Preclinical Study Shows ARCUS Genome Editing May Be a Promising Approach for the Treatment of Transthyretin Amyloidosis
DURHAM, N.C.–(BUSINESS WIRE)–Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, today announced that the following poster, highlighting a preclinical research collaboration using its ARCUS® genome editing platform for treatment of transthyretin amyloidosis (ATTR), will be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, scheduled for May 11-14, 2021.
Title: Translation of an AAV-delivered gene editing approach for transthyretin amyloidosis in animal models
Poster Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases, Abstract 497
Date/Time: Tuesday May 11, 2021 8:00 AM – 10:00 AM
Presenting Author: Jenny A. Greig, Ph.D., Senior Director, Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania
Co-Authors: Cassandra Gorsuch2, Joanna K. Chorazeczewski1, Melanie K. Smith1, Thomas Furmanak1, Alexa N. Avitto1, Scott N. Ashley1, Wendy Sharer2, Hui Li2, Jeff Smith2, Peter Clark1, Camilo Breton1, Derek Jantz2, and James M. Wilson1
Transthyretin amyloidosis is a rare disease caused by the progressive accumulation of misfolded transthyretin (TTR) protein into amyloid fibrils, which leads to peripheral neuropathy and/or cardiomyopathy. Research to be presented at the annual ASGCT meeting, led by Dr. Jenny A. Greig at the Perelman School of Medicine, University of Pennsylvania, used an AAV vector for in vivo delivery of ARCUS gene editing nucleases to knock out the TTR gene, which is responsible for ATTR.
“With this program, we are excited to continue building a dataset demonstrating in vivo gene editing in large animal models using ARCUS nucleases,” said Derek Jantz, Ph.D., Chief Scientific Officer and Co-Founder of Precision BioSciences. “In this study, use of an optimized ARCUS nuclease to knock out the TTR gene was found to be effective in both mice and nonhuman primates, where we observed a good correlation between TTR gene editing in the liver and reductions of TTR in the serum. This approach addresses the root cause of the disease and results in genomic edits that are expected to be permanent. These results continue to demonstrate the power and versatility of ARCUS nucleases, particularly for in vivo editing.”
Abstracts for the ASGCT 2021 Meeting are available on the meeting website.
ARCUS® is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA. Precision’s platform and products are protected by a comprehensive portfolio including more than 75 patents to date.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Company’s pipeline consists of multiple “off-the-shelf” CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding statements regarding the potential results, uses and advancement of our in vivo gene editing programs and ARCUS-based gene editing technology, including, without limitation, its attributes and effects upon the transthyretin gene, transthyretin serum levels and transthyretin amyloidosis. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “believe,” “could,” “eligible,” “expect,” “expected”, “should,” “plan,” “intend,” “estimate,” “target,” “mission,” “goal,” “may,” “will,” “would,” “should,” “could,” “target,” “potential,” “potentially”, “promising”, “project,” “predict,” “contemplate,” “potential,” or the negative thereof and similar words and expressions.
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1 Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA
2 Precision BioSciences, Inc., Durham, NC, USA
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