4A3-SC7
Instructions for Use
Product information
4A3-SC7 is a proprietary, ionizable lipid component central to the SORT LNP platform developed for targeted organ delivery. It features a unique branched-tail structure designed to enhance mRNA encapsulation and endosomal escape. In the study, it served as the primary ionizable lipid in both Liver SORT LNPs and updated Lung SORT LNPs. For liver targeting, it was formulated at 15.04 mol% alongside helper lipids (DOPE: 23.04%, Cholesterol: 38.72%), PEG-lipid (DMG-PEG2000: 3.2%), and the liver-targeting lipid 4A3-Cit (20 mol%). This specific composition (Total lipid:RNA = 20:1 wt/wt) yielded LNPs with ~74 nm size, low PDI (0.17), and high encapsulation efficiency (87%) for large mRNAs like ABE editors (~5000 nt). Its branched-tail architecture was critical for stabilizing nanoparticles encapsulating large RNAs, overcoming a key limitation of previous formulations. 4A3-SC7-based Liver SORT LNPs enabled >40% base editing in hepatocytes in vivo, achieving durable correction of the disease-causing SERPINA1 mutation in PiZ mice and significantly reducing pathological protein aggregates. In the updated DualSORT system, 4A3-SC7 was also paired with DORI (instead of DOTAP) for improved lung targeting, demonstrating its versatility as a foundational ionizable lipid for multi-organ gene editing therapeutics.
| CAS No. | Not available |
| Chemical Name | 4A3-SC7 |
| Formula | C71H131N3O16S4 |
| M.Wt | 1,411.07 |
| Purity | ELSD-HPLC>95% |
| Storage | Original product: up to 1 year under the storage conditions stated on the vial, kept tightly sealed. Stock solutions: aliquots at -20°C, generally up to one month. |
| Shipping condition | Ships from Shanghai. Pure lipid is stable during ice-pack transport. |
Formulation & study context
Literature information refers to the cited study and its particular formulation. Administration routes are research context, not clinical instructions or a guarantee of performance.
A complete product-specific formulation has not been verified. No standard ratio is substituted.
| N/P ratio | Not reported in the verified record |
| Cargo | Not available in the verified record |
| Administration route | Not available in the verified record |
| Animal model | Not available in the verified record |
| Formulation process | Not available in the verified record |
N/P is the molar ratio of specified lipid nitrogen groups to nucleic-acid phosphate groups. A lipid:cargo mass ratio is a different quantity and must not be used as N/P.
Stock-solution preparation table
Calculated from the product-page molecular weight: 1411.07 g/mol. Volumes are final solution volumes.
| Lipid mass | 1 mM | 5 mM | 10 mM |
|---|---|---|---|
| 10 mg | 7.087 mL | 1.417 mL | 0.709 mL |
| 25 mg | 17.717 mL | 3.543 mL | 1.772 mL |
| 50 mg | 35.434 mL | 7.087 mL | 3.543 mL |
| 100 mg | 70.868 mL | 14.174 mL | 7.087 mL |
| 250 mg | 177.171 mL | 35.434 mL | 17.717 mL |
Volume (mL) = mass (mg) × 1000 / [MW (g/mol) × concentration (mM)]. These are mathematical conversions, not measured solubility or validated solvent recommendations. Confirm solvent compatibility and the molecular weight of the exact material before use.
Open editable solution calculators →Handling & storage
Provided that storage conditions are as stated on the product vial and the vial is kept tightly sealed, the product can be stored for up to 1 year.
Wherever possible, prepare and use solutions on the same day. If stock solutions must be prepared in advance, store them as aliquots in tightly sealed vials at -20°C. Generally, these will be usable for up to one month.
Before use, and prior to opening the vial, allow the product to equilibrate to room temperature for at least 1 hour.
References
Dual SORT LNPs for multi-organ base editing-June 2025 Nature Biotechnology-Minjeong Kim1, Daniel J. Siegwart .etl