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Precision BioSciences Announces Presentation at UMDF Mitochondrial Medicine 2024 Supporting Advancement of PBGENE-PMM Program Towards IND and/or CTA in 2025
DURHAM, N.C.--(BUSINESS WIRE)-- Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform
About this update from Precision Biosciences, Inc.
[{"type":"text","content":" DURHAM, N.C.--(BUSINESS WIRE)--\nPrecision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, insertion, and excision, is presenting data today at the United Mitochondrial Disease Foundation’s (UMDF) Mitochondrial Medicine 2024 Conference being held in Cleveland, Ohio from June 26-29, 2024.\n\n\n“Our team is proud to share details from our lead PBGENE-PMM program targeting m.3243 mitochondrial disease and showcase the broader applicability of ARCUS to target other mitochondrial mutations,” said Jeff Smith, Co-Founder and Chief Research Officer at Precision Biosciences. “Our work targeting mitochondrial DNA takes advantage of the simplicity of ARCUS as a single component editor with both recognition and catalytic activity all in one protein that does not require a guide-RNA like most other gene editing tools. PBGENE-PMM is designed to eliminate mutant mitochondrial DNA leaving normal functioning wild-type mitochondrial DNA intact to repopulate the cell, resulting in a shift in heteroplasmy and improvement in mitochondrial function. Given ARCUS’ ability to target the root cause of disease, we are excited by our data generated to date and are working diligently to advance PBGENE-PMM towards an Investigational New drug (IND) and/or Clinical Trial Application (CTA) in 2025.”\n\n\nPresentation Details:\nTitle: Shifting Heteroplasmy with PBGENE-PMM: Gene Editing Therapy for m.3243A>G Associated Mitochondrial Myopathy\nPresenter: Wendy Shoop, PhD, Senior Scientist, Precision Biosciences\nDate and Time: Thursday, June 27, 2024, 11:00 AM EDT.\n\n\nThe data for PBGENE-PMM, Precision’s lead mitochondrial editing therapeutic program, demonstrated ARCUS’ ability to localize exclusively to mitochondria, avoiding any detectable off-target editing in the nuclear genome, and generate substantial shifts in heteroplasmy and improvements in mitochondrial function.\n\n\nThe data showcases the ability of ARCUS to selectively eliminate mutant mitochondrial DNA, including the common deletion (del_mtDNA4977), mouse m.5024T, and human m.3243G, which highlights the broad applicability of the ARCUS platform for heteroplasmic mitochondrial DNA mutations.\n\n\nAbout PBGENE-PMM\n\n\nPBGENE-PMM is o...