JWP24 is the first cell-permeable peptide inhibitor of MAGE-A4. JWP24 effectively disruptes the interaction between MAGE-A4 and RAD18, exhibiting an IC50 of 200 nM in a TR-FRET displacement assay and 0.5 μM in a NanoBRET cellular assay.

C210 is a novel HSP90 inhibitor, which overcomes doxorubicin (DOX) resistance of quiescent breast cancer cells by targeting TRAP1. C210 blocks the fragile energy flux of quiescent breast cancer cells prevalent in solid breast cancer tissue by dual inhibition of TRAP1 and HSP90α.

SKPer1 (XMU-MP-8) is a small-molecule degrader of SKP2 that triggers degradation of the oncoprotein SKP2 and specifically kills SKP2-expressing cancer cells.

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.

ATX-054 which is from Arcturus RNA delivery platform, is a novel ionizable lipid used in the formulation of lipid nanoparticles (LNPs) for the delivery of RNA.

MG-101 is a calpain inhibitor (IC50 = 0.09 μM) that activates p53-dependent apoptosis in tumor cell lines.

MG-115(MG 115) is an inhibitor of NFκB in the cytoplasm by acting on IKK or the proteasome.

DMG-PEG-Mal, MW 2,000 is a lipid PEG consisting of DMG and maleimide for reacting with thiols between pH 6.5 to 7.5

Emricasan (IDN-6556, PF-03491390) is a potent irreversible pan-caspase inhibitor.

Vinorelbine is a semi-synthetic Vinca alkaloid which is currently used in treatment of different cancer types mainly advanced breast cancer (ABC) and advanced/metastatic non-small cell lung cancer (NSCLC). Vinorelbine-loaded SSM can be developed as a new,

Izilendustat is a potent inhibitor of prolyl hydroxylase which can stabilize hypoxia inducible factor- 1 alpha (HIF- lα) and hypoxia inducible factor-2 (HIF-2). Izilendustat has the potential for researching diseases that relate to the body’s inmmune response like colitis and other inflammatory bowel diseases (extracted from patent WO2011057115A1 and WO2011057121A1).

An allosteric, sepecific inhibitor of substrate recognition by the SCFCdc4 ubiquitin ligase with IC50 of 6.2 uM in FP assyas.

Seltorexant hydrochloride (JNJ-42847922 hydrochloride) is an orally active, high-affinity, and selective OX2R antagonist (pKi values of 8.0 and 8.1 for human and rat OX2R). Seltorexant hydrochloride crosses the blood-brain barrier and quickly occupies OX2R binding sites in the rat brain.

NP161 is a potent and selective inhibitor of extracellular TRX (Thioredoxin 1) in vitro with IC50 of 0.54 uM.

ARN 23746 (ARN-23746) is a potent, selective inhibitor of Na+-K+-Cl- importer NKCC1, shows NKCC1 inhibition 31.8% at 10 uM, and 95.2% at 100 uM in the Cl− influx assay on NKCC1-transfected HEK293 cells.ARN23746 did not show significant NKCC2 inhibition and KCC2 inhibition at 10 uM.ARN23746 selectively blocks NKCC1 in a human cell line and restore the physiological [Cl−]i in murine DS neurons in culture, has excellent solubility and metabolic stability, and displays no issues with off-target activity in vitro.ARN23746 demonstrated in vivo efficacy in rescuing cognitive impairment in a DS mouse model and social deficits and repetitive behaviors in an autism mouse model.

CICL1 (L829)​​ is a ​​novel ionizable cationic lipid​​ specifically engineered for ​​targeted lipid nanoparticles (tLNPs)​​ that enables efficient in vivo delivery of mRNA payloads to ​​CD8+ T cells​​. Designed to overcome limitations of conventional LNPs, CICL-1 (L-829)​​significantly ​​reduces off-target delivery to the liver​​ and exhibits ​​rapid clearance​​ compared to benchmark lipids like ALC-0315, while demonstrating ​​enhanced biodegradability and tolerability​​ in rodent and primate models. When incorporated into CD8-targeted tLNPs, CICL 1 (L829 enables ​​preferential transfection of CD8+ T cells​​ over other immune subsets, facilitating the generation of functional ​​anti-CD19 or anti-CD20 CAR T cells directly *in vivo​​*. These tLNP-engineered CAR T cells mediate ​​rapid, deep B-cell depletion​​ in humanized mice and cynomolgus monkeys, with repopulating B cells exhibiting a naïve phenotype suggestive of immune reset. By eliminating the need for ex vivo manufacturing or lymphodepleting chemotherapy, the L829-tLNP platform represents a ​​safer, scalable approach​​ for accessible CAR T therapy in oncology and autoimmune diseases.

ISM012-042 is an orally active PHD1 and PHD2 inhibitor with IC50 values of 1.9 and 2.5 nM, respectively. ISM012-042 (2.5 μM) can protect Caco-2 cells from DSS-induced barrier disruption. In lipopolysaccharide (LPS)-induced mouse bone marrow-derived dendritic cells (BMDC), ISM012-042 has anti-inflammatory effects and can dose-dependently reduce the expression of IL-12 subunit IL-12p35 and TNF. ISM012-042 restores intestinal barrier function and alleviates intestinal inflammation in various experimental colitis models. ISM012-042 can be used for intestinal mucosal repair and research into immune diseases.

Montelukast is a potent, selective and orally active antagonist of cysteinyl leukotriene receptor 1 (CysLT1). Montelukast can be used for the reseach of asthma and liver injury. Montelukast also has an antioxidant effect in intestinal ischemia-reperfusion injury, and could reduce cardiac damage.

Zafirlukast(Accolate)

DSPE-PEG-N3 is a click chemical PEG reagent. Azide group is easy to react with alkynyl group under the catalysis of copper catalyst. It can also react directly with DBCO without any catalyst. DSPE (1,2-distearoyl-sn-glycerol-3-phosphoethanolamine) is a saturated 18 carbon phospholipid, which is commonly used in the synthesis of liposomes. Polyethylene glycol (PEG) - coupled DSPE is hydrophilic and can be used for drug delivery, gene transfection and biomolecular modification. The PEG of phospholipid significantly improved the blood circulation time and stability of the capsule drug. Polyethylene glycol can enhance the solubility and stability, reduce the non-specific binding of charged molecules on the surface, and reduce the immunogenicity of polypeptides. Azide (- N3) can react with copper catalyzed alkynes in aqueous solution and be reduced to amino groups. Polyethylene glycol can increase solubility and stability. It can also inhibit the nonspecific binding of charged molecules on the modified surface.

Potent inhibitor of protein arginine methyltransferases (PRMTs)

Thalidomide-5-CH2-NH2 (hydrochloride) is the Thalidomide-based cereblon ligand used in the recruitment of CRBN protein. Thalidomide-5-CH2-NH2 (hydrochloride) can be connected to the ligand for protein by a linker to form PROTACs.

C12-200 is a well-known cationic lipid used in the formulation of lipid nanoparticles (LNPs) for the delivery of therapeutic nucleic acids, including siRNA, mRNA, and CRISPR components. It is widely recognized for its high in vivo potency at low doses and is often used as a positive control ionizable lipid in research exploring new ionizable lipids.

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).

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.

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.

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.

Lipid 2,2(8,8) 4C CH3 is an ionizable cationic lipid (pKa = 6.69).1 It has been used in the generation of lipid nanoparticles (LNPs) for the delivery of siRNA in vivo. LNPs containing lipid 2,2(8,8) 4C CH3 and encapsulating siRNA targeting Factor VII decrease plasma Factor VII protein levels by 90% in mice.

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.

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.