| 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. |
|
| DC45847 |
ART558
Featured
|
ART558 is a nanomolar potent, selective, low molecular weight, allosteric DNA polymerase activity of Polθ inhibitor (IC50=7.9 nM). Inhibition of TMEJ with ART558 increases the efficiency of HDR-mediated repair on Cas9-induced DSBs, most profoundly in combination with a NHEJ/DNA-PKcs inhibitor. |
|
| DC60076 |
DEPC
Featured
|
DEPC, also known as DC22:1PC or SJN79954, is a phospholipid containing erucic acid at the sn-1 and sn-2 positions. DEPC is a useful reagent in drug formulation study. It has been used in the study of lipid membranes and to determine the effect of long-chain phospholipids on the secondary structure of human islet amyloid polypeptide (hIAPP).
|
|
| DC57046 |
ATX-126(ATX-0126, lipid 10p)
Featured
|
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. |
|
| DC57100 |
Lipid A9
Featured
|
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. |
|
| DC46965 |
Tri-GalNAc-COOH
Featured
|
tri-GalNAc-COOH is an asialoglycoprotein receptor (ASGPR) ligand that can be used for LYsosome TArgeting Chimera (LYTAC) research. |
|
| DC47280 |
Nusinersen
Featured
|
Nusinersen is an antisense oligonucleotide drug that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein. |
|
| DC47285 |
Tofersen
Featured
|
Tofersen (BIIB067) is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce the synthesis of SOD1 protein. Tofersen can be used for the research of amyotrophic lateral sclerosis (ALS). |
|
| DC47287 |
Tivanisiran
Featured
|
Tivanisiran (SYL1001) is a siRNA used for the study of dry eye disease. Tivanisiran was designed to silence transient receptor potential vanilloid 1 (TRPV1). |
|
| DC47290 |
Remlarsen
Featured
|
Remlarsen (MRG-201), a miR-29b mimic, acts a miR-29b agonist. Remlarsen has the potential for preventiong formation of a fibrotic scar or cutaneous fibrosis. |
|
| DC47291 |
Miravirsen
Featured
|
Miravirsen (SPC-3649), a β-d-oxy-locked nucleic acid-modified phosphorothioate antisense oligonucleotide, inhibit the biogenesis of miR-122. Miravirsen (SPC-3649) is used in the study for HCV infections. |
|
| DC47292 |
Inclisiran
Featured
|
Inclisiran (ALN-PCSsc) is a double-stranded small interfering RNA (siRNA) molecule that inhibits the transcription of PCSK-9. Inclisiran can be used for hyperlipidemia and cardiovascular disease (CVD) research. |
|
| DC47306 |
ARO-AAT
Featured
|
ARO-AAT is a second-generation RNAi drug. ARO-AAT consistes of a cholesterol-conjugated RNAi trigger (chol-RNAi) to selectively degrade AAT mRNA by RNAi and a melittin-derived peptide conjugated to N-acetylgalactosamine (NAG) formulated as the excipient EX1 to promote endosomal escape of the chol-RNAi in hepatocytes. |
|
| DC47308 |
Lumasiran
Featured
|
Lumasiran (ALN-G01), a siRNA product, reduces hepatic oxalate production by targeting glycolate oxidase. By silencing the gene encoding glycolate oxidase, Lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of Primary hyperoxaluria type 1 (PH1). |
|
| DC47310 |
Rovanersen
Featured
|
Rovanersen (WVE-120101) is an antisense oligonucleotide that can be used for huntington’s disease research. |
|
| DC58046 |
C12-200
Featured
|
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. |
|
| DC58047 |
DSPE-PEG 2000
Featured
|
PEG2000-DSPE is used for creating micelles that are able to carry drugs with low solubility. |
|
| DC47877 |
tri-GalNAc-COOH (acetylation)
Featured
|
tri-GalNAc-COOH acetylation is the acetylated and modified form of tri-GalNAc-COOH. tri-GalNAc-COOH acetylation can be used for the synthesis of LYTAC. |
|
| DC59002 |
ssPalmO-Phe(SS-OP)
Featured
|
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. |
|
| DC59010 |
C14-4 (C14-494,Lipid B-4,Lipid B4)
Featured
|
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. |
|
| DC48291 |
ATX-002 |
ATX-002 is a property-tunable lipid for RNA drug delivery. |
|
| DC59126 |
Genevant CL1 (lipid 10)
Featured
|
