MOCHOL is described as positively charged in the acidic RNA-loading phase (pKa 6.5); raising pH after vesicle formation produces the charge-converted final system.
NOV340/MTL-CEBPA process · Paragraphs [000599]-[000604], [000794]sales@dcchemicals.com
MOCHOL GMP-like Quality
Formulation & Performance
Literature-reported formulation composition
| Component | mol% | Role |
|---|---|---|
| POPC | 6 | Helper phospholipid |
| DOPE | 24 | Helper phospholipid |
| CHEMS | 23 | anionic_sterol_lipid |
| MOCHOL | 47 | ionizable_sterol_lipid |
| Reported total | 100 | Source values retained |
Formulation calculator unavailable. The 4 reported lipid components total 100 mol%. Obtain and verify the complete composition and component molecular weights before planning component quantities.
Literature-reported performance for the cited formulation
Literature-reported50-150 nm; PDI <0.200; zeta potential < -30 mV at pH 7.2-7.8MTL-CEBPA release specificationParagraph [000613]
Literature-reported>75%RiboGreen total/free RNA methodParagraphs [000613]-[000615]
Literature-reportedAbout 50-60%CEBPA-51 concentration and pH experimentsParagraphs [000794]-[000805], Figs. 34-35
Literature-reportedImproved body weight, liver-function markers, ammonia, total protein, ascites and survival1 mg/kg MTL-CEBPA started at week 8 or 11Paragraphs [000826]-[000830], Figs. 36-38
MOCHOL formulation and stability findings
| Item | Condition | Reported result |
|---|---|---|
| Composition | NOV340 lipids | POPC/DOPE/CHEMS/MOCHOL = 6/24/23/47 mol% |
| Release profile | pH 7.2-7.8 | >75% RNA encapsulation; 50-150 nm; PDI <0.200; zeta < -30 mV |
| Frozen stability | -20 +/- 5 C | No meaningful change reported through 6 months |
| Thermal stress | 72 h at 25 C / 40 C | MOCHOL decreased 16% / 25% with cholesterol formation |
WO2016/170349 A1 paragraphs [000597], [000613]-[000615], [000820]-[000825].
Additional reported findings
Under 25-40 C stress, MOCHOL content fell while cholesterol increased; the other lipid excipients and total saRNA did not show significant changes in the described test.
Single stability batch · Paragraphs [000822]-[000824]Literature Data DisclaimerAll formulation parameters and performance values shown in this section are derived from the cited publication and are provided for reference only. These results were obtained using the specific materials, formulation process, cargo, dose, analytical method, model and administration route described in that study. Unless otherwise stated, the data were not generated or independently verified by DC Chemicals. DC Chemicals supplies the lipid compound only and does not guarantee that customers will reproduce the reported LNP properties or biological performance.
The administration route shown above was used in the cited study and is not a clinical-use instruction or recommendation by DC Chemicals.
The administration route shown above was used in the cited study and is not a clinical-use instruction or recommendation by DC Chemicals.
Cited literature for this formulation and performance datasetMiNA Therapeutics Ltd. C/EBP alpha saRNA compositions and methods of use. WO2016/170349 A1. Published 27 October 2016. DOI: https://patents.google.com/patent/WO2016170349A1/en
All formulation, stability and animal statements refer to the MOCHOL-containing NOV340/MTL-CEBPA system described in the patent.
All formulation, stability and animal statements refer to the MOCHOL-containing NOV340/MTL-CEBPA system described in the patent.