LP-01
DC31000
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Literature evidence · DC68021
Formulation & Performance
The selected downstream formulation is distinguished from the initial screen and the higher-mean but more variable luciferase formulation.
Reported performance refers to the complete research formulation and the stated experimental model.
Formulation components and molecular-weight conversion
| Component | DC Chemicals product | Molecular weight | Molar ratio | Mass at 1 µmol total lipid (mg) |
|---|---|---|---|---|
| BIP-20 | DC68021 | 729.2128 | 50 | 0.364606 |
| DOPE | DC33634 | 744.03 | 10 | 0.074403 |
| Cholesterol | DCD-041 | 386.65 | 38.5 | 0.148860 |
| DMPE-PEG2000 | Not uniquely mapped | Not established | 1.5 | Not calculable from disclosed data |
Selected downstream BiP-20 formulation: BIP-20/DOPE/cholesterol/DMPE-PEG2000 = 50:10:38.5:1.5 mol%. DMPE-PEG2000 is a PEG phospholipid, not DMG-PEG2000. Its commercial mapping and MW remain unassigned; N/P is not inferred. Source: formulation-optimization Results and Supplementary Fig. 5.
Component mass calculator
Not all inputs are assigned to a single verified study formulation; unassigned fields are 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 % |
|---|---|---|---|---|
| BIP-20 | ||||
| DOPE | ||||
| Cholesterol | ||||
| DMPE-PEG2000 |
Initial screening formulation
| Component | DC Chemicals product | Molecular weight | Molar ratio | Mass at 1 µmol total lipid (mg) |
|---|---|---|---|---|
| BIP-20 | DC68021 | 729.2128 | 35 | 0.255224 |
| DOPE | DC33634 | 744.03 | 16 | 0.119045 |
| Cholesterol | DCD-041 | 386.65 | 46.5 | 0.179792 |
| DMPE-PEG2000 | Not uniquely mapped | Not established | 2.5 | Not calculable from disclosed data |
Initial screen: 35:16:46.5:2.5 mol%; ionizable lipid:mRNA 10:1 by weight in this screening context. Do not transfer that mass ratio to every later editing formulation.
Component mass calculator
Not all inputs are assigned to a single verified study formulation; unassigned fields are 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 % |
|---|---|---|---|---|
| BIP-20 | ||||
| DOPE | ||||
| Cholesterol | ||||
| DMPE-PEG2000 |
BiP-20-19: higher mean reporter signal, greater variability
| Component | DC Chemicals product | Molecular weight | Molar ratio | Mass at 1 µmol total lipid (mg) |
|---|---|---|---|---|
| BIP-20 | DC68021 | 729.2128 | 45 | 0.328146 |
| DOPE | DC33634 | 744.03 | 16 | 0.119045 |
| Cholesterol | DCD-041 | 386.65 | 38.5 | 0.148860 |
| DMPE-PEG2000 | Not uniquely mapped | Not established | 0.5 | Not calculable from disclosed data |
45:16:38.5:0.5 mol%. This reporter-screen formulation was not selected for the downstream studies despite its higher mean expression.
Component mass calculator
Not all inputs are assigned to a single verified study formulation; unassigned fields are 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 % |
|---|---|---|---|---|
| BIP-20 | ||||
| DOPE | ||||
| Cholesterol | ||||
| DMPE-PEG2000 |
Formulation selection and characterization
| Experiment / parameter | Study conditions | Result / interpretation |
|---|---|---|
| Apparent pKa | TNS assay; main Fig. 1e | 6.56 for the characterized research LNP; not a new isolated-lipid specification. |
| Helper-lipid comparison | Supplementary Fig. 4 | DOPE gave stronger protein expression than DSPC in the tested formulation. |
| Selection rationale | Supplementary Fig. 5 | BiP-20-19 had the highest mean luciferase signal, 1.72-fold above the common ratio, but greater variability; the 50:10:38.5:1.5 formulation was selected for further studies. |
| Final particle metrics | RNA cargo and formulation dependent | Exact size, PDI and encapsulation values for the selected editing formulation are not assigned from screening graphs. |
Reporter expression and uptake dependence
| Experiment / parameter | Study conditions | Result / interpretation |
|---|---|---|
| Luciferase comparison | CD1 mice; 3 µg mRNA IV; Fig. 1f | 5.9-fold higher expression than MC3 and similar expression to SM-102 in the reported comparison. |
| Receptor dependence | ApoE- and LDLR-knockout mice; Supplementary Fig. 6 | Signal was nearly abolished without ApoE and reduced without LDLR, supporting an ApoE-associated liver-delivery mechanism. |
TTR editing and protein reduction
| Experiment / parameter | Study conditions | Result / interpretation |
|---|---|---|
| Liver genomic editing | Cas9/guide RNA; mouse IV dose response; day 7; n = 4; Fig. 1h | Approximately 20% editing at 2 µg total RNA and 64% at 10 µg; LP01 required approximately 30 µg for comparable editing. |
| Circulating TTR | Same dose-response study; Fig. 1i | The 10 µg BIP-20 group showed near-complete serum TTR reduction. Protein reduction is distinct from genomic editing percentage. |
| Separate low-dose experiment | 1 µg RNA; Supplementary Fig. 7 | Approximately 50% serum TTR reduction (P = 0.0093); indels reached up to 40%. “Up to” is not a group mean. |
Prime editing and endolysosomal escape
| Experiment / parameter | Study conditions | Result / interpretation |
|---|---|---|
| PCSK9 prime editing | Mouse liver, day 7; n = 4; Fig. 1j | BIP-20 4.30%, SM-102 3.33%, MC3 1.33%, LP01 0.1225%. Significance was reported versus MC3 and LP01, not versus SM-102. |
| Dose terminology | Prime-editing experiment | The paper uses both total RNA and total mRNA wording for the 50 µg dose. Component amounts are not reconstructed from that ambiguous wording. |
| LysoBC assay | In vivo DNA-barcode compartment analysis | Approximately 8% at 30 min and 6% at 1 h were estimated outside the isolated endolysosomal compartment. This is a barcode-based proxy, not the fraction of cells transfected or mRNA productively translated. |
Acute tolerability
| Experiment / parameter | Study conditions | Result / interpretation |
|---|---|---|
| Mouse systemic evaluation | 50 µg RNA/mouse IV; 24 h; n = 4 | ALT/AST were comparable with untreated controls; IL-6 was not significantly elevated and TNF was below detection in the reported assessment. |
| Interpretation | Single-dose short observation window | These findings do not establish chronic, repeat-dose or human safety. |
Original study figures
Liver expression, editing and endolysosomal compartment measurements — Original source figures.
References and source locations
| CAS No. | Not available |
| Chemical Name | BIP-20 |
| Synonyms | BIP20;BIP 20 |
| SMILES | CCCCCCCCCCC(CCCCCCCC)COP(NCCN(C)C)(OCC(CCCCCCCC)CCCCCCCCCC)=O |
| Formula | C44H93N2O3P |
| M.Wt | 729.2128 |
| Purity | >95% by ELSD-HPLC |
| Storage | Pure form: −20 °C, 1 year; In solvent: −80 °C, 6 months; −20 °C, 1 month. Sealed and protected from light. |
| Shipping condition | Ships from Shanghai. Pure lipid is stable during ice-pack transport. |
| Publication |