“In 2025, our steadfast focus remains on continued clinical execution as we work to deliver on the promise of our ARCUS gene editing technology through robust clinical data,” continued Mr. Amoroso. “The recent reported clinical experience for PBGENE-HBV focused on viral editing and elimination of replicating cccDNA and integrated HBV DNA in Hepatitis B, an extremely large patient population, adds to the compelling clinical validation of the ARCUS platform observed in severe OTC deficiency, a rare and extremely dire disease. In January, our partner iECURE announced news from the OTC-HOPE study, where first-in-human data demonstrated a complete clinical response in severe neonatal OTC deficiency using an ARCUS nuclease for in vivo gene insertion. We look forward to building on this clinical momentum throughout 2025.”
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