Precision BioSciences Reports Second Quarter 2025 Financial Results, Highlights Strong Clinical Progress in Gene Editing Programs, and Extends Cash Runway Into 2027
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical-stage biotechnology company harnessing its proprietary ARCUS® genome editing platform to develop transformative in vivo gene editing therapies for diseases with significant unmet medical need, today announced its financial results for the second quarter ended June 30, 2025, and provided a comprehensive business update outlining recent clinical progress, regulatory milestones, pipeline priorities, and corporate strategy.
CEO Commentary: Focused Execution and Clinical Momentum
“Our team continues to operate with focus, discipline, and urgency,” stated Michael Amoroso, Chief Executive Officer of Precision BioSciences. “We have made meaningful progress advancing our lead clinical program, Precision BioSciences PBGENE-HBV, while moving rapidly to prepare PBGENE-DMD for entry into the clinic. The initial Phase 1 safety and efficacy data from our first dosing cohort in the ELIMINATE-B trial establishes proof of activity for PBGENE-HBV in chronic hepatitis B — a condition for which a true cure remains elusive.”
Amoroso emphasized that all treated patients in Cohort 1 demonstrated a measurable antiviral response, with one-third showing durable effects consistent with the program’s intended mechanism: elimination of covalently closed circular DNA (cccDNA) — the persistent viral reservoir that drives chronic HBV infection.
“We are encouraged by the safety profile observed so far in Cohorts 1 and 2, which allowed the Data Monitoring Committee to authorize advancement to Cohort 3 at a higher dose,” Amoroso continued. “Meanwhile, our DMD program is progressing with urgency, and the receipt of Rare Pediatric Disease and Orphan Drug designations from the FDA highlights the critical need for innovative therapies in this space.”
Looking ahead, Amoroso confirmed that Precision BioSciences is reallocating resources to fully invest in PBGENE-HBV and PBGENE-DMD while executing cost reductions across non-core areas. These measures are projected to extend the company’s cash runway into the second half of 2027, enabling initiation of a Phase 2 HBV study and a potential pivotal trial for DMD.
Pipeline Update
Wholly-Owned In Vivo Gene Editing Programs
PBGENE-HBV: Viral Elimination Program for Chronic Hepatitis B
PBGENE-HBV is the first and only potentially curative gene editing program in the clinic specifically engineered to eliminate Precision BioSciences cccDNA and inactivate integrated HBV DNA — directly addressing the root cause of chronic infection.
The ongoing ELIMINATE-B Phase 1 trial is evaluating three ascending dose levels, with each patient receiving three administrations approximately eight weeks apart.
- Cohort 1 (0.2 mg/kg) enrolled three patients. PBGENE-HBV was well-tolerated, with no treatment-related adverse events above Grade 2, no dose-limiting toxicities, and no clinically significant lab abnormalities.
- All three patients achieved reductions in hepatitis B surface antigen (HBsAg) levels — 56%, 69%, and 47% — with one patient maintaining a ~50% reduction seven months post-treatment, signaling durable antiviral activity.
- Cohort 2 (0.4 mg/kg) initial safety data also demonstrated a favorable profile, with no serious dose-related toxicities. Precision BioSciences One patient experienced a transient infusion-related reaction that resolved within minutes and was deemed unrelated to dose level.
Given these results, the Data Monitoring Committee has authorized advancement to Cohort 3. Precision expects to complete Cohort 2 dosing and initiate Cohort 3 before the end of 2025, with a data update anticipated later in the year.
PBGENE-DMD: Muscle-Targeted Excision Program for Duchenne Muscular Dystrophy
DMD is a progressive, fatal genetic disorder caused by mutations in the dystrophin gene. Affecting approximately 15,000 patients in the U.S., it leads to loss of mobility and premature death. Existing treatments offer limited functional improvement.
PBGENE-DMD utilizes two complementary ARCUS nucleases, delivered via a single AAV vector, to excise exons 45–55 of the dystrophin gene. This approach aims to restore a near-full-length functional dystrophin protein, in contrast to truncated versions produced by other approaches.
