" class="no-js "lang="en-US"> Precision BioSciences Announces Gene Editing Research for DMD
Friday, June 21, 2024

Precision BioSciences Announces Late-Breaking Abstract of Preclinical In Vivo Gene Editing Research for DMD

Precision BioSciences, 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 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.

“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.”


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