Formulation & Performance
The study compares liver delivery, repeat protein expression in rats and nonhuman primates, and intramuscular reporter delivery.
The product is catalogued as Lipid 10, the lipid identifier used in Lam et al. The study’s siRNA and mRNA formulations differ, and their results are presented separately.
Formulation components and molecular-weight conversion
| Component | DC Chemicals product | Molecular weight | Molar ratio | Mass at 1 µmol total lipid (mg) |
|---|---|---|---|---|
| Lipid 10 / Genevant CL1 | DC59126 | 588.0 | 54.6 | Not calculable from disclosed data |
| DSPC | DC57005 | 790.15 | 10.9 | Not calculable from disclosed data |
| Cholesterol | DCD-041 | 386.65 | 32.8 | Not calculable from disclosed data |
| PEG-conjugated lipid (anchor unspecified) | Not uniquely mapped | Not established | 1.6 | Not calculable from disclosed data |
The component table describes the main mRNA ratio. siRNA uses a different ratio. PEG anchor identity and N/P are not specified in the reviewed main text; the ratios total 99.9 or 100.1 because of source rounding.
Component mass calculator
The source does not disclose all inputs; missing fields are intentionally blank. Enter your own values to calculate a research scenario. 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 % |
|---|---|---|---|---|
| Lipid 10 / Genevant CL1 | ||||
| DSPC | ||||
| Cholesterol | ||||
| PEG-conjugated lipid (anchor unspecified) |
Formulation and particle properties
| Cargo | Lipid 10 : DSPC : cholesterol : PEG-lipid | Total lipid:RNA (w/w) | Diameter (nm) | PDI | EE (%) | Apparent pKa |
|---|---|---|---|---|---|---|
| TTR siRNA | 55 : 11 : 33 : 1.1 | 9 : 1 | 74 | 0.02 | 97 | 6.3 |
| EGFP mRNA | 54.6 : 10.9 : 32.8 : 1.6 | 20 : 1 | 76 | 0.14 | 99 | 6.3 |
Main Table 1 and LNP Production. Reported molar ratios are not silently renormalized.
Biological performance
| Cargo / study | Model and context | Result | Source |
|---|---|---|---|
| TTR siRNA | BALB/c mice; IV 0.01 mg/kg; 48 h; n=4 | Liver TTR knockdown 92%; KC2 62%; MC3 74%. | Fig.1A |
| EGFP mRNA | BALB/c mice; IV 1 mg/kg; 24 h; n=4 | Higher liver expression than KC2 and MC3. | Fig.1B |
| EGFP dose comparison | BALB/c mice; IV 0.3, 1 or 3 mg/kg; n=4 | Liver/spleen expression and ALT/AST at 24 h; MCP-1 at 6 h. | Fig.2 |
| hOTC repeat dosing | Sprague–Dawley rats; IV 0.25 mg/kg weekly ×8; n=3 | Hepatic expression maintained; no notable toxicity in the reported monitoring window. | Fig.3B–E; section2.4 |
| hEPO repeat dosing | Cynomolgus monkeys; IV 0.25 mg/kg weekly ×5; n=3 | Plasma hEPO expression maintained. | Fig.3F |
| Luciferase mRNA | BALB/c mice; IM 5 µg; 6 h | Injection-site expression comparable to SM-102 and ALC-0315. | Fig.4 |
Tolerability and interpretation
| Scope | Result / limit |
|---|---|
| Eight weekly rat doses | No reported weight loss/adverse reactions or notable (>3× PBS) ALT, AST or cytokine increases. |
| Five weekly NHP doses | Supports repeat expression under the tested regimen; it does not establish clinical safety or indefinite repeat dosing. |
SI-only numerical fLuc particle properties and detailed toxicology tables were not reverified and are excluded.