Chemical structureResearch use only

Structure redrawn from WO2024131874A1, Table 01-1 (Compound 01-1). Stereochemistry unspecified.

CAT. NO. DCL0009Featured

Lipid 01-1

RNA delivery · cationic/ionizable lipids

Research highlights

mRNA-encoded CD19/CD3 bispecific antibody deliveryIntravenous antibody expression in miceAntitumor study in a human PBMC / Nalm6 leukemia model

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Description & Application

Lipid 01-1 is an ionizable lipid investigated for lipid nanoparticle delivery of mRNA encoding a CD19/CD3 bispecific T-cell engager developed by Abogen. In WO2024131874A1, 01-1-based LNPs supported serum antibody expression and were used in a Nalm6 leukemia model with human PBMCs. A single intravenous dose of mRNA1-LNPs reduced tumor bioluminescence relative to controls and the recombinant antibody regimen tested.

Research category: Ionizable lipids for in vivo T-cell engager

In vivo T-cell engager research

01-1 LNPs were used for CD19/CD3 T-cell engager expression and antitumor experiments in a human PBMC-engrafted Nalm6 leukemia model. Route: Intravenous.

WO2024131874A1, Example 7, paragraphs [0362–0363], Figures 3B and 4A–4B.

Chemical Name	Lipid 01-1
Catalog No.	DCL0009
Patent compound ID	01-1 (WO2024131874A1, Table 01-1)
Formula	C52H102N2O5
M.Wt	835.40
SMILES	CCCCCCCCC(CCCCCC)COC(=O)CCCCCN(CCCCCC(=O)OCC(CCCCCC)CCCCCCCC)CCN(CCO)C1CCC1
Stereochemistry	Not specified; two unassigned branched-tail stereocenters
Salt / solvate	Neutral parent depicted; no counterion or solvate included
CAS No.	Not confirmed
Purity	Inquire for batch-specific information
Availability	Inquire for availability
Classification	Cationic/Ionizable Lipids

SMILES, molecular formula and average molecular weight are calculated from the explicitly labeled neutral structure in the patent. They are not quoted analytical measurements.

Serum antibody expression

Cargo	mRNA1 encoding a CD19/CD3 bispecific antibody
Administration	Intravenous (tail vein)
N/P ratio	Not reported for the cited experiment
Exact formulation	01-1 / DSPC / cholesterol / PEG lipid. Exact molar percentages and PEG-lipid identity are not resolved for this experiment.
Model	NOD/SCID mice
Dose	1 μg mRNA per mouse, single dose
Timepoints shown	24 and 48 hours
Comparators	03-135, 03-208, 02-1 and 02-3

Figure 3B shows serum antibody expression following delivery with 01-1. Among the five formulations plotted, 03-135 has the highest mean expression at 24 and 48 hours; this comparison does not establish 01-1 as the expression leader.

Figure 3B: serum antibody expression using five lipid formulations

WO2024131874A1, Example 7, paragraph [0362], Figure 3B (PDF p. 137).

Literature results apply to the cited formulation and study conditions; they do not guarantee the performance of the supplied lipid.

Antitumor activity in a humanized leukemia model

Cargo	mRNA1 encoding a CD19/CD3 bispecific antibody
Administration	Intravenous (tail vein)
N/P ratio	Not reported for the cited experiment
Exact formulation	01-1 / DSPC / cholesterol / PEG lipid. Exact molar percentages and PEG-lipid identity are not resolved for this experiment.
Model	Human PBMC-engrafted M-NSG mice with Nalm6-luc leukemia
Dose	3 μg mRNA per mouse, single dose
Comparators	PBS; non-specific mRNA (mRNA-NST), 3 μg/mouse once; recombinant antibody (blinatumomab), 3 μg/mouse daily for three administrations
Endpoint	Tumor bioluminescence (total flux), followed through day 14

The patent explicitly identifies 01-1 as the cationic lipid used in this experiment. The mRNA1-LNP group had lower tumor bioluminescence than the control groups and the recombinant antibody regimen. Figure 4A marks p < 0.01 for the indicated day-14 comparison with recombinant protein. This is a comparison of complete treatment regimens, not equal cumulative doses or proof of tumor eradication.

WO2024131874A1 Figure 4A antitumor study

WO2024131874A1 Figure 4B antitumor study

WO2024131874A1, Example 7, paragraph [0363], Figures 4A–4B (PDF pp. 138–139). No 01-1-specific subcutaneous or non-human-primate experiment is reported here.

Literature results apply to the cited formulation and study conditions; they do not guarantee the performance of the supplied lipid.

Laboratory planning

Solution Calculators

Use the calculated neutral-parent molecular weight of 835.40 g/mol. Confirm solvent compatibility and batch identity before preparing solutions.

Mass = concentration × volume × molecular weight

MassConcentrationVolumeMolecular weightg/mol

Enter any three values to calculate the fourth.

C₁V₁ = C₂V₂

C₁mMV₁mLC₂mMV₂mL

Enter any three values to calculate the fourth.