals, and biotechnology. the ability of us, our collaborators or other licensees to identify, develop and commercialize product candidates; pending and potential product liability claims and penalties against us, our collaborators or other licensees related to us, our technology and product candidates; the U.S. and foreign regulatory landscape applicable to the development of product candidates by us and our collaborators or other licensees; the ability of us, our collaborators or other licensees to advance, successfully design, implement and complete product candidates into clinical or field trials; potential manufacturing issues related to the development or commercialization of our product candidates; delays or difficulties in our, our collaborators’ and other licensees’ ability to enroll patients; Changes to preliminary “top line” and initial data announced or published by the Company. if our product candidates do not function as intended or cause unwanted side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the ability of us or our licensees to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; the ability of us, our collaborators or other licensees to obtain and maintain regulatory approval for our product candidates and the associated restrictions, limitations and/or warnings contained on the labels of approved product candidates; . the rate and degree of market acceptance of our product candidates; our ability to effectively manage the growth of our business; our ability to attract, retain and motivate management and employees; the impact of system failures or security breaches; Exposure to Insurance Expenses and Uninsured Liabilities. Impact of tax regulations. the impact of the novel coronavirus pandemic and its variants, or infectious disease pandemics, epidemics, or outbreaks; the success of existing cooperative agreements and the ability to enter into new cooperative agreements; current and future relationships with and reliance on third parties, including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and our product candidates; potential litigation related to infringement or misappropriation of intellectual property rights; the effects of natural and man-made disasters, public health emergencies and other natural disasters; the impact of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks associated with ownership of our common stock, including fluctuations in our stock price; our ability to meet and maintain the requirements for listing our common stock on Nasdaq or other public securities exchanges; and other important factors described under the caption “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2023. Such factors may be updated from time to time in other filings with the SEC. These can be accessed from the investor page of the SEC’s website (https://www.sec.gov/) and the SEC Filings of our website (investor.precisionbiosciences.com).
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