Commentary
Video
Unveiling ARCUS Gene Editing Platform and Its Potential in DMD: Cassandra Gorsuch, PhD
Author(s):
The chief scientific officer at Precision Biosciences provided clinical commentary on ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy. [WATCH TIME: 4 minutes]
"We designed this approach with durability in mind—once the ARCUS edit happens, it’s permanent. That’s the real advantage over traditional vector-based delivery."
The understanding of genetics in Duchenne muscular dystrophy (DMD) has evolved significantly over the past few decades, shifting the paradigm from symptom management to targeted, disease-modifying strategies. Advances in molecular biology have enabled precise identification of mutation types–such as deletions, duplications, and point mutations–paving the way for personalized genetic interventions. There is already 1 approved gene therapy for DMD; however, there are questions behind its long-term applicability, leaving the door open for other gene editing approaches.
The 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific meeting, held March 16-19, in Dallas, Texas, brought together the latest in genetic-based approaches for neuromuscular conditions like DMD. One poster presentation focused on preclinical research testing Precision Biosciences’ ARCUS gene editing platform, an innovative approach that uses a single adeno-associated vector to excise exons 45-55 of the dystrophin gene. Following AAV delivery, the preclinical data showed del45-55 dystrophin in multiple tissue types including heart and skeletal muscle, with evidence of the del45-55 dystrophin transcript in PAX7+ cells, a marker for muscle satellite stem cells.
During the meeting, Cassandra Gorsuch, PhD, chief scientific officer at Precision, sat down to discuss the mechanism behind the gene editing platform, highlighting how its unique design could offer advantages over traditional CRISPR approaches. In targeting DMD, ARCUS aims to create a permanent, DNA-level edit in the dystrophin gene. In the conversation, Gorsuch discussed the long-term plans for the innovative approach, emphasizing its potential for long-term efficacy, independent of AAV persistence, and next steps for a first-in-human trial.
