Precision BioSciences, Inc. is a biotechnology company, which engages in the development of genome editing technologies. It operates through the Therapeutics and Food segments. The Therapeutics segment focuses on the development of products in the field of immuno-oncology and of novel products outside immuno-oncology to treat human diseases. The Food segment applies ARCUS, the company's proprietary genome editing platform, to develop food and nutrition products through collaboration agreements with consumer-facing companies. The company was founded by Derek N. Jantz, Jeff Smith, and Matthew R. Kane on January 26, 2006 and is headquartered in Durham, NC.
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- Precision BioSciences dosed the first patient in the Phase 1/2 FUNCTION-DMD trial evaluating PBGENE-DMD, the first clinical gene-editing program for Duchenne muscular dystrophy (DMD). - PBGENE-DMD uses two complementary ARCUS nucleases in a single AAV to excise exons 45–55, aiming to restore near full-length functional dystrophin rather than truncated microdystrophin. - The trial enrolls ambulatory DMD patients aged 2–7 with mutations between exons 45 and 55, a population representing up to 60% of boys living with DMD. - Initial safety data are expected by year-end 2026, following FDA Orphan Drug, Fast Track, and Rare Pediatric Disease designations.
- The global gene editing therapeutics market is projected to grow at a compound annual growth rate of 12.5%, fueled by expanding clinical applications and rising disease burden. - CRISPR-based technologies dominate the market due to their efficiency and cost-effectiveness, while oncology represents the largest therapeutic application segment. - High development costs, regulatory complexity, and off-target safety concerns remain significant barriers to broader adoption and commercialization. - Key players including CRISPR Therapeutics, Intellia Therapeutics, Editas Medicine, and Beam Therapeutics are driving innovation through partnerships and investments in next-generation editing platforms.
- Precision BioSciences' ARCUS gene-editing platform demonstrated exceptional efficiency with gene insertion rates exceeding 85% in T-lymphocytes and 40% in non-dividing human hepatocytes, according to a study published in Nucleic Acids Research. - The company's PBGENE-HBV program for hepatitis B treatment has initiated patient recruitment at its first U.S. clinical trial site as part of the Phase 1 ELIMINATE-B trial. - The PBGENE-DMD program targeting Duchenne muscular dystrophy showed compelling preclinical results, with treated mice exhibiting up to 93% of maximum force production capacity and 66% improvement in injury resistance. - Despite reporting net losses, the company has secured sufficient liquidity to fund operations through the second half of 2027, positioning it for potential sustainable growth as clinical programs mature.
- Primary hyperoxaluria pipeline features over 6 companies developing more than 6 therapeutic candidates, with notable advances in gene editing and RNA interference therapies. - Arbor Biotechnologies achieved a significant milestone in July 2025 with the first patient treated in their Phase 1/2 redePHine trial for ABO-101, a gene-editing therapy for PH1. - The FDA has approved two breakthrough therapies: nedosiran (Rivfloza) in September 2023 for PH1 patients with preserved kidney function, and lumasiran in November 2020 for both adults and children. - Leading pipeline candidates include CHK-336, BBP-711, BMN 255, Oxabact, and Nedosiran, spanning various development stages from preclinical to Phase III trials.
- Precision BioSciences' PBGENE-HBV demonstrated substantial antiviral activity in all three patients in the lowest dose cohort, achieving 47-69% reductions in hepatitis B surface antigen levels. - The gene editing therapy showed a well-tolerated safety profile with no serious adverse events or dose-limiting toxicities observed across multiple dose administrations. - One patient maintained a durable 50% reduction in HBsAg levels seven months after initial treatment, providing first clinical evidence of sustained viral DNA editing. - PBGENE-HBV represents the first and only clinical modality designed to eliminate covalently closed circular DNA and inactivate integrated DNA for potential hepatitis B cure.
- The CAR T-cell therapy pipeline has experienced significant growth, with over 180 companies actively developing more than 200 innovative cell therapy candidates across various stages of clinical development. - Recent breakthroughs include Hemogenyx's first human administration of HG-CT-1 for acute myeloid leukemia and NICE's approval of lisocabtagene maraleucel for large B-cell lymphoma treatment. - Strategic industry developments are accelerating progress, with companies like CARsgen Therapeutics forming alliances to advance allogeneic CAR-T products and multiple firms reporting successful trial milestones.
• TG Therapeutics has secured access to MaxCyte's flow electroporation technology to develop azercabtagene zapreleucel, an off-the-shelf CAR T-cell therapy for progressive multiple sclerosis. • The Phase 1 clinical trial will evaluate azer-cel in up to 32 patients with progressive MS forms, focusing on determining optimal dosing and safety profiles. • This innovative therapy targets CD19-expressing B-cells using donor-derived T-cells, potentially reducing MS-driving autoantibodies while avoiding graft-versus-host disease complications.
- iECURE's ECUR-506, an in vivo gene insertion therapy, demonstrated a complete clinical response in an infant with neonatal-onset Ornithine Transcarbamylase (OTC) deficiency. - The Phase 1/2 OTC-HOPE study showed that ECUR-506 was generally well-tolerated, with no significant clinical safety concerns, offering potential lasting benefits. - Treatment allowed discontinuation of ammonia scavenger medication and increased protein intake to age-appropriate levels, maintaining normal ammonia levels for six months. - Precision BioSciences' ARCUS gene editing platform, utilized in ECUR-506, shows promise for treating genetic diseases, boosting confidence in its therapeutic potential.
- Precision BioSciences' PBGENE-HBV demonstrated a favorable safety profile in initial Phase 1 trial results, with no serious adverse events reported in the first cohort. - Early data indicates substantial reduction in Hepatitis B surface antigen (HBsAg) in two of three participants after a single dose administration. - The ELIMINATE-B trial is expanding globally, with active recruitment in Moldova, Hong Kong, and New Zealand, and plans for U.S. and U.K. expansion. - PBGENE-HBV leverages the ARCUS platform to target and eliminate covalently closed circular DNA (cccDNA), the root cause of chronic Hepatitis B.
• Precision BioSciences received approval in Moldova for a clinical trial of PBGENE-HBV, an in vivo gene editing therapy targeting chronic hepatitis B. • PBGENE-HBV aims to eliminate covalently closed circular DNA (cccDNA), the viral reservoir, offering a potential functional cure for HBV. • The trial will commence with patient dosing in Moldova, with plans to expand internationally to gather safety and efficacy data. • Political factors in Moldova could influence the trial's progress, necessitating vigilance from Precision BioSciences to maintain operational stability.
