Precision Biosciences Inc. (PBS0)

Overview

Precision BioSciences, Inc. is a pioneering entity in the field of advanced gene editing, concentrating its efforts on developing in vivo gene editing therapies. The company stands out for its cutting-edge ARCUS genome editing platform, which facilitates precise DNA genome modification processes, including insertion, deletion, and repair. Having been established in 2006, Precision BioSciences is rooted in Durham, North Carolina, where it spearheads innovative solutions targeting a range of genetic disorders and diseases. Its comprehensive approach extends beyond mere development, encompassing collaborative partnerships with leading biotechnology and pharmaceutical entities to expedite the availability of groundbreaking therapies for various conditions. Such collaborations span agreements with Caribou Biosciences, Inc., TG Cell Therapy, Inc., Eli Lilly and Company, Cellectis S.A., iECURE, Inc., Duke University, and Novartis Pharma AG, illustrating its commitment to leveraging collective expertise for the advancement of in vivo gene editing technologies.

Products and Services

• PBGENE-HBV

Focuses on the treatment of chronic hepatitis B virus (HBV) infection, aiming to eliminate covalently closed circular DNA and to inactivate integrated HBV DNA. This approach seeks to achieve long-lasting reductions in hepatitis B surface antigen, potentially transforming the therapeutic landscape for individuals living with chronic HBV.

• PBGENE-PMM

Targeted towards the treatment of m.3243 associated primary mitochondrial myopathy (PMM), Precision BioSciences is preparing for regulatory submissions such as an IND and/or CTA. This therapy reflects the company’s drive to address genetic disorders at their root, offering hope to patients afflicted by PMM.

• PBGENE-NVS

Designed for sickle cell disease/beta thalassemia, this product underscores the company’s commitment to tackling hereditary blood disorders. The focus is on gene insertion techniques as a means to provide lasting therapeutic outcomes for patients suffering from these debilitating conditions.

• PBGENE-DMD

This product aims at addressing Duchenne Muscular Dystrophy through gene excision strategies. By targeting specific genetic anomalies, PBGENE-DMD represents a beacon of hope for patients and families grappling with this progressive muscular disorder.

• PBGENE-LL2 and PBGENE-LL3

Both products exemplify Precision BioSciences' versatility, with PBGENE-LL2 being liver-directed and PBGENE-LL3 targeting the central nervous system. These initiatives highlight the company’s commitment to diversifying its therapeutic portfolio to cater to a broader spectrum of genetic disorders.

• iECURE-OTC

Focused on treating ornithine transcarbamylase deficiency, this insertion-based therapy is part of a broader collaboration with iECURE, Inc. It underscores the potential of ARCUS-based gene-insertion therapies to revolutionize the treatment of metabolic disorders.