Precision BioSciences Announces Presentation at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference

Key Terms

in vivo medical

In vivo describes tests or experiments performed inside a living organism, such as an animal or human, to observe how a drug, device or biological process behaves in a real, functioning body. Investors care because in vivo results reveal safety, effectiveness and possible side effects that lab tests cannot, much like road-testing a prototype car in traffic rather than only on a bench — outcomes can strongly influence regulatory approval, clinical success and a company’s valuation.

gene editing medical

Gene editing is a set of laboratory techniques that change the DNA inside living cells to add, remove, or alter specific genes, like using precise 'cut-and-paste' tools to edit an instruction manual for a living organism. It matters to investors because successful edits can produce new therapies, improve agricultural products, or create commercial advantages; progress, safety, regulatory approvals, and patent control all affect a company’s value and risk profile.

muscular dystrophy medical

Muscular dystrophy is a group of inherited conditions that progressively weaken and break down skeletal muscles, making everyday movements harder over time; think of muscle fibers like ropes that gradually fray and lose strength. It matters to investors because the chronic, often long-term nature of these diseases creates clear demand for drugs, gene therapies and supportive care, shaping clinical trial activity, regulatory attention, market size and potential returns — as well as reimbursement and pricing debates.

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 study data supporting the potential safety and long-term efficacy of PBGENE-DMD has been selected as a poster presentation at the upcoming 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference taking place March 8-11 in Orlando, Florida.

Details of the presentation:

Abstract title: PBGENE-DMD gene editing treatment leads to safe and long-term functional improvement in humanized DMD-disease mouse model
Publication number: 142M
Type of presentation: Poster presentation
Authors: Adam Mischler, Whitney Lewis, Wendy Shoop, Traci Reddick, Dan Nazarenko, Nicole W. Heard, Ben Morris, Haley Grimason, Krsna Rangarajan, Cheng-Wei Wang, Kalpana Kodi, Dominique Burgess, Katie Poe, Gary Owens, Jeff Smith, Cassandra Gorsuch
Date: Monday, March 9, 2026

About PBGENE-DMD

PBGENE-DMD, a novel first-in-class gene editing therapy, utilizes a gene excision approach that is clearly differentiated from existing microdystrophin and exon skipping treatments. PBGENE-DMD is designed to potentially provide durable functional muscle improvement for DMD patients with mutations between exons 45 and 55, which impact up to 60% of boys with DMD. A single AAV encodes two ARCUS proteins designed to permanently edit a patient’s DNA within the dystrophin gene, resulting in a naturally-expressed, near full-length, functional dystrophin protein. Supported by robust preclinical evidence, PBGENE-DMD is designed to drive functional improvement in skeletal and cardiac muscle over time with the ability to target and edit muscle satellite cells.

In preclinical studies, PBGENE-DMD demonstrated the ability to target key muscle types involved in the progression of DMD and produced significant, durable functional improvements in a humanized DMD mouse model. PBGENE-DMD restored production of a near full-length functional dystrophin protein across multiple muscles, including cardiac tissue and various key skeletal muscle groups. In addition, PBGENE-DMD edited satellite muscle stem cells, believed to be critical for long-term durability and sustained functional improvement.

PBGENE-DMD has received Investigational New Drug clearance from the U.S. Food and Drug Administration (FDA) enabling initiation of clinical trial site activation. PBGENE-DMD was previously granted FDA Rare Pediatric Disease (RPD) and Orphan Drug (ODD) designation for the treatment of DMD.

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, expectations about operational initiatives, strategies, further development including dose expansion, and timing of additional updates or data releases of PBGENE-DMD and the FUNCTION-DMD Trial; translation of results in preclinical studies of PBGENE-DMD to clinical studies in humans; expected timing and outcome of regulatory and institutional review board processes for PBGENE-DMD site activation; the potential long term efficacy of PBGENE-DMD as show in new preclinical study data; the design of PBGENE-DMD to potentially provide durable functional muscle improvement for DMD patients with mutations in exons 45-55 impacting up to 60% of boys with DMD; and the design of PBGENE-DMD to drive functional improvement in skeletal and cardiac muscle over time with the ability to target and edit muscle satellite cells. 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, 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; 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; our ability to advance product candidates into, and successfully design, implement and complete, clinical trials; changes in interim “top-line” and initial data that we announce or publish; our current and future relationships with and reliance on third parties including suppliers and manufacturers; and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2024 and our Quarterly Reports on Form 10-Q for the quarterly periods ended March 31, 2025, June 30, 2025, and September 30, 2025 as any such factors may be updated from time to time in our other filings with the U.S. Securities and Exchange Commission (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.

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Investor and Media Contact:

Naresh Tanna

Vice President of Investor Relations

naresh.tanna@precisionbiosciences.com

Source: Precision BioSciences, Inc.