| DC12145 |
DLinDMA
|
DLinDMA is a key lipid component of stable nucleic acid lipid particles as a benchmark. |
| DC40169 |
DMG-PEG2000
|
DMG-PEG 2000 is used for the preparation of liposome for siRNA delivery with improved transfection efficiency in vitro. DMG-PEG 2000 is also used for the lipid nanoparticle for an oral plasmid DNA delivery approach in vivo through a facile surface modific |
| DC59010 |
C14-4 (C14-494,Lipid B-4,Lipid B4)
|
C14-4 (C14-494,Lipid B-4,Lipid B4) is a novel ionizable lipid with the highest T-cell transfection efficiency and low cytotoxicity.The C14-4 ionizable lipid has been explored for CAR-T therapy.To screen the excellent formulations for mRNA delivery, a
lipid library of 24 ionizable lipids was constructed to make
iLNPs, which were used to deliver luciferase mRNA into
Jurkat cells.[115] The optimal iLNPs formulation was C14-4
iLNPs (C14-4 ionizable lipid, DOPE, chol, and PEG at a molar
ratio of 35%, 16%, 46.5%, and 2.5%) (Figure 6c). The optimal
dose of luciferase mRNA for C14-4 iLNPs was 30 ng.
Compared with electroporated CAR T cells, the CAR T cells engineered
via C14-4 iLNPs showed potent cancer-killing activity
when they were cocultured with Nalm-6 acute lymphoblastic leukemia
cells. To obtain a safer and more effective CAR mRNA
delivery vehicle, the orthogonal design provided 256 potential
formulations, and 16 representative iLNPs formulations were
evaluated.Through evaluating the safety, delivery efficiency,
and transfection efficiency of 16 iLNPs, the formulation B10
(C14-4 ionizable lipid, DOPE, chol, PEG at a molar ratio of
40%, 30%, 25%, and 2.5%) was screened out as the optimal performing formulation. The luciferase expression based on B10
formulation was increased threefold than the initial formulation.
Reducing the accumulation and clearance of iLNPs in the liver
can increase the expression of CAR mRNA in T cells, further
improving the therapeutic effect of CAR-T. Studies have shown
that cholesterol analogs can alter the mechanisms of intracellular
circulation and enhance the delivery of mRNA, which may be
related to the reduced recognition of iLNPs by the Niemann
Pick C1 (NPC1) enzyme.The addition of a hydroxyl
group to various locations in the cholesterol molecule can alter
the binding kinetics between the modified cholesterol and NPC1,
and reduced NPC1 recognition of cholesterol. The results
showed that replacement of 25% and 50% 7 α-hydroxycholesterol
for cholesterol in iLNPs improved mRNA delivery to
primary human T cells in vitro by 1.8-fold and twofold,
respectively.C14-4 is one of the ionizable lipids to efficiently deliver mRNA
to Jurkat cells or primary human T cells. It will effectively promote
the development of mRNA delivery by iLNPs for CAR-T
therapy. |
| DC52028 |
MVL5
|
MVL5 is a new Multivalent Cationic Lipid for siRNA Delivery.Improved total gene silencing and Lower non-specific gene silencing,Lower toxicity. |
| DC53130 |
93-O17S
|
93-O17S is an imidazole-based synthetic lipidoid for in vivo mRNA delivery. Lipid nanoparticles (LNPs) with 93-O17S promotes both the cross-presentation of tumor antigens and the intracellular delivery of cGAMP (STING agonist). |
| DC52020 |
ALC-0159
|
ALC-0159 is a PEG/lipid conjugate (i.e. PEGylated lipid). Formulations containing ALC-0159 have been used in the development of lipid nanoparticles (LNPs) for the delivery of mRNA-based vaccines. |
| DC57046 |
ATX-126(ATX-0126, lipid 10p)
|
ATX-126(ATX-0126, 10p) is an ionizable cationic lipid (pKa = 6.38).It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of siRNA. Intravenous administration of LNPs containing ATX-126(ATX-0126, 10p) and encapsulating Factor VII siRNA decrease Factor VII blood levels in mice. |
| DC59002 |
ssPalmO-Phe(SS-OP)
|
ssPalmO-Phe(SS-OP) is a self-degradable material for the delivery of oligonucleotides. ssPalmO-Phe is a self-degradable derivative of ssPalm that is self-degraded in the intraparticle space by a specific hydrolytic reaction. ssPalmO-Phe is beneficial for overcoming the plasma/endosomal membrane, LNP-ssPalmO-Phe can be used to deliver both nucleic acids. |
| DC57008 |
BAMEA-O16B
|
BAMEA-O16B is a disulfide bond-containing ionizable cationic lipid. BAMEA-O16B, a lipid nanoparticle integrated with disulfide bonds, can efficiently deliver Cas9 mRNA and sgRNA into cells while releasing RNA in response to the reductive intracellular environment for genome editing as fast as 24 h post mRNA delivery. |
| DC58046 |
C12-200
|
C12-200 is a cationic lipid for Lipid NanoParticles as delivery systems for enabling the therapeutic potential of siRNA, mRNA or CRISPR as they exhibit remarkable in vivo potencies at low doses.C12-200 is a common positive control ionizable lipid for
exploring new ionizable lipids. C12-200 is an ionizable
lipid with five tails. The tri-palmitoyl-Sglyceryl
cysteine linked to the pentapeptide (Pam3)-modified C12-200 iLNPs was developed to deliver tumor antigen mRNA
for enhancing the mRNA-mediated cancer immunotherapy.
The results of the therapeutic evaluation showed that Pam3-
modified C12-200 iLNPs could almost inhibit tumor growth in
tumor-bearing mice. To improve delivery efficiency, the
formulation was optimized to replace the phospholipids from
DSPC to DOPE, and the result showed that optimized
formulation induced EPO protein expression was seven times
that of the initial formulation,[35] and the formulation has been
used in the study for mRNA-mediated human α-galactosidase
protein replacement therapy in mice and nonhuman
primates. |
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