Genevant CL1 (lipid 10) is a novel ionizable lipid for rna delivery.Lipid 10 rapidly accumulated in the liver within the first hour of dosing (reflecting LNP uptake), but levels then steadily declined over the ensuing 2 weeks period, similar to MC3.Lipid 10 afforded more than double the expression of either approved lipid. We also observed high splenic expression for ALC-0315, which correlated with higher MCP-1 levels.Animals received a single 5 µg IM dose of LNP encapsulating firefly luciferase (fLuc) mRNA. Whole body imaging was performed 6 h later and expression at the injection site quantified. Lipid 10, ALC-0315, and SM-102 showed similar expression at the injection site, all greater than the older generation benchmarks lipids (DLinDMA, KC2, MC3). Lipid 10 and ALC-0315 also showed high expression in the liver, while SM-102 was less, and more similar to MC3.Lipid 10-based LNP reported similar anti-HA IgG titers to MC3 and ALC-0315 (Comirnaty) LNP, and higher than the SM-102 (SpikeVax) LNP composition. MCP-1 levels were generally similar, although the ALC-0315 composition had a significantly higher response at the 5 µg dose. All formulations reported good stability when stored frozen at −80 °C or at 2–8 °C for 1 month. |
|
| DC59217 |
Arcturus lipid 2 |
Arcturus lipid 2(Lipid 2,2 (8,8) 4C CH3,ATX0114 ) is a novel ionizable lipid for mRNA delivery. |
|
| DC49193 |
PEG2000C-DMG
Featured
|
PEG2000-C-DMG, a lipid, can be used for the preparation of Onpattro. Onpattro, a hepatically directed investigational RNAi therapeutic agent, harnesses this process to reduce the production of mutant and wild-type transthyretin by targeting the 3′ untranslated region of transthyretin mRNA. |
|
| DC49194 |
DPPG sodium
Featured
|
DPPG sodium (1,2-Dipalmitoyl-sn-glycero-3-PG sodium) is a phospholipid containing the long-chain(16:0) palmitic acid inserted at the sn-1 and sn-2 positions. DPPG sodium is used in the generation of micelles, liposomes and other types of artificial membranes. |
|
| DC49249 |
DOPS-NA
Featured
|
DOPS-NA is a ubstitute for Phosphoserine/phosphatidylserine. DOPS-NA can be used in lipid mixtures with DOPC and DOPE as effective nontoxic and nonviral DNA vectors. |
|
| DC49257 |
DLin-K-C3-DMA
Featured
|
DLin-K-C3-DMA, a nucleic acid, shows in vivo silencing activity. DLin-K-C3-DMA can be used in the synthesis of nucleic acid-lipid particle to delivery of nucleic acid. |
|
| DC49306 |
DSPE-PEG-Maleimide (MW 2000)
Featured
|
DSPE-PEG-Maleimide, MW 2,000 is a PEG compound with DSPE and maleimide groups. The DSPE-PEGs have been FDA approved for medical applications. The hydrophobic properties of the DSPE allow for the encapsulation and congregation of other hydrophobic drugs. The hydrophilic polyPEG increases the water solubility of the overall compound allowing for the delivery of the drug. Maleimide groups can react with thiol groups between pH 6.5 to 7.5. |
|
| DC49845 |
TT3
Featured
|
TT3 is an ionizable cationic amino lipid that has been used in combination with other lipids in the formation of lipid-like nanoparticles (LLNs). Administration of LLNs containing TT3 and encapsulating mRNA encoding human coagulation Factor IX induces human coagulation Factor IX expression in the plasma of mice. |
|
| DC49882 |
CKK-E12
Featured
|
CKK-E12 is a ionizable lipid in combination with other lipids make up the lipid nanoparticles which are used to deliver RNA-based therapeutics. cKK-E12 was highly selective toward liver parenchymal cell in vivo.Multitail lipids usually have three or more tails and tend to form
more cone-shaped structures due to the increase of tail crosssection,
which enhances the endosome escape and mRNA
delivery efficiency.CKK-E12 is an ionizable lipid with four
lipid tails and diketopiperazine core-based head. It has shown
excellent efficiency in delivering CRISPR-Cas9 mRNA and
sgRNA.cKK-E12 iLNPs encapsulated mRNA was used to
investigate the effect of Toll-like receptor 4 (TLR4) on iLNPsmediated
mRNA delivery, and it has been demonstrated that
the targeting, safety and efficacy of iLNPs are closely related
to disease state. In other words, even though iLNP delivers
therapeutic mRNA to a given cell type in one disease state, it
is not guaranteed to deliver mRNA to the same cell type in
another disease. As same as MC3 and C12-200, CKK-E12 is also
used to be a positive control ionizable lipid when exploiting new
ionizable lipids. |
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