“At 26 weeks following IV administration of 5x1012 to 9x1013 gc/kg HEMGENIX in NHPs, high levels of vector DNA were detected in a dose-dependent manner in the liver.”
✓ VerifiedFDA summary basis for approval, p. 11Source ↗
Pivotal GLP studies
GLP single-dose IV studies in C57Bl/6 mice and cynomolgus monkeys (13-26 weeks observation); mouse paternal germline transmission study
“The single IV dose toxicology studies and the analysis of the safety parameters were performed under GLP compliance.”