On April 18, 2023 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, reported 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) (Free ASGCT Whitepaper) 26th Annual Meeting being held May 16-20, 2023 in Los Angeles, CA (Press release, Precision Biosciences, APR 18, 2023, View Source [SID1234630256]).
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"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 (Free ASGCT Whitepaper) presentation highlighting new data from Precision’s DMD gene editing program."
The abstract will be publicly accessible through the ASGCT (Free ASGCT Whitepaper) website at View Source beginning on May 2, 2023.
Presentation Details:
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
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), 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.