Achieving Long-Duration Hepatic Expression with Optimized Self-Amplifying RNA to Transform the Future of Protein Replacement Therapies
- Deliver 30-day liver expression after IV administration for the first time by integrating RNA engineering with advanced formulation strategies to overcome historic expression failure in hepatic tissues
- Unlock therapeutic feasibility for saRNA-based protein replacement by matching the durability of viral systems without relying on genome integration or high-dose repeat administration
- Reveal mechanistic and translational insights from optimizing saRNA constructs to guide the next wave of durable, redose-ready therapeutic applications beyond vaccines