Company Description
Precision BioSciences, Inc. (Nasdaq: DTIL) is a clinical stage gene editing company in the biotechnology sector that focuses on in vivo genome editing for genetic and infectious diseases with high unmet medical need. The company is dedicated to improving life using its novel, proprietary ARCUS® genome editing platform, which it describes as differing from other technologies in the way it cuts DNA, its smaller size, and its simpler structure. According to company disclosures, key capabilities and differentiating characteristics of ARCUS nucleases may enable more intended and defined therapeutic outcomes across a range of sophisticated gene edits.
Precision BioSciences’ pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for a broad range of genetic and infectious diseases where no adequate treatments exist. The company’s work spans three main types of edits enabled by ARCUS: gene insertion (inserting DNA into a gene to cause expression or add function), elimination (removing a genome such as viral DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single adeno-associated virus, or AAV, vector).
Core ARCUS® Platform
The ARCUS platform is based on engineered homing endonucleases and is designed to support precise and efficient genome editing in both dividing and non-dividing cells. Company communications highlight that ARCUS can generate 3’ overhang ends at the DNA break, which has been shown to support homology-directed repair and high-frequency transgene insertion. Published work referenced by Precision describes ARCUS-mediated gene insertion, single base editing, specific small and large deletions, and replacement of large stretches of genomic DNA. These capabilities underpin Precision’s strategy to address diseases that may not be amenable to traditional gene therapy approaches.
ARCUS is being used by Precision and its partners to pursue multiple therapeutic strategies: gene insertion programs for metabolic and liver disorders, viral genome elimination programs for chronic infections, and gene excision programs for large structural corrections in disease-causing genes. The platform’s relatively small size and structure are positioned by the company as advantageous for delivery using viral and non-viral methods, including AAV vectors and lipid nanoparticles.
Wholly Owned In Vivo Programs
Precision BioSciences’ wholly owned pipeline includes clinical and preclinical in vivo gene editing candidates. A central focus is PBGENE-HBV, described as the company’s wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial called ELIMINATE-B. PBGENE-HBV is designed as a potentially curative treatment for chronic hepatitis B infection by directly targeting the viral templates that drive persistence of the disease.
PBGENE-HBV is specifically designed to eliminate covalently closed circular DNA (cccDNA), which the company identifies as the root cause of chronic hepatitis B, and to inactivate integrated HBV DNA in infected liver cells. Company updates describe PBGENE-HBV as the first and only potentially curative gene editing program to enter clinical investigation that is specifically designed with this dual mechanism. The program uses ARCUS nucleases delivered with lipid nanoparticle technology provided by Acuitas Therapeutics, Inc.
In the ELIMINATE-B Phase 1/2a study, PBGENE-HBV is being evaluated in HBeAg-negative chronic hepatitis B patients at clinical sites in locations including Hong Kong, New Zealand, the United States, Moldova, and, with regulatory approvals, additional sites such as the United Kingdom. The trial is designed to define the optimal dose, frequency, and number of dose administrations for safely eliminating cccDNA and inactivating integrated HBV DNA. Precision has reported dose-dependent antiviral activity, measurable reductions in hepatitis B surface antigen (HBsAg) across treated patients, biopsy evidence of ARCUS-mediated viral DNA editing, and a safety profile that has supported dose escalation and repeat dosing in multiple cohorts.
Another wholly owned program is PBGENE-DMD, described as a muscle-targeted excision program for Duchenne muscular dystrophy (DMD). PBGENE-DMD is designed to employ two complementary ARCUS nucleases delivered in a single AAV to excise exons 45–55 of the dystrophin gene. Company materials state that this approach aims to restore a near full-length functional dystrophin protein within the body that more closely resembles normal dystrophin, in contrast to synthetic, truncated dystrophin approaches. PBGENE-DMD is designed to address DMD patients with mutations in exons 45–55, which Precision notes impact a substantial proportion of boys with DMD.
Preclinical data shared by the company from a DMD mouse model have shown dystrophin protein detected in multiple muscles after PBGENE-DMD administration, increased dystrophin expression over time, and improvements in functional muscle output. Precision has described PBGENE-DMD as a first-in-class gene editing therapy that uses a gene excision approach and targets muscle satellite cells, with the goal of providing durable functional muscle improvement.
