In several in vitro and in vivo preclinical studies, the data demonstrated:
- Editing efficiency in mouse liver of up to approximately 60 percent at the transthyretin (TTR) target site after a single intravenous administration, consistently across different lobes. This resulted in an associated decrease in serum TTR protein levels of up to approximately 80 percent;
- Dose-dependent editing by LNP delivery;
- Undetectable Cas9 mRNA and guide RNA (gRNA) in the liver at 72 hours post administration;
- Repair patterns in mouse liver cells in vivo being best predicted by primary mouse liver cells in vitro.