Efficacy ResultsEfficacy results across key biomarkers are encouraging, showing statistically significant evidence of changes from baseline in glutamine and ureagenesis.
Safety ProfileARCT-810 was generally safe and well tolerated with the optimized infusion regimen, a clear positive, particularly given the lack of steroid pre-treatment.
Strategic PositioningArcturus' highly purified mRNA gets into the liver cells of patients and generates functional enzymes, which is seen as derisking for the entirety of Arcturus' liver-focused pipeline.