| 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. |
| DC31000 |
LP-01
|
LP-01 is an ionizable cationic amino lipid (pKa = ~6.1). It has been used in the generation of lipid nanoparticles (LNPs). LNPs containing LP-01 and encapsulating both Cas9 mRNA and modified single-guide RNA (sgRNA) for the transport protein transthyretin (Ttr) induce gene editing in liver cells in mice in a dose-dependent manner resulting in reduced serum Ttr levels for at least 12 months. |
| 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. |
| DC42537 |
ALC-0315
|
ALC-0315 is an ionisable aminolipid that used for mRNA compaction and aids mRNA cellular delivery. ALC-0315 can be used to form lipid nanoparticle (LNP) delivery vehicles. |
| DC57006 |
L319
|
L319 (LIPID 319) is a novel ionizable, biodegradable lipid for delivery of short interfering RNAs (siRNAs). L319-LPN displays rapid elimination with pKa of 6.38 and also shows well tolerated up to 10 mg/kg. |
| DC52025 |
SM-102
|
SM-102 is an ionizable amino lipid that has been used in combination with other lipids in the formation of lipid nanoparticles.Administration of luciferase mRNA in SM-102-containing lipid nanoparticles induces hepatic luciferase expression in mice. Formulations containing SM-102 have been used in the development of lipid nanoparticles for delivery of mRNA-based vaccines. |
| DC31024 |
SM-86
|
SM86 is a cationic, ionizable lipid developed by Moderna as a core component of its lipid nanoparticle (LNP) platform for mRNA therapeutic delivery.SM-086 is structurally optimized and analogous to SM-102 (used in Moderna’s COVID-19 vaccines), with modifications aimed at enhancing mRNA delivery efficiency and safety.SM-86 serves as the primary cationic lipid in three investigational mRNA therapies targeting rare metabolic disorders:mRNA-3927: Restores propionyl-CoA carboxylase activity in propionic acidemia (PA).
mRNA-3705: Delivers methylmalonyl-CoA mutase mRNA for methylmalonic acidemia (MMA).
mRNA-3210: Provides phenylalanine hydroxylase mRNA to treat phenylketonuria (PKU). |
| DC57100 |
Acuitas Lipid A9
|
Lipid A9 is an ionizable cationic lipid (pKa = 6.27) that has been used in the generation of lipid nanoparticles (LNPs) for the delivery of mRNA and siRNA in vivo. LNPs containing lipid A9 and encapsulating non-stimulatory siRNA increase plasma levels of chemokine (C-C motif) ligand 2 (CCL2), indicating activation of the innate immune response, and decrease body weight in mice. |
| DC65412 |
Acuitas Lipid III-2
|
Acuitas Lipid III-2 is an ionizable amine lipid with two identical ester tails adjacent to C6 position relative to amine from patent:WO2017075531A1 with the similar activity as ALC-0315. The head of lipid is propanolamine which can effectively encapsulate mRNA used in gene therapies which depends on the availability of a safe and efficient delivery vehicle. |
| DC67217 |
Moderna Lipid 48
|
Moderna Lipid 48 is an novel ionizable amine lipid used for mRNA delivery from Moderna patent WO2017049245A2 |
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