Redefining Non-Hepatic RNA Delivery to Unlock Safer Self-Amplifying RNA at Clinically Relevant Doses
- Demonstrating how extra-hepatic delivery overcomes dose-limiting toxicities, enabling unprecedented clinical dose escalation of self-amplifying RNA without Grade 3 adverse events
- Connect RNA design, formulation, and process decisions to myocarditis risk, using integrated clinical, preclinical, and mechanistic data to explain why safety failures occur
- Translate mechanistic insight into actionable delivery strategies, showing how non-hepatic targeting mitigates innate immune activation and supports repeat dosing in next-generation RNA therapeutics