Target | Dose (mg/kg) | Species | Measurement(s) | Reference(s) |
---|---|---|---|---|
AT | 1–5 (s.c., SD) | Mice | Plasma, liver, and RISC PK, AT mRNA, plasma AT | Sehgal et al., 2015; Nair et al., 2017 |
AT | 25 (i.v. and s.c., SD) | Mice | Plasma PK, liver PK (s.c.) | Akinc et al., 2012 |
AT | 0.75–3 (s.c., MD) | Mice | Plasma AT | Sehgal et al., 2015 |
TTR | 10 (i.v. and s.c., SD) | Mice | Plasma, kidney PK | Nair et al., 2017 |
ALAS1 | 1–10 (s.c., SD) | Rat | Liver, RISC PK | Lee et al., 2019 |
AT | 1–30 (s.c., SD) | NHP | Plasma AT | Sehgal et al., 2015 |
AT | 0.015–1.8 (s.c., SD, MD) | Human | Plasma PK, plasma AT | Pasi et al., 2017 |
ALAS1, delta-aminolevulinate synthase 1; MD, multiple dosing; NHP, nonhuman primate; PK, pharmacokinetics; SD, single dose.