Formulation & Performance
The primary patent supports a bounded hepatic siRNA-delivery record. Compound 17 is assigned to an ED50 interval rather than an exact potency value; particle and silencing results apply to the complete LNP.
The catalog connectivity was matched to Compound 17 in WO2013086354A1, rather than the same-formula isomer Compound 22. The neutral formula is C49H95NO6.
Formulation components and molecular-weight conversion
| Component | DC Chemicals product | Molecular weight | Molar ratio | Mass at 1 µmol total lipid (mg) |
|---|---|---|---|---|
| L-369 / Compound 17 | DC65701 | 794.28 | 50 | 0.397140 |
| DSPC | DC57005 | 790.15 | 10 | 0.079015 |
| Cholesterol | DCD-041 | 386.65 | 38.5 | 0.148860 |
| PEG-DMG | DC40169 | 2507 | 1.5 | 0.037605 |
Example 35 reports ionizable lipid:DSPC:cholesterol:PEG-DMG = 50:10:38.5:1.5 mol%. No unique Compound 17 N/P or lipid:siRNA mass ratio was identified. PEG-DMG mass conversion uses the catalog convention 2507.
Component mass calculator
Prefilled proportions follow the source specified above. Molecular weights and ratios are editable. Results are calculated, not measured study data. No N/P or RNA dose is inferred.
| Component | MW | Molar ratio | Mass (mg) | Mass % |
|---|---|---|---|---|
| L-369 / Compound 17 | ||||
| DSPC | ||||
| Cholesterol | ||||
| PEG-DMG |
Formulation evidence
| Record | Reported result | Interpretation |
|---|---|---|
| Payload | AD-1661 siRNA targeting Factor VII | Hepatic gene-silencing model |
| Lipid composition | Compound 17:DSPC:cholesterol:PEG-DMG = 50:10:38.5:1.5 mol% | Example 35 |
| Preparation approach | In-line mixing followed by buffer exchange | Reported experimental approach, not validated manufacturing instructions |
| N/P and lipid:siRNA mass ratio | Not established for the specific Compound 17 record | Do not infer from general LNP conventions |
WO2013086354A1, Example 35; printed p202 onward.
Mouse Factor VII silencing
| Record | Reported result | Interpretation |
|---|---|---|
| Model | Female C57BL/6 mice, 7–9 weeks; n=3 per group | Example 36 |
| Administration / timing | Intravenous; Factor VII readout at 24 h | Complete siRNA-LNP formulation |
| Initial screen | 0.1, 0.3, 1.0 and 5.0 mg/kg siRNA; active formulations retested at lower ranges | Screen design, not a dosing recommendation |
| Compound 17 potency | ED50 bin 0.001–0.10 mg/kg, designated ** | An interval, not a precise ED50 |
| Endpoint | 50% reduction in circulating Factor VII relative to control | Gene silencing, not disease-model efficacy |
| Controls | PBS and benchmark formulations | No exact superiority claim inferred |
WO2013086354A1, Example 36. Compound 17 row: printed p207 / PDF p208 in the archived visual review.
Particles and evidence limits
| Record | Reported result | Interpretation |
|---|---|---|
| Diameter | 56 nm before and 56 nm after overnight dialysis | Example 37 exact structure row; no uncertainty supplied |
| Stability | No reported diameter change during dialysis | Not long-term shelf life, serum or freeze–thaw stability |
| Other parameters | Compound-specific PDI, encapsulation, zeta potential and apparent pKa not established | Leave unavailable values blank |
| Tolerability and NHP | No Compound 17-specific NHP or wide-safety-margin dataset identified | Broad patent embodiments are not verified Compound 17 findings |
| Evidence type | Public primary patent | Not clinical validation |
WO2013086354A1, Example 37, printed p209 / PDF p210. Synthesis and chemical analytical measurements are omitted from this public summary.