Table 2
Component: Lipidoid | Molecular weight: Not confirmed | Reported molar entry: Not recovered | Calculated mass: Not calculated
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: Lipidoid | Molecular weight: Not confirmed | Reported molar entry: Not recovered | Calculated mass: Not calculated
Component: DSPC | Molecular weight: 790.15 | Reported molar entry: Not recovered | Calculated mass: Not calculated
Component: Cholesterol | Molecular weight: 386.65 | Reported molar entry: Not recovered | Calculated mass: Not calculated
Component: PEG lipid | Molecular weight: Not confirmed | Reported molar entry: Not recovered | Calculated mass: Not calculated
Parameter: Organic phase | Reported procedure / limitation: C12-200, DSPC, cholesterol and mPEG2000-DMG were dissolved in 90% ethanol. | Source: Main Methods, Formulation of LNPs; retained prior extraction
Parameter: Aqueous phase | Reported procedure / limitation: siRNA in 10 mM citrate buffer at pH 3. | Source: Main Methods, Formulation of LNPs; retained prior extraction
Parameter: RNA loading | Reported procedure / limitation: Total lipid:siRNA 7:1 by mass. This refers to all lipid components together, not C12-200 alone. N/P was not reported in the retained formulation record. | Source: Main Methods, Formulation of LNPs; retained prior extraction
Parameter: Mixing | Reported procedure / limitation: Lipid and siRNA streams were pumped at equivalent volumetric flow rates through a T-junction. Absolute flow rate was not recovered. | Source: Main Methods, Formulation of LNPs; retained prior extraction
Parameter: Buffer exchange | Reported procedure / limitation: Dialysis against PBS containing 155 mM NaCl, 3 mM Na2HPO4 and 1 mM KH2PO4, pH 7.5. | Source: Main Methods, Formulation of LNPs; retained prior extraction
Parameter: Experiment separation | Reported procedure / limitation: Cell-screening lipidoid/siRNA complexes used distinct mass ratios; those values are not the four-component in vivo LNP composition. | Source: Main Results, Figs.2–3
Measurement / batch: Particle diameter | Reported result: Approximately 80 nm | Evidence context: Main Methods; retained prior extraction; LNP value, not neat-lipid specification
Measurement / batch: siRNA entrapment | Reported result: Approximately 90%, assessed by RiboGreen | Evidence context: Main Methods; retained prior extraction
Measurement / batch: N/P, PDI and apparent pKa | Reported result: No experiment-specific numerical values recovered in this review | Evidence context: Do not infer N/P from the total-lipid mass ratio or borrow values from later C12-200 studies
Experiment and dose: Initial in vivo lipidoid screen C57BL/6 mice; single intravenous tail-vein administration; anti-FVII siRNA 3 mg/kg; 12 purified lipidoids; n=3 or 4 | Readout and comparator: Readout: 72 h Comparator: Other library members and serum FVII baseline | Result and source: The lead C12-200 emerged from a fixed-weight-composition screen. This initial screen is distinct from the optimized low-dose C12-200 experiments. Main Results, initial in vivo screen and Fig.3
Experiment and dose: Low-dose Factor VII response C57BL/6 mice; intravenous anti-FVII siRNA in C12-200 LNP | Readout and comparator: Readout: Assay timing is experiment-specific; 48/72 h is not pooled into one time point Comparator: LNP01 reference; nontargeting siRNA control at 1 mg/kg | Result and source: Activity reported below 0.01 mg/kg siRNA; more than two orders of magnitude greater dose potency than the earlier LNP01 system. No exact ED50 is inferred from the abstract claim. Main Abstract and Results, Fig.3
Experiment and dose: Duration of Factor VII suppression C57BL/6 mice; single intravenous administration of 0.1 or 1 mg/kg anti-FVII siRNA; n=3 | Readout and comparator: Readout: 24 h to approximately 35 days Comparator: Predose/baseline serum Factor VII | Result and source: Complete silencing at 24 h; Factor VII protein returned to baseline by approximately 20 days at 0.1 mg/kg and 35 days at 1 mg/kg. Main Results, Fig.4
Experiment and dose: Five-target liver silencing Mouse liver; FVII, ApoB, PCSK9, XBP1 and SORT1 siRNAs; single intravenous administration; 0.2 mg/kg per siRNA, totaling 1 mg/kg; n=5 | Readout and comparator: Readout: 72 h Comparator: Saline and control-siRNA groups | Result and source: More than 65% silencing for each of the five target mRNAs. The reported 0.2 mg/kg is per target and must not be presented as the total mixture dose. Main Results, Fig.5
Experiment and dose: Cellular uptake mechanism HeLa cells; fluorescent siRNA/C12-200; dextran, transferrin and cholera toxin B trafficking markers; EIPA and cytochalasin D inhibition experiments | Readout and comparator: Readout: Membrane actin ruffling observed after 15 min; uptake-assay timings depend on experiment Comparator: Marker colocalization and inhibitor controls | Result and source: Dextran colocalization, actin changes and inhibitor-sensitive uptake support a macropinocytosis-associated route in this cell model. They do not establish an exclusive uptake route in vivo. Main Results, Fig.6
Experiment and dose: Hepatic TTR silencing in nonhuman primates Cynomolgus monkeys; anti-TTR siRNA 0.03, 0.1 or 0.3 mg/kg; 15-minute cephalic-vein infusion; n=3 per dose | Readout and comparator: Readout: 48 h liver biopsy Comparator: PBS and irrelevant-siRNA controls | Result and source: Dose-dependent and sequence-specific hepatic TTR mRNA knockdown. Exact percentage values are not transcribed from the inaccessible figure in this pass. Main Results, nonhuman-primate TTR experiment; NHP methods retained from prior extraction
Experiment and dose: Reported mouse tolerability observations C12-200 siRNA LNP at 1 mg/kg in the reported mouse experiments | Readout and comparator: Readout: Study-specific observations Comparator: PBS/control measurements | Result and source: No reported indication of toxicity or body-weight change; liver chemistry results were assessed in the paper. This limited observation does not establish long-term or clinical safety. Main Results, Table1 and Fig.4 discussion