Precision BioSciences is at the forefront of genetic medicine, leveraging its proprietary ARCUS genome editing technology to develop innovative therapies aimed at treating challenging conditions, particularly rare genetic diseases. The ARCUS platform utilizes sequence-specific DNA-cutting enzymes, known as nucleases, to perform precise modifications in the DNA of living cells. This technology enables the insertion, removal, or repair of genetic material, offering a potential pathway to cure genetic disorders. The company is actively advancing its ARCUS technology through various preclinical and clinical programs, including efforts to address chronic hepatitis B and mitochondrial diseases.

In addition to its core genome editing capabilities, Precision BioSciences is exploring the application of ARCUS in various therapeutic contexts, such as the treatment of Duchenne muscular dystrophy and primary mitochondrial myopathy. The company has reported promising preclinical data demonstrating the efficacy and safety of its gene editing approaches, which are designed to achieve high efficiency in gene insertion and excision. By focusing on targeted gene therapies, Precision BioSciences aims to restore hope for patients suffering from genetic diseases, positioning itself as a leader in the rapidly evolving field of genetic medicine.

United States

Precision BioSciences, Inc. (Nasdaq: DTIL), is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform designed with therapeutic safety, delivery, and control in mind. Using ARCUS, Precision is developing allogeneic CAR T and in vivo gene correction therapies for cancer and infectious diseases.

Cancer

Infectious Diseases

Precision BioSciences

B-cell lymphoma

Diffuse large B cell lymphoma

CAR T

Azer-cel (PBCAR0191) (azercabtagene zapreleucel)

Non-Hodgkin lymphoma

PBCAR19B is a next-generation, stealth cell candidate for patients with CD19-positive malignancies such as relapsed/refractory Non-Hodgkin Lymphoma. PBCAR19B is designed to improve the persistence of allogeneic CAR T cells following infusion by preventing rejection by T cells and natural killer (NK) cells. In addition to the CAR gene, the stealth vector carries a short hairpin RNA (shRNA) that suppresses expression of beta-2 microglobulin (B2M), a component of Class 1 major histocompatibility complex (MHC) molecules found on the cell surface. Reducing or knocking down Class 1 expression on allogeneic CAR T cells has been shown to reduce CAR T cell killing by cytotoxic T cells. The stealth vector also carries a HLA-E gene intended to prevent rejection of CAR T cells by NK cells that can be stimulated as a result of reduced MHC molecule expression on the cell surface.
CAR T

PBCAR19B (CD19 Allogeneic CAR-T)

Ornithine transcarbmylase deficiency

ECUR-506 comprises two vectors, an ARCUS® nuclease vector targeting gene editing in the well-characterized PCSK9 gene locus and a donor vector that inserts the desired functional OTC gene. iECURE has licensed the ARCUS nuclease for ECUR-506 from Precision BioSciences (Nasdaq: DTIL). The cut in the PCSK9 site serves as the insertion site for the OTC gene, providing a potential path to permanent expression of a functional gene.

ECUR-506

Chronic hepatitis B

Liver disease

Viral infection

PBGENE-HBV is a highly specific, novel therapeutic approach to treating patients with chronic HBV infection. It is designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures.&nbsp;
 
Chronic hepatitis B infection is primarily driven by persistence of HBV cccDNA and integration of HBV DNA into the human genome in liver cells, the primary source of HBsAg in late-stage disease. Current treatments for patients with HBV infection include agents that result in long-term viral suppression as indicated by reduction of circulating HBV DNA, but these therapies do not eradicate HBV cccDNA, rarely lead to functional cure, and require lifelong administration.

PBGENE-HBV

Multiple myeloma

Precision is advancing a pipeline of cell-phenotype optimized allogeneic CAR T therapies, leveraging fully scaled, proprietary manufacturing processes. The platform is designed to maximize the number of patients who can potentially benefit from CAR T therapy. Precision carefully selects high-quality T cells derived from healthy donors as starting material, then utilizes its unique ARCUS genome editing technology to modify the cells via a single-step engineering process. By inserting the CAR gene at the T cell receptor (TCR) locus, this process knocks in the CAR while knocking out the TCR, creating a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease.
CAR T

PBCAR269A (BCMA) 

Chronic lymphocytic leukemia

Precision is advancing a pipeline of cell-phenotype optimized allogeneic CAR T therapies, leveraging fully scaled, proprietary manufacturing processes. The platform is designed to maximize the number of patients who can potentially benefit from CAR T therapy. Precision carefully selects high-quality T cells derived from healthy donors as starting material, then utilizes its unique ARCUS genome editing technology to modify the cells via a single-step engineering process. By inserting the CAR gene at the T cell receptor (TCR) locus, this process knocks in the CAR while knocking out the TCR, creating a consistent product that can be reliably and rapidly manufactured and is designed to prevent graft-versus-host disease.
CAR T

Precision BioSciences

Company Details