- Presentation to Highlight Preclinical Development Progress of PBGENE-DMD Program for Potential Treatment of Duchenne Muscular Dystrophy
Precision 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 that a late-breaking abstract featuring preclinical data from its PBGENE-DMD program for the potential treatment of Duchenne muscular dystrophy (DMD), was selected for an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting being held May 16-20, 2023 in Los Angeles, CA.
“ARCUS nucleases have been shown to be exceptionally versatile for complex gene editing approaches, including large gene excisions. The goal of the PBGENE-DMD program is to utilize a pair of ARCUS nucleases, delivered by a single AAV, that are designed to excise an approximately 500,000 base pair mutation “hot spot” region from the dystrophin gene in order to make a variant of the dystrophin protein that is functionally competent,” said Jeff Smith, Chief Research Officer of Precision BioSciences. “We look forward to the upcoming ASGCT presentation highlighting new data from Precision’s DMD gene editing program.”
The abstract will be publicly accessible through the ASGCT website at https://annualmeeting.asgct.org/abstracts beginning on May 12, 2023.
Title: ARCUS-Mediated Excision of the “Hot Spot” Region of the Human Dystrophin Gene for the Treatment of Duchenne Muscular Dystrophy (DMD)
Date and Time: Friday, May 19, 2023, 10:15 AM - 12:00 PM
Session Title: Late-breaking Abstracts 2
Session Room: Concourse Hall 152 & 153
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), excise (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 and stimulate gene insertion at the cut site by homologous recombination. Precision's platform and products are protected by a comprehensive portfolio including nearly 100 patents to date.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly precise 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 ex vivo “off-the-shelf” CAR T immunotherapy clinical candidates and several in vivo gene editing candidates designed to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com.
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 expected conference participation and disclosure of preclinical data, and the clinical development and goals of our PBGENE-DMD program. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “target,” “will,” “would,” or the negative thereof and similar words and expressions.
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