Commentary
Video
Durability and Next Steps in Advancing ARCUS Gene Editing Platform in DMD: Cassandra Gorsuch, PhD
Author(s):
The chief scientific officer at Precision Biosciences provided clinical insights on the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
"With ARCUS, once the edit happens, it’s permanent—designed to outlast the vector and deliver a long-term, meaningful benefit for patients."
Duchenne muscular dystrophy (DMD) is a severe X-linked disorder caused by mutations in the DMD gene, resulting in absent or non-functional dystrophin and progressive muscle degeneration, typically leading to death in early adulthood. Current therapeutic strategies, including gene therapies that deliver truncated, synthetic dystrophin constructs, have shown only modest clinical benefit and may be limited by durability concerns, particularly in pediatric populations. Several in the field have begun to explore more innovative gene editing approaches, including Precision Biosciences, which has developed an ARCUS-mediated gene editing platform.
This new method aims to restore native dystrophin function by utilizing 2 ARCUS nucleases, delivered using a single adeno-associated virus (AAV), to excise exons 45-55 of the dystrophin gene. In a preclinical study presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific conference, held March 16-19, in Dallas, Texas, results showed that the maximum force output (MFO) was significantly improved after ARCUS treatment, reaching 89% of the MFO levels observed in non-diseased mice. In addition, the data revealed significant resistance to eccentric injury, reaching a 66% improvement compared with disease untreated mice.
Following the data, NeurologyLive® sat down with Cassandra Gorsuch, PhD, chief scientific officer at Precision, to discuss the company’s strategy in bringing this gene editing platform into clinical trials. She emphasized the approach’s durability, noting that once ARCUS edits the dystrophin gene, the change is permanent—even if the AAV delivery vector is later silenced or lost. Furthermore, Gorsuch highlighted the platform’s functional benefits, including is targeted exon 45-55 deletion strategy and potential to correct satellite stem cells for lasting effect.
Click here for more MDA 2025 coverage.
