LP-01
DC31000
Recommended because it shares a closely related research category or delivery context. Formulation and performance are product- and study-specific.
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Literature evidence · DC67546
Formulation & Performance
The 2021 CD4-targeting study supplies direct formulation and biological evidence. Its antibody-conjugated research particles and the later k-abe clinical formulation are separate records.
Tombácz et al. explicitly identify ALC-0307 as the default ionizable lipid. The platform developer’s official 16 May 2025 announcement separately names ALC-0307 and ALC-0159 in the k-abe program. The latter connection is based on a corporate primary source rather than a named-lipid statement in the clinical paper.
Formulation components and molecular-weight conversion
| Component | DC Chemicals product | Molecular weight | Molar ratio | Mass at 1 µmol total lipid (mg) |
|---|---|---|---|---|
| ALC-0307 | DC67546 | 863.5 | 50 | Not calculable from disclosed data |
| Phosphatidylcholine (specific species not resolved) | Not uniquely mapped | Not established | 10 | Not calculable from disclosed data |
| Cholesterol | DCD-041 | 386.65 | 38.5 | Not calculable from disclosed data |
| PEG lipid (specific identity not resolved) | Not uniquely mapped | Not established | 1.5 | Not calculable from disclosed data |
Tombácz 2021 core ratio: 50:10:38.5:1.5 mol/mol. RNA:total lipid approximately 0.04:1 by mass, equivalent to approximately 1:25. CD4-targeted particles also have antibody functionalization; this core ratio is not a complete conjugated-particle recipe. Do not substitute DSPC, ALC-0159 or DMG-PEG2000 for the generic components without confirming identity. These are not verified k-abe clinical ratios.
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 % |
|---|---|---|---|---|
| ALC-0307 | ||||
| Phosphatidylcholine (specific species not resolved) | ||||
| Cholesterol | ||||
| PEG lipid (specific identity not resolved) |
Particle characterization
| Particle | Reported result | Source |
|---|---|---|
| Unconjugated LNP | Approximately 80 nm | Materials and methods: Characterization of LNPs |
| Antibody-conjugated LNP | 88.37 ± 4.2 nm; PDI 0.1 | Materials and methods: Characterization of LNPs |
Values describe the published research formulation, not specifications for free ALC-0307.
Cell-based evidence
| System | Observation | Source |
|---|---|---|
| Human donor CD4+ T cells | Selective binding and dose-responsive luciferase expression relative to isotype-IgG LNP controls | Figure 1 |
| Ai6 mouse splenocytes | Approximately 80% reporter-positive cells in the CD3+CD8− gate with targeted Cre mRNA-LNPs | Figure 2; CD4 staining was transiently reduced after targeting, hence the alternative gate |
Animal delivery and reporter activity
| Readout | Finding | Study boundary |
|---|---|---|
| Cre/loxP reporter recombination | Roughly 60% reporter-positive CD4+ cells in spleen and 40% in lymph nodes | Figure 5, 24-hour mouse endpoint; dose-response groups n=8–9; not CAR-T generation or antitumor response |
| Luciferase in isolated splenic CD3+ cells | 33-fold greater signal with anti-CD4 targeting than isotype-IgG controls | Figure 4D, 5-hour endpoint, n=3 |
| High-dose limitation | The 90 μg RNA dose was toxic in all LNP groups and was excluded from subsequent experiments | Results preceding Figure 5; do not describe the platform as universally nontoxic |
CD4 specificity belongs to the antibody-conjugated formulation, not the free lipid molecule.
Separate k-abe clinical-program evidence
| Evidence | Finding | Source and boundary |
|---|---|---|
| Lipid identity | ALC-0307 and ALC-0159 explicitly named | Acuitas official release, May 16, 2025, PDF p2; corporate primary identity confirmation |
| Initial clinical report | One infant, two administrations and seven weeks of early follow-up; increased dietary protein tolerance and reduced nitrogen-scavenger requirement | Musunuru 2025 NEJM, Summary and Clinical Course |
| Clinical tolerability | Transient aminotransferase elevations; no serious adverse events in the initial short report | Single-patient evidence; not general lipid safety |
| Monkey preclinical assessment | Transient ALT/AST elevations; plasma lipid excipient concentrations >99.5% below peak by two weeks | NEJM Preclinical Validation; plasma decline is not complete whole-body clearance |
The clinical paper does not name ALC-0307; the explicit platform-developer announcement supplies that identity connection. The release mentions three administrations, whereas the initial paper covers two. Keep their reporting cutoffs distinct.
Original study figures
CD4-targeted biological results, Figures 1–7 — Original source figures.
References and source locations
- Tombácz I, et al. Highly efficient CD4+ T cell targeting and genetic recombination using engineered CD4+ cell-homing mRNA-LNPs. Molecular Therapy 29 (2021), 3293–3304.
- Acuitas Therapeutics. Official May 16, 2025 ASGCT announcement, p2 (lipid identity confirmation).
- Musunuru K, et al. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. NEJM 392 (2025), 2235–2243.
| CAS No. | Not available |
| Chemical Name | ALC-0307 |
| Synonyms | ALC0307， ALC 0307 |
| SMILES | CCCCCCCCC(CCCCCCCC)OC(=O)CCCCCCCN(CCCCCCCC(=O)OC(CCCCCCCC)CCCCCCCC)CCCN(C)C |
| Formula | C55H110O4N2 |
| M.Wt | 863.5 |
| 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. |
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