Announcement • May 15

Precision Biosciences Presents New Preclinical Data Supporting Advancement of Pbgene-Dmd into Clinic

Precision BioSciences, Inc. announced new preclinical data from its PBGENE-DMD program in an oral presentation at the American Society of Gene & Cell Therapy 2026 Annual Meeting in Boston, Massachusetts. The new data show that treatment with PBGENE-DMD in early-juvenile mice resulted in significantly higher efficacy across key skeletal and respiratory muscles than treatment in late-juvenile mice over a comparable timeframe. This new data further supports evaluating PBGENE-DMD in younger DMD patient populations, including the 2- to 3-year-old patients, who are a key demographic of the ongoing Phase 1/2 FUNCTION-DMD trial evaluating PBGENE-DMD in boys ages 2 to 7. The greater efficacy observed in early-juvenile mice is also expected to be an important point of differentiation for PBGENE-DMD versus microdystrophin approaches. With microdystrophin approaches the AAV dilution effect driven by muscle growth and turnover would be expected to be even more pronounced in younger DMD patients. New preclinical data: strong efficacy in early-juvenile mice (age 2 weeks, equivalent to a patient population aged 2–3 years) PBGENE-DMD in early-juvenile mice achieved up to 3x higher dystrophin protein restoration in skeletal muscle, and up to 12x higher dystrophin protein restoration in respiratory muscle, compared with late-juvenile mice at equivalent dose levels. Strong efficacy which exceeded the expected dystrophin protein restoration therapeutic threshold of 5% was observed in respiratory muscle tissues, with mice achieving up to 12% dystrophin restoration in the diaphragm and up to 30% in the intercostals, muscles whose function is critical to preventing respiratory failure in patients with DMD. PBGENE-DMD drove high levels of dystrophin-positive fibers in early-juvenile mice, with levels 2–3x higher in skeletal and respiratory muscle tissues than in late-juvenile mice after three months, reaching up to 70% dystrophin-positive fibers. Similar therapeutic efficacy was achieved in cardiac muscle in both early- and late-juvenile mice. These new data extend Precision's previously reported preclinical findings supporting the safety, efficacy, and durability profile of PBGENE-DMD: A toxicology study in a humanized DMD mouse model showed that PBGENE-DMD treatment led to greater than 45% reduction in serum creatine kinase across multiple dose levels, alongside improvements in muscle pathology relative to vehicle-treated controls, supporting the safety profile of the program. Beyond safety, PBGENE-DMD has demonstrated sustained efficacy over time through dystrophin protein restoration and dystrophin-positive myofibers, translating into durable muscle function. Treated mice maintained up to 92% of the maximum force output of non-diseased animals, while untreated, diseased mice showed progressively declining force output. 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 Mischler, Ph.D., DMD Research Lead, Precision BioSciences Date and time: Thursday, May 14, 2026, 8:45 A.M. ET 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. Compared with DMD, deletion of exons 45-55 is often associated with a more mild prognosis for patients. This protein more closely resembles normal dystrophin than synthetic, truncated microdystrophin approaches, which currently offer minimal proven 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 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.

Announcement • Nov 11

+ 1 more update

Precision BioSciences, Inc. Presents Late-Breaking Phase 1 PBGENE-HBV Data at AASLD The Liver Meeting Showing Safety, Tolerability and Cumulative, Dose-Dependent Antiviral Activity in First Three Cohorts