Regulatory Milestones:
- Rare Pediatric Disease Designation (June 2025) — potentially qualifying for a Priority Review Voucher upon FDA approval.
- Orphan Drug Designation (July 2025) — reinforcing the program’s high unmet need.
Preclinical Highlights:
- At the 2025 ASGCT Annual Meeting, PBGENE-DMD demonstrated restoration of dystrophin production across skeletal and cardiac muscles in a humanized DMD mouse model.
- New July 2025 data revealed a three-fold increase in dystrophin-positive cells over nine months, with up to 85% positivity in gastrocnemius muscle, indicating robust and sustained gene editing.
PBGENE-3243: Mutant Mitochondrial DNA Elimination
This program targets m.3243-associated mitochondrial disease by selectively eliminating mutant mitochondrial DNA. Precision BioSciences Development is paused to prioritize HBV and DMD.
Partnered In Vivo Gene Editing Programs
iECURE-OTC: Gene Insertion for OTC Deficiency
The ECUR-506 program, led by iECURE, is in a Phase 1/2 OTC-HOPE trial for neonatal-onset ornithine transcarbamylase deficiency. Early data show complete clinical response in the first treated patient, sustained beyond one year of age. Enrollment is ongoing across the U.S., U.K., Australia, and Spain, with final data expected in 1H 2026.
PBGENE-NVS: Collaboration with Novartis
Precision BioSciences and Novartis are co-developing a custom ARCUS nuclease for in vivo gene insertion in hemoglobinopathies such as sickle cell disease and beta thalassemia, aiming to deliver a one-time therapy that overcomes limitations of ex vivo approaches.
Non-Core Ex Vivo Programs
Azer-Cel: Allogeneic CAR T for Oncology
Partner Imugene Limited reported in July 2025 that azer-cel achieved:
- 75% overall response rate in relapsed/refractory DLBCL
- 55% complete response rate
FDA Fast Track Designation has been granted, and Imugene plans an end-of-Phase 1 FDA meeting in Q4 2025 to discuss registrational trial design.
Leadership and Advisory Expansion
In July 2025, Mark Sulkowski, M.D., Professor of Medicine at Johns Hopkins University and an internationally recognized expert in hepatology and infectious diseases, expanded his advisory role with Precision. As Head Clinical Development Advisor, he will guide trial optimization for PBGENE-HBV and support planning for later-stage studies.
Financial Results for Q2 2025
Alex Kelly, Chief Financial Officer, highlighted Precision’s disciplined approach to cost control:“We have extended our cash runway into the second half of 2027 by reducing non-core expenses, pausing PBGENE-3243, and launching an operating efficiency program expected to lower annual cash operating expenses by ~$25 million in both 2026 and 2027 compared to 2025 levels.”
Key Financial Highlights:
- Cash Position: $84.8 million in cash, cash equivalents, and restricted cash as of June 30, 2025.
- Revenues: <$0.1 million (down from $49.9 million in Q2 2024 due to the conclusion of the Prevail Therapeutics collaboration in April 2024).
- R&D Expenses: $12.8 million (down from $17.2 million YoY), reflecting lower HBV manufacturing/toxicology costs and paused PBGENE-3243, partially offset by accelerated DMD spend.
- G&A Expenses: $9.1 million (up from $8.5 million YoY), driven by higher employee-related costs.
Strategic Funding Outlook:
Precision BioSciences anticipates supplementing its cash runway through:
- Non-dilutive business development collaborations.
- Monetization of non-core program royalties/milestones.
- Use of its ATM facility as needed.
Outlook and Strategic Priorities
Precision BioSciences enters the second half of 2025 with two wholly-owned programs positioned to deliver value-driving clinical milestones:
- PBGENE-HBV — advancing toward higher-dose cohorts and planning for Phase 2 initiation.
- PBGENE-DMD — moving toward IND/CTA filing by year-end and potential pivotal trial design.
With a Precision BioSciences strengthened balance sheet, streamlined operations, and regulatory recognition for both programs, the company remains committed to bringing curative gene editing therapies to patients in need while delivering long-term shareholder value.
