Precision BioSciences to Present at UMDF 2024 on PBGENE-PMM Progress Towards 2025 IND/CTA

Precision BioSciences, Inc. (Nasdaq: DTIL), a leader in gene editing technology, is set to present groundbreaking data at the United Mitochondrial Disease Foundation’s (UMDF) Mitochondrial Medicine 2024 Conference in Cleveland, Ohio from June 26-29, 2024. The presentation will focus on the company’s innovative PBGENE-PMM program, which targets the m.3243 mitochondrial disease using Precision’s proprietary ARCUS® platform.

Jeff Smith, Co-Founder and Chief Research Officer at Precision BioSciences, expressed his team's dedication to advancing mitochondrial gene editing. He highlighted the unique aspects of ARCUS, a single-component editor that integrates recognition and catalytic activity in one protein, eliminating the need for a guide-RNA. This simplicity facilitates the elimination of mutant mitochondrial DNA while preserving normal mitochondrial DNA, resulting in improved mitochondrial function. Smith emphasized the potential of ARCUS to address the root causes of diseases and the promising data generated so far, with plans to move PBGENE-PMM towards an Investigational New Drug (IND) or Clinical Trial Application (CTA) in 2025.

The presentation, titled "Shifting Heteroplasmy with PBGENE-PMM: Gene Editing Therapy for m.3243A>G Associated Mitochondrial Myopathy," will be delivered by Wendy Shoop, PhD, Senior Scientist at Precision BioSciences, on Thursday, June 27, 2024, at 11:00 AM EDT. The data presented will underline ARCUS' capability to localize exclusively to mitochondria, avoiding off-target edits in the nuclear genome, and to significantly shift heteroplasmy and enhance mitochondrial function.

This research demonstrates ARCUS' ability to selectively eliminate various forms of mutant mitochondrial DNA, including the common deletion (del_mtDNA4977), mouse m.5024T, and human m.3243G. These findings underscore the potential of the ARCUS platform in treating heteroplasmic mitochondrial DNA mutations.

PBGENE-PMM is Precision BioSciences' leading mitochondrial editing therapeutic program, focusing on m.3243 mitochondrial disease, a prevalent hereditary metabolic disorder affecting 1 in 4,300 people. This disease, which currently lacks a definitive cure, impacts 30-40% of patients with mitochondrial disease. In a 2023 publication in Nature Metabolism, Precision BioSciences presented data showcasing the high specificity and single-component nature of PBGENE-PMM, which effectively edits and eliminates mutant mitochondrial DNA, allowing normal mitochondrial DNA to repopulate and restore cellular function. The company aims to submit a CTA and/or IND for this program in 2025.

ARCUS, Precision BioSciences’ proprietary genome editing technology, was discovered and developed by the company's scientists. It utilizes DNA-cutting enzymes, or nucleases, that are engineered to insert, excise, eliminate, or repair DNA in living cells and organisms. ARCUS is derived from the naturally occurring enzyme I-CreI, which evolved in the algae Chlamydomonas reinhardtii to make precise DNA cuts and promote gene insertion through homologous recombination. The ARCUS platform is protected by over 130 patents, highlighting its innovative approach to genome editing.

Precision BioSciences is an advanced gene editing company committed to leveraging its ARCUS® platform to develop lasting therapies for a wide range of genetic and infectious diseases that currently lack adequate treatments. The company's pipeline includes in vivo gene editing candidates designed to deliver durable cures by utilizing ARCUS for sophisticated gene edits, including DNA insertion, elimination, and excision.

With a focus on improving life through novel gene editing technologies, Precision BioSciences continues to advance its research and development efforts, aiming to bring transformative therapies to patients in need.