Precision BioSciences Announces Paper Presentation at the Association for Research in Vision and Ophthalmology 2021 Annual Meeting

Rhea-AI Summary

Precision BioSciences, Inc. (Nasdaq: DTIL) announced a preclinical study presentation at the ARVO Annual Meeting, focusing on ARCUS genome editing for autosomal dominant Retinitis Pigmentosa (adRP). The study demonstrates that ARCUS can rejuvenate rod photoreceptor structure and function in a pig model with the common P23H rhodopsin mutation. Significant improvements in rod-driven signals were observed, suggesting potential for late-stage treatment in humans. This research adds to growing preclinical support for ARCUS in addressing rare genetic conditions.

Positive

• Significant improvements in rod-driven signals in treated animals suggest potential for effective human treatments.
• The preclinical study demonstrates the potential of ARCUS technology in addressing late-stage adRP.

Negative

• None.

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.

About ARCUS

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.

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 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 autosomal dominant Retinitis Pigmentosa (adRP). In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “believe,” “could,” “eligible,” “expect,” “should,” “plan,” “intend,” “estimate,” “target,” “mission,” “goal,” “may,”, “suggest”, “will,” “would,” “should,” “could,” “target,” “potential,” “project,” “predict,” “contemplate,” “potential,” or the negative thereof and similar words and expressions.

Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumpti

FAQ

What is the significance of the ARCUS platform in treating adRP for DTIL?

The ARCUS platform shows potential for rejuvenating rod photoreceptors in adRP, particularly beneficial for late-stage patients.

When was the ARCUS gene editing study presented by Precision BioSciences?

The study was presented on May 3, 2021, at the ARVO Annual Meeting.

What is the P23H mutation in relation to DTIL's research?

The P23H mutation in rhodopsin is the most common form of autosomal dominant Retinitis Pigmentosa in North America, affecting 25-30% of cases.

Who presented the ARCUS research at the ARVO meeting for Precision BioSciences?

The research was presented by Archana Jalligampala, Ph.D., from the University of Louisville.

What are the expected outcomes of ARCUS treatment for adRP according to Precision BioSciences?

The expected outcomes include improved rod photoreceptor function and potential visual improvements in patients.