Precision BioSciences, Inc. announced a late-breaking oral presentation at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting 2025. The presentation includes data from the ongoing ELIMINATE-B Phase 1 study evaluating PBGENE-HBV, a first-in-class in vivo gene editing therapy designed to eliminate cccDNA, the root cause of chronic Hepatitis B, and inactivate integrated HBV DNA. AASLD Presentation Shows Evidence of Antiviral Activity and Favorable Safety Profile As of the October 31, 2025 data cutoff date, nine evaluable patients have been dosed across three ascending cohorts (0.2, 0.4, and 0.8 mg/kg), with a total of 22 administered doses. Key clinical findings from the late-breaking presentation include: · Consistent antiviral activity across all treated patients regardless of baseline HBsAg Every participant receiving PBGENE-HBV exhibited measurable reductions in hepatitis B surface antigen (HBsAg) following treatment, confirming on-target antiviral effects across all dose levels. Importantly, consistent levels of antiviral activity were observed in patients regardless of baseline HBsAg levels ranging from 370 to 11,813 IU/mL, with no upper limit in the study. The magnitude and persistence of these declines increased with higher doses, providing evidence of cumulative antiviral activity from pre-planned repeat administrations without cumulative toxicities. · Durable HBsAg reductions sustained over time In Cohort 1 (0.2 mg/kg), there was evidence of antiviral activity in all three patients with one patient showing a durable ~50% reduction in HBsAg 9 months after the initial dose of PBGENE-HBV and holding. In Cohort 2 (0.4 mg/kg), all three patients achieved durable HBsAg declines that were maintained for 8 weeks after the first administration and continue to show durable suppression following the third dose administration with HBsAg reductions up to 66%. Demonstrated dose-dependent antiviral activity Cohort 3 (0.8 mg/kg) demonstrated further dose-responsive antiviral effects, with all three patients showing early (2 weeks) HBsAg reductions following the first dose, with additional administrations still planned to decrease HBsAg. One patient has received two administrations with a deepening response following the second dose, as evidenced by a 64% decrease in HBsAg without increases in HBsAg levels between dose administrations as was observed in lower dose cohorts. With all participants in Cohort 3 achieving substantially reduced HBsAg levels, as low as 188 IU/mL there is an emerging path to potentially stopping nucleos(t)ide analogs and testing for cure following additional planned administrations of PBGENE-HBV at this dose level. · Biopsy evidence of direct HBV DNA editing Unlike existing therapies, PBGENE-HBV is designed to target the source of viral infection by directly eliminating cccDNA and inactivating integrated HBV DNA. This approach has the potential to permanently halt viral transcription and silence antigen production, with the goal of achieving complete cccDNA eradication and cure. paired liver biopsy from Patient 5 (part of Cohort 2) confirmed the presence of ARCUS-mediated gene editing events within viral DNA, marking the first direct molecular evidence of HBV viral gene editing in humans. Following two administrations at 0.4 mg/kg, this patient’s biopsy showed promising evidence of viral DNA editing by inactivation with insertion/deletion (indel) edits in viral DNA, further supporting the observed clinical antiviral effects and the PBGENE-HBV mechanism of cccDNA elimination and integrated HBV DNA inactivation. Continuing reductions of HBsAg were observed after the biopsy was taken due to the third dose of PBGENE-HBV, suggesting cumulative gene editing of the viral genome. · Favorable safety and tolerability profile PBGENE-HBV was well tolerated across all cohorts, with no observed dose-limiting toxicities. Adverse events were transient and generally resolved within 12 hours. Transient infusion-related reactions resolved without intervention and were deemed not dose-limiting by the independent data safety monitoring committee. Platelet fluctuations were transient and asymptomatic. · Stable liver enzyme laboratory values across repeat administrations Transient elevations in alanine aminotransferase (ALT) and aspartate aminotransferase (AST) which resolved within days of the infusion were observed shortly after dose administrations of PBGENE-HBV across all dose levels. Transaminase elevations were transient with no associated changes in bilirubin and no evidence of liver dysfunction. There was one case of reversible Grade 3 AST elevation without any associated bilirubin elevation that resolved within 3 days. The data were reviewed by the independent data monitoring committee and an independent liver safety committee, an expert group of global hepatologists, and deemed not dose-limiting. Collectively, these findings support the potential tolerability and hepatic safety for repeat administrations to drive antiviral responses.

Interest coverage ratio	n/a
Cash	US$101.18m
Equity	US$76.10m
Total liabilities	US$67.79m
Total assets	US$143.89m

Data	Last Updated (UTC time)
Company Analysis	2026/06/14 00:15
End of Day Share Price	2026/06/12 00:00
Earnings	2026/03/31
Annual Earnings	2025/12/31

Package	Data	Timeframe	Example US Source ^*
Company Financials	10 years	Income statement Cash flow statement Balance sheet	SEC Form 10-K SEC Form 10-Q
Analyst Consensus Estimates	+3 years	Forecast financials Analyst price targets	Analyst Research Reports Blue Matrix
Market Prices	30 years	Stock prices Dividends, Splits and Actions	ICE Market Data SEC Form S-1
Ownership	10 years	Top shareholders Insider trading	SEC Form 4 SEC Form 13D
Management	10 years	Leadership team Board of directors	SEC Form 10-K SEC Form DEF 14A
Key Developments	10 years	Company announcements	SEC Form 8-K

Analyst	Institution
Debjit Chattopadhyay	Guggenheim Securities, LLC
Debjit Chattopadhyay	H.C. Wainwright & Co.
Patrick Trucchio	H.C. Wainwright & Co.

Precision BioSciences Balance Sheet Health

Financial Health criteria checks 5/6

Key information

Recent financial health updates

Precision BioSciences Receives Approval from The Nasdaq Stock Market LLC to Transfer the Listing of its Common Stock from the Nasdaq Global Select Market to the Nasdaq Capital Market

Is Precision BioSciences (NASDAQ:DTIL) Using Debt In A Risky Way?

Is Precision BioSciences (NASDAQ:DTIL) Weighed On By Its Debt Load?

Recent updates

Price target decreased by 8.9% to US$29.60

Precision Biosciences Presents New Preclinical Data Supporting Advancement of Pbgene-Dmd into Clinic

Analyst Forecasts For Precision BioSciences, Inc. (NASDAQ:DTIL) Are Surging Higher

Consensus revenue estimates increase by 64%

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New minor risk - Share price stability

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Full year 2025 earnings: EPS and revenues exceed analyst expectations

New major risk - Revenue and earnings growth

Consensus EPS estimates fall by 30%

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Precision BioSciences Presents Preclinical PBGENE-DMD Data Highlighting Durable Dystrophin Expression and Functional Benefit

President notifies of intention to sell stock

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President notifies of intention to sell stock

Precision Biosciences Sets Strategic Priorities for 2026 Focused on Continued Advancement of Clinical-Stage Programs Pbgene-Hbv for Chronic Hepatitis B and Pbgene-Dmd for Duchenne Muscular Dystrophy

New minor risk - Market cap size

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New minor risk - Share price stability

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Precision BioSciences, Inc., Annual General Meeting, Jun 04, 2024

Precision BioSciences, Inc. to Report Q4, 2023 Results on Mar 27, 2024

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Price target decreased by 15% to US$2.60

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Analysts Just Made A Meaningful Upgrade To Their Precision BioSciences, Inc. (NASDAQ:DTIL) Forecasts

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