Unveiling ARCUS Gene Editing Platform and Its Potential in DMD: Cassandra Gorsuch, PhD
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.
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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.
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