DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases, today announced that new preclinical data from its PBGENE-DMD program have been accepted for an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting, taking place May 11-15, 2026, in Boston, Massachusetts. The accepted abstract highlights new data demonstrating compelling efficacy observed in early-juvenile mice supporting the potential benefit of earlier intervention with PBGENE-DMD in younger patient populations. These data build on previously shared updates showing treatment with PBGENE-DMD leads to durable functional improvement in a humanized Duchenne muscular dystrophy (DMD) mouse model.
Details of the presentation:
Abstract title: PBGENE-DMD gene editing drives safe, efficacious, and durable functional improvement in a humanized Duchenne muscular dystrophy mouse model
Session: Emerging molecular therapeutic strategies for muscular dystrophies
Presenter: Adam Michler Ph.D., DMD Research Lead
Presentation Type: Oral Presentation
Presentation Time: May 14, 2026 at 8:45 a.m. ET
About PBGENE-DMD, A Muscle-Targeted Excision Program
PBGENE-DMD is Precision's development program for the treatment of Duchenne Muscular Dystrophy (DMD), a devastating genetic disease caused by mutations in the dystrophin gene that prevents production of the dystrophin protein, which is essential for maintaining muscle structural integrity and function. DMD affects approximately 15,000 patients in the U.S. alone, and there are currently no approved therapies capable of driving significant, durable functional improvements over time.
PBGENE-DMD is designed to durably improve function for approximately 60% of patients with DMD by employing two complementary ARCUS nucleases, delivered using a single AAV, to excise exons 45-55 of the dystrophin gene, restoring expression of a near full-length dystrophin protein. This protein more closely resembles normal dystrophin than synthetic, truncated microdystrophin approaches, which offer minimal functional benefit. Precision’s Phase 1/2 FUNCTION-DMD study is expected to enroll ambulatory DMD patients with mutations between exons 45 and 55, which impact approximately 60% of boys with DMD. The clinical trial will employ an appropriate immune modulation regimen and safety monitoring program to treat patients at world class specialized DMD clinical sites.
PBGENE-DMD was granted Orphan Drug Designation by the FDA in July 2025. The PBGENE-DMD program is eligible for a Priority Review Voucher (PRV) via the Rare Pediatric Disease Priority Review Voucher (PRV) program, which was signed into law on February 3, 2026, as part of the Consolidated Appropriations Act of 2026. PBGENE-DMD received Fast Track designation from the FDA in February 2026.
Further details on the trial can be found on Precision’s website and clinicaltrials.gov identifier NCT07429240.
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 matters of historical fact should be considered forward-looking statements, including, without limitation, the design of PBGENE-DMD to improve function over time and address approximately 60% of patients with DMD; the potential for PBGENE-DMD to provide durable functional improvement with a single dose of AAV; translation of results in preclinical studies of ARCUS nucleases including safety, efficacy and durable functional improvement to clinical studies in humans; the employment of an appropriate immune modulation regimen and safety monitoring program to treat ambulatory patients at world class specialized DMD clinical sites; the eligibility of PBGENE-DMD for a Priority Review Voucher (PRV) via the Rare Pediatric Disease Priority Review Voucher (PRV) program; and the preclinical and clinical development and demonstrated, potential and expected safety, efficacy, durability, and benefit of PBGENE-DMD. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “belief”, “believe,” “contemplate,” “could,” “design,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “suggest,” “target,” “will,” “would,” or the negative thereof and similar words and 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, and involve a 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 to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, 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 progression and 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; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators’ or other licensees’ development of product candidates; our or our collaborators’ or other licensees’ ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators’ and other licensees’ ability to enroll patients; changes in interim “top-line” and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees’ ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators’ or other licensees’ ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our 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 any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-K for the annual period ended December 31, 2025, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com.
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.
Contacts
Investor and Media Contact:
Naresh Tanna
Vice President of Investor Relations
naresh.tanna@precisionbiosciences.com
