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Precision BioSciences Announces Preclinical Data Showcasing Premier In Vivo Gene Editing Capabilities at American Society of Gene & Cell Therapy Annual Meeting
Multiple Oral Presentations and Posters to Demonstrate Differentiated Attributes of ARCUS® Genome Editing Platform for Efficient Gene Insertion and Gene
About this update from Precision Biosciences, Inc.
[{"type":"text","content":"\nMultiple Oral Presentations and Posters to Demonstrate Differentiated Attributes of ARCUS® Genome Editing Platform for Efficient Gene Insertion and Gene Knockout\n\nNew Primary Hyperoxaluria Type 1 (PH1) Preclinical Data Demonstrate a Robust ARCUS Nuclease Optimization Process Leading to 98% Knockdown of HAO1 Protein in Non-human Primates (NHP)\n\n DURHAM, N.C.--(BUSINESS WIRE)--\nPrecision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today announced preclinical data will be presented this week on ARCUS genome editing including two oral presentations and two posters at the American Society of Gene & Cell Therapy (ASGCT), May 16-19, 2022 at the Walter E. Washington Convention Center in Washington, D.C.\n\n“The preclinical findings presented this week at the ASGCT conference are very encouraging and further support our novel gene editing approach with ARCUS and our plans to advance three wholly owned product candidates, PBGENE-PH1, PBGENE-HBV and PBGENE-PCSK9, to the clinic over the next three years,” said Derek Jantz, Ph.D., Chief Scientific Officer and Co-founder of Precision BioSciences. “Translating these preclinical results to the clinic will further validate ARCUS as a premier gene editing platform with the precision and versatility needed to develop novel therapeutics that aim to deliver functional cures for conditions including PH1 and chronic hepatitis B.”\n\nOral Presentations:\n\nAbstract #447: Targeting the Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to Eliminate Hepatitis B Virus In Vivo\n\nData from this preclinical study demonstrate Precision’s gene editing approach designed to eliminate hepatitis B virus (HBV). ARCUS efficiently targeted and degraded HBV covalently closed circular (cccDNA) by 85% and reduced expression of Hepatitis B Surface Antigen (HBsAg) by 77% in HBV-infected primary human hepatocytes (PHH). Similar levels of editing were achieved in novel mouse and NHP models following lipid nanoparticle (LNP) delivery of ARCUS mRNA, resulting in a 96% reduction in HBsAg in mice. These data suggest that LNP-delivered ARCUS mRNA is a promising approach and potential functional cure for chronic hepatitis B. Precision will continue developing its PBGENE-HBV product candidate using LNP de...