Partnered In Vivo Gene Editing Programs
Beyond its wholly owned programs, Precision BioSciences licenses its ARCUS technology to partners for additional in vivo applications. One example is ECUR-506, developed by iECURE, Inc., which uses an ARCUS nuclease licensed from Precision for neonatal onset ornithine transcarbamylase (OTC) deficiency. ECUR-506 is an in vivo targeted gene insertion therapy evaluated in the first-in-human OTC-HOPE study in male infants with genetically confirmed severe neonatal onset OTC deficiency.
In this program, two AAV vectors using the same capsid are used: one carrying an ARCUS nuclease designed to create an insertion site within the PCSK9 gene locus, and a donor vector carrying a functional copy of the OTC gene for targeted insertion. iECURE has reported a complete clinical response in the first infant treated at the lowest dose level, and ECUR-506 has received U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) designation, as well as other regulatory designations. Precision notes that iECURE has licensed ARCUS nucleases from the company for multiple gene insertion programs.
Non-Core Ex Vivo and CAR T Programs
Precision BioSciences also has non-core ex vivo programs based on its ARCUS platform. One highlighted program is azer-cel (azercabtagene zapreleucel), an allogeneic CAR T treatment originally created by Precision and now being developed by partners for oncology and autoimmune indications. Imugene Limited is developing azer-cel for diffuse large B-cell lymphoma in a Phase 1b clinical trial and has reported response rates and durable remissions in treated patients. TG Therapeutics is evaluating azer-cel in a Phase 1 clinical trial for progressive multiple sclerosis and other autoimmune diseases. Precision is eligible for milestone payments from these partnered efforts and has reported receiving milestone consideration tied to clinical progress.
Research, Intellectual Property, and Scientific Publications
Precision BioSciences emphasizes ongoing research to characterize and extend the capabilities of ARCUS. The company has highlighted a peer-reviewed publication in Nucleic Acids Research describing high-efficiency homology-directed insertion into the genome using engineered ARCUS nucleases. According to the company’s summary, the work demonstrates high-frequency transgene insertion in dividing T lymphocytes and substantial insertion rates in non-dividing primary human hepatocytes, along with applications for single base editing, specific deletions, and replacement of large genomic segments.
The company also reports building an intellectual property estate around ARCUS and specific therapeutic applications. For example, Precision announced issuance of a U.S. patent titled “Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B Virus genome,” with composition of matter claims encompassing the PBGENE-HBV ARCUS nuclease. Related patents have been granted in Europe and Hong Kong, supporting the company’s hepatitis B program.
Capital Markets and Corporate Status
Precision BioSciences’ common stock trades on Nasdaq under the ticker symbol DTIL. The company has used public equity offerings, at-the-market facilities, and milestone payments from partners to support its research and development activities. An 8-K filing dated November 12, 2025 describes an underwritten offering of common stock, pre-funded warrants, and warrants, with net proceeds intended to fund ongoing and planned research and development, working capital, and general corporate purposes.
Company disclosures describe Precision as a clinical stage organization, with programs at various stages of clinical and preclinical development and a focus on diseases where no adequate treatments exist. Management communications reference efforts to align clinical trial design with regulatory objectives, secure regulatory designations such as Breakthrough Therapy for PBGENE-HBV and Rare Pediatric Disease and Orphan Drug designations for PBGENE-DMD, and manage operating expenses and cash runway to support key clinical milestones.
Role Within Biotechnology and Gene Editing
Within the broader biotechnology and professional, scientific, and technical services sector, Precision BioSciences positions itself as a genome editing company focused on in vivo therapeutic applications. Its work centers on using ARCUS nucleases to directly edit DNA in patients’ cells, with programs targeting chronic viral infections such as hepatitis B, inherited muscle diseases such as DMD, and metabolic liver disorders such as OTC deficiency through partners.
The company’s disclosures emphasize that chronic hepatitis B remains a major cause of morbidity and mortality worldwide and that current antiviral therapies typically require lifelong administration without eradicating cccDNA. By designing PBGENE-HBV to eliminate cccDNA and inactivate integrated HBV DNA, Precision aims to address what it identifies as the underlying source of viral persistence. Similarly, for DMD, the company notes that existing therapies have not demonstrated durable and significant functional improvements over time, and PBGENE-DMD is designed to restore a near full-length dystrophin protein native to the human body.
Through its wholly owned and partnered programs, Precision BioSciences uses the same ARCUS platform to pursue gene insertion, elimination, and excision strategies across different disease areas. This platform-centric approach, as described in company materials, is intended to support a pipeline of in vivo gene editing candidates that could offer lasting therapeutic benefit in conditions where adequate treatments do not exist.