Preclinical Research with ARCUS Genome Editing Rejuvenated Rod Photoreceptor Structure and Function in a Transgenic Pig Model of Human Blindness
DURHAM, N.C.--(BUSINESS WIRE)--May 3, 2021-- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, announced today that the following paper presentation, highlighting preclinical research using its ARCUS® genome editing platform for autosomal dominant Retinitis Pigmentosa (adRP), will be presented today at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting.
Title: Rho 1-2 meganuclease, an allele-specific gene-editing therapy, rejuvenates rod photoreceptor structure and function in a pig model of autosomal dominant Retinitis Pigmentosa (adRP)
Paper Presentation: Gene Therapy - Physiology/Pharmacology, Abstract 3543926
Date/Time: Monday, May 3, 2021, 4:30 – 6:00 p.m. EDT
Presenting Author: Archana Jalligampala, Ph.D., Postdoctoral Associate, Department of Ophthalmology and Visual Sciences, University of Louisville
Co-Authors: Jennifer Noel1, James W. Fransen1, Wei Wang1, Maha H. Jabbar1, Nazarul Hasan1, Gobinda Pangeni1, Bhubanananda Sahu1, Whitney Lewis2, Jeff Smith2, Victor Bartsevich2, Kristi Viles2, Derek Jantz2, Maureen A. McCall1
The P23H mutation in the rhodopsin gene represents the most common form of adRP in North Americans. Mutations in the rhodopsin gene account for 25 to 30 percent of all cases of adRP in the United States. This leads to the progressive loss of rods, which are responsible for vision at low light levels.
“This form of adRP, an inherited eye disease that leads to complete loss of central vision, is not amenable to conventional gene replacement therapies,” said Archana Jalligampala, Ph.D., Postdoctoral Associate, Department of Ophthalmology and Visual Sciences, University of Louisville. “We used ARCUS nucleases to target the P23H human rhodopsin mutation and found that, at our latest scheduled structural and functional assessment at 44 weeks post-injection, rods in the treated P23H retinas were more numerous, had elongated outer segments, and correctly localized rhodopsin compared to the untreated area in the same P23H retina or to an untreated P23H retina. We’re very encouraged by these results as they indicate that the gene editing approach using ARCUS has the potential to treat adRP in human patients with the same point mutation and, importantly, at late disease stages.”
In this study, ARCUS nuclease-treated animals showed significant improvements in rod driven signals, namely an increase in electroretinography (ERG) b-wave response compared to untreated animals. This effect was observed as early as postnatal day (P) 60 and was maintained through P300. These results suggest that the level of gene editing achieved in this study may facilitate functional visual improvements.
“We are excited to see that this latest large animal, preclinical dataset using ARCUS to address adRP will be presented at the annual ARVO meeting,” commented Derek Jantz, Ph.D., Co-Founder and Chief Scientific Officer at Precision BioSciences. “This work adds to the growing body of preclinical research where in vivo gene editing with ARCUS has resulted in sustained effective responses. We look forward to the continued development of ARCUS for rare genetic conditions like adRP that lack a safe and effective treatment option.”
Video-recorded presentations from the ARVO 2021 meeting are available on the Pathable online platform.
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.
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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.
1 University of Louisville, Louisville, KY, USA
2 Precision BioSciences, Inc., Durham, NC, USA
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