Precision BioSciences Announces Key Opinion Leader Event and Provides Update on PBGENE-DMD Program for Duchenne Muscular Dystrophy

Durham, NC – March 17, 2026 – Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company, has released a Form 8-K highlighting significant developments in its PBGENE-DMD program targeting Duchenne muscular dystrophy (DMD). The Company hosted a Key Opinion Leader (KOL) event featuring leading experts and provided an in-depth update on its clinical and operational progress, which could have important implications for investors and the company’s share price.

Key Highlights from the Report

• PBGENE-DMD Program Advances:
  • The Company’s PBGENE-DMD gene editing therapy is designed to permanently and safely restore muscle function for the majority of patients with DMD by excising the ‘hot’ region between exons 45-55 of the dystrophin gene.
  • PBGENE-DMD is positioned to target approximately 60% of DMD patients, a sizable market segment with significant unmet need.
  • The program has received clearance from the US FDA for its Investigational New Drug (IND) application in early Q1 2026. Institutional Review Board (IRB) processes and site activations are currently underway.
  • The Phase 1/2 FUNCTION-DMD clinical trial aims to enroll 3-5 patients in 2026, with initial safety data and early efficacy readouts—specifically, the percentage of near full-length dystrophin protein expression from muscle biopsies at 12 weeks—anticipated by year-end 2026.
• Event Details and Expert Participation:
  • The KOL event featured Dr. Aravindhan Veerapandiyan, a Pediatric Neurologist and Associate Professor at University of Arkansas for Medical Sciences and Arkansas Children’s Hospital, and Pat Furlong, Founding President of Parent Project Muscular Dystrophy.
  • Experts discussed current treatment landscapes, ongoing unmet needs, and the promise of gene editing approaches for DMD.
• Robust Preclinical and Clinical Rationale:
  • Preclinical data indicate PBGENE-DMD delivers durable dystrophin protein expression, improved muscle integrity, and significant increases in muscle function in DMD mouse models—including up to 85% dystrophin-positive myofibers and functional improvements lasting up to 9 months post-dosing.
  • Non-human primate (NHP) studies show strong AAV transduction and no observed off-target editing above background levels at human conserved potential off-target sites, supporting the program’s safety and efficacy profile at the clinical dose (1 x 10¹⁴ vg/kg).
• Market Opportunity and Competitive Positioning:
  • The global DMD market is estimated at \$8 billion with a projected 20% annual growth rate through 2030.
  • PBGENE-DMD’s design and clinical approach may offer a best-in-class therapeutic profile, addressing a broader patient segment, with potential for long-term durability and single-dose administration.
  • The program will be investigated at lower or equal vector doses compared to other AAV-based DMD therapies in development or approved, potentially mitigating dose-related toxicity risks.
• Financial Position and Runway:
  • As of year-end 2025, Precision BioSciences reported approximately \$137 million in cash, cash equivalents, and restricted cash.
  • With expected milestone proceeds and disciplined fiscal management, the company forecasts a cash runway extending through at least the end of 2028, covering multiple clinical catalysts.

Potentially Price Sensitive Information for Shareholders

• Clinical Milestones in 2026: Initial patient data from the FUNCTION-DMD trial (safety and early efficacy) is expected by the end of 2026. Positive results could significantly de-risk the program and drive share price appreciation.
• Regulatory Progress: FDA IND clearance and ongoing site activations position PBGENE-DMD as a front-runner in gene editing for DMD, and continued progress may attract investor interest and potential partnership opportunities.
• Financial Stability: The disclosed cash runway through 2028 reduces near-term financing risk and supports the company’s ability to reach key inflection points without dilutive capital raises in the immediate future.
• Competitive Differentiation: If PBGENE-DMD demonstrates the predicted best-in-class profile, it could capture significant market share in a rapidly expanding global DMD market.

Additional Information for Investors

• Forward-Looking Statements: The company cautions that forward-looking statements are subject to numerous risks and uncertainties, including the ability to execute clinical plans, obtain regulatory approvals, and achieve commercial success.
• Presentation Materials: The full KOL event presentation is available as Exhibit 99.1 on the company’s investor relations website, offering further details on the science, clinical rationale, and market opportunity.
• Other Programs: Precision BioSciences is also advancing programs in hepatitis B virus (HBV) and other indications, with additional clinical data expected from ongoing trials in 2026.

Conclusion

The developments disclosed by Precision BioSciences represent a potentially significant inflection point for the company. The advance of the PBGENE-DMD program into the clinic, the anticipated initial patient data in 2026, and the company’s solid financial footing all combine to make this a newsworthy update for current and prospective shareholders. Positive clinical outcomes could materially impact the company’s valuation and competitive position in the high-growth DMD space.

Disclaimer: This article is for informational purposes only and does not constitute investment advice. Forward-looking statements are subject to risks as detailed in Precision BioSciences’ filings with the SEC. Investors should conduct their own due diligence before making investment decisions.

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