Iso-A11B5C1 and SM-102 produced comparable editing at the injected quadriceps. SM-102 produced 83.83% liver and 72.67% spleen editing, whereas editing in those tissues was undetectable with iso-A11B5C1 in the reported experiment.
Cre mRNA; mTmG reporter mice; 0.5 mg/kg IM; 36 h · Main Figure 3C-E and text, PNAS p. 5sales@dcchemicals.com
Iso-A11B5C1
Formulation & Performance
Literature-reported formulation composition
| Component | mol% | Role |
|---|---|---|
| Iso-A11B5C1 | 60 | Ionizable lipid |
| DOPE | 10 | Helper phospholipid |
| Cholesterol | 29 | Sterol |
| C14-PEG2000 (14:0 PEG2000 PE) | 1 | PEG lipid |
| Reported total | 100 | Source values retained |
Component molecular weights and calculated amounts
Composition retained from the cited study below. Research planning aid only. Results cover lipid component weights and do not include RNA, buffer, solvent, process loss or an N/P ratio.
| Component | Molecular weight | Molar parts | Normalized mol% | Amount (µmol) | Weight (mg) |
|---|---|---|---|---|---|
| Iso-A11B5C1 | 60.000 | 98.2439 | 65.0473 | ||
| DOPE | 10.000 | 16.3740 | 12.1827 | ||
| Cholesterol | 29.000 | 47.4846 | 18.3599 | ||
| C14-PEG2000 (14:0 PEG2000 PE) | 1.000 | 1.6374 | 4.4100 |
Calculated total: 100.0000 mg lipid.
Calculated catalog planning quantities; RNA, solvent and process loss are excluded. DMG-PEG2000 uses 2507. Component mass = total lipid mass × molar parts × molecular weight / sum(parts × molecular weight). This conversion does not infer N/P.
Literature-reported performance for the cited formulation
Literature-reported7.21Post-formulation characterization · SM-102 LNP approximately 6.7Main Figure 3B and text, PNAS p. 5
Literature-reportedComparable to the SM-102 benchmark at the injection site6 h after 0.3 mg/kg IM mLuc · No statistically significant difference versus SM-102Main Figure 2D,E and text, PNAS p. 4
Literature-reportedNo measurable expression in examined off-target organs6 h after 0.3 mg/kg IM mLuc · SM-102 produced high liver and spleen expressionMain Figure 2D,E and text, PNAS p. 4
Literature-reportedCy5-mRNA signal localized primarily at the injection site with minimal liver or spleen transfer6 h after 0.3 mg/kg IM Cy5-mLuc · SM-102 showed broader injection-site, liver and spleen distributionMain Figure 2F,G and text, PNAS p. 4
Additional reported findings
Relative to SM-102 LNP, iso-A11B5C1 LNP showed 42-fold higher transfection in HeLa cells but 28-fold and 24-fold lower transfection in HepG2 cells and splenocytes, respectively.
mLuc LNP; HeLa, HepG2 and primary splenocyte comparison · Main Figure 3A and text, PNAS p. 4Iso-A11B5C1 LNP carrying full-length Trp2 mRNA significantly slowed B16-F10-Luc tumor growth and outperformed the MC3 comparator; it also increased IFN-gamma-positive CD8 T cells and intratumoral CD8 T-cell infiltration versus MC3. The authors reported lower efficacy than the SM-102 group.
C57BL/6 melanoma model; two LNP/mTrp2 doses on days 7 and 12 after inoculation; dose not reported · Main Figure 5 and text, PNAS p. 7Four hours after IM dosing, the measured GM-CSF, IFN-gamma, IL-1alpha, IL-5, IL-6 and TNF-alpha levels did not differ discernibly between iso-A11B5C1-treated and PBS-treated mice.
BALB/c mice; 0.5 mg/kg IM; acute cytokine assessment at 4 h · Supplementary Figure S8 and main text, PNAS p. 5Literature 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 datasetChen J, Xu Y, Zhou M, et al. Combinatorial design of ionizable lipid nanoparticles for muscle-selective mRNA delivery with minimized off-target effects. Proceedings of the National Academy of Sciences of the United States of America. 2023;120(50):e2309472120. DOI: 10.1073/pnas.2309472120
Structure and synthesis: main Figure 1F and Supporting Information pp. 19-20; DOE composition: main Figure 2 and text p. 4 plus Supplementary Table S2; pKa and muscle-selective delivery: main Figures 2-3; vaccine and melanoma studies: main Figures 4-5.
Structure and synthesis: main Figure 1F and Supporting Information pp. 19-20; DOE composition: main Figure 2 and text p. 4 plus Supplementary Table S2; pKa and muscle-selective delivery: main Figures 2-3; vaccine and melanoma studies: main Figures 4-5.