Literature-reported formulation composition
Component: ssPalmO-Phe | Reported molar ratio: 70 | Role: Ionizable lipid
Search the tables and observations reproduced from the product literature guide. Rows may represent measurements, recipes or experimental context; they are not all independent experiments.
Component: ssPalmO-Phe | Reported molar ratio: 70 | Role: Ionizable lipid
Component: Cholesterol | Reported molar ratio: 30 | Role: Sterol
Component: DMG-PEG2000 | Reported molar ratio: 1.5 | Role: PEG lipid
Component: Reported ratio sum | Reported molar ratio: 101.5 | Role: Original ratio retained
Cargo: siFVII | Reported lipid ratio: ssPalmO-Phe/Cholesterol/DMG-PEG2000 = 70/30/+1.5 | Lipid:cargo basis: 400 nmol lipid per microgram siRNA | Key result: ED50 4.4 microgram/kg; 98.7 ± 1.3% knockdown at 40 microgram/kg
Cargo: ASO | Reported lipid ratio: ssPalmO-Phe/DOPC/Cholesterol/DMG-PEG2000 = 40/20/40/+1.5 | Lipid:cargo basis: 25 nmol lipid per microgram ASO | Key result: IC50 0.0097 mg/kg ASOap; 0.092 mg/kg ASOgr
Literature-reported Particle diameter 112 nm 1.5 mol% additional DMG-PEG2000 formulation Main Table 1
Literature-reported PDI 0.135 Same formulation Main Table 1
Literature-reported siRNA encapsulation 100% Same formulation Main Table 1
Literature-reported Factor VII knockdown 98.7 ± 1.3% 40 microgram/kg siFVII · 24 h Main Results 3.3; Fig. 4
Literature-reported Factor VII ED50 4.4 microgram/kg Mouse dose-response fit · 24 h Main Results 3.3; Fig. 4
ASO-dependent hepatotoxicity Severe hepatotoxicity was reported for LNP-ASOap at 0.37 mg/kg, while LNP-ASOgr did not show the same response at that dose. The authors attributed the difference to ASO sequence chemistry rather than empty-carrier toxicity. Cargo-specific mouse study · Main Results 3.6; Fig. 6
Empty-LNP tolerability range Empty LNP doses of 49-782 mg/kg total lipid (29-470 mg/kg ssPalmO-Phe) did not significantly alter AST or ALT in the reported 96 h experiment. This does not establish a general safe dose · Main Results 3.6; Fig. 6