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Precision’s gene editing event will feature presentations from Company management as well as key opinion leaders. The R&D Day event will reflect Precision’s go-forward singular focus on in vivo gene editing through ARCUS, its proprietary, wholly owned genome editing platform. The agenda will include an overview of the broad potential and differentiation of ARCUS, new pre-clinical data, and timelines for leading in vivo gene editing programs.

Wholly Owned Development Programs

Given the unique advantages for ARCUS, including the cut, size, and simplicity, program development will focus on sophisticated edits. Wholly owned programs include:

PBGENE-HBV
is being developed for the treatment of patients with chronic hepatitis B, with submission of a clinical trial application (CTA) and/or investigational new drug (IND) application targeted for 2024. During the R&D Day, Precision will show data highlighting the potential of PBGENE-HBV to eliminate covalently closed circular DNA and inactivate integrated hepatitis B virus (HBV) DNA to drive durable antigen loss, leading to a potential functional cure.

HBV Expert Perspectives

PBGENE-PMM
is being developed as a first-in-class opportunity for treatment of m.3243 associated primary mitochondrial myopathy. Mitochondrial diseases are the most common hereditary metabolic disorder, affecting 1 in 4,300 people. PMM currently lacks a curative treatment and impacts approximately 50% of patients navigating mitochondrial disease. The high specificity and single component nature of Precision’s mitoARCUS nucleases are designed to enable specific editing of mutant mitochondrial DNA while allowing the normal (wild-type) mitochondrial DNA to repopulate in the mitochondria and restore normal function. Precision expects to file a CTA and/or IND in 2025.

Mitochondrial Myopathy Expert Perspectives

Presentation

Speakers