Advancing ADAR Mediated RNA Editing Therapies: An Optimal Platform for Treatment of Rare & Common Genetic Diseases
- Platform Innovation: RESTORE+ platform leverages proprietary GalNAc delivery and enhanced chemistry to maximize RNA editing potency, durability, and safety
- Targeted Liver Delivery: Engineered for precise ADAR-mediated editing with minimized safety risks, ensuring effective hepatic targeting
- Lead Program in AATD: Focused on addressing the genetic root of Alpha-1 Antitrypsin Deficiency to offer a transformative therapeutic option