Setoxaximab is an IgG1-κ humanized chimeric antibody targeting shiga toxin type 1.

Pritoxaximab is an IgG1κ antibody targeting shiga toxin type 1.

TDI-015051 is a first-in-class non-covalent inhibitor of the viral guanine-N7 methyltransferase (MTase) NSP14 with Kd of 61 pM and IC50 ≤ 0.15  nM, respectively.

Cholesterol is a component of Lipid nanoparticles (LNPs) for RNA delivery.

LTX-401 is a novel oncolytic amino acid derivative that specifically targets the Golgi apparatus, demonstrating significant potential in cancer therapy. In vitro studies reveal that LTX-401 effectively reduces the viability of various tumor cell lines, exhibiting cytotoxic activity across a range of concentrations. Notably, it shows the highest potency against the human malignant melanoma cell line MDA-MB-435S (IC50 = 13.5 μM) and the lowest activity against the human hepatocellular carcinoma cell line HEPG2 (IC50 = 35.4 μM). For other cell lines, the IC50 values fall within a narrow range of 19-32 μM. Importantly, LTX-401 does not induce hemolysis in red blood cells at concentrations effective for cancer cell death, with hemolytic activity only observed at much higher concentrations (400 μg/mL = 1087 μM). Additionally, LTX-401 demonstrates cytotoxicity against non-malignant cell lines, including HUV-EC-C endothelial cells, HaCat keratinocytes, and MRC-5 fibroblasts, suggesting a broad but selective mechanism of action.

Prodrug of the glycolysis inhibitor 3-bromopyruvate (3-BrPA) which targets hexokinase II (HK2) and GAPDH

Indacaterol is a potent, orally administered ultra-long-acting β2 adrenergic receptor (ADRB2) agonist, widely recognized for its therapeutic potential in respiratory and cardiovascular conditions. By selectively activating ADRB2, indacaterol not only enhances bronchodilation but also exhibits a unique mechanism of action by inhibiting NF-κB activity in a β-arrestin2-dependent manner. This dual functionality helps mitigate lung inflammation, prevent further pulmonary damage, and improve lung function in patients with chronic obstructive pulmonary disease (COPD). Beyond its respiratory applications, indacaterol has also emerged as a valuable tool in cardiovascular disease research, offering insights into its broader therapeutic effects. Its multifaceted pharmacological profile positions indacaterol as a promising candidate for addressing complex disease mechanisms and advancing treatment strategies in both respiratory and cardiovascular fields.

FSLLRY-NH2 is a protease-activated receptor 2 (PAR2) inhibitor.

Edonerpic, also known as T-817, is a neuroprotectant. Edonerpic is a candidate therapeutic agent for Alzheimer's disease that inhibits oxidative stress and nitric oxide-induced neurotoxicity and acts as a neurotrophic factor. Edonerpic protects against MPTP-induced neurotoxicity by blocking lipid peroxidation in the SNc, and imply that this compound may be useful for treating neurodegenerative disorders related to oxidative stress, such as Parkinson's disease.

GNF-PF-3777 (8-Nitrotryptanthrin) is a potent human indoleamine 2,3-dioxygenase 2 (hIDO2) inhibitor which significantly reduces IDO2 activity with Ki of 0.97 μM.

m7GpppAmpG Na salt is a trinucleotide 5′ cap analog with the capping efficiencies for the obtained RNAs of 90%.

m7GpppAmpG ammonium is a trinucleotide 5′ cap analog with the capping efficiencies for the obtained RNAs of 90%.

3'OMe-m7GpppAmpG ammonium solution (100mM) is a trinucleotide Cap analogue. 3'OMe-m7GpppAmpG ammonium shows a significant translational efficiency. 3'OMe-m7GpppAmpG ammonium can be used as a potential molecular biology tool in the field of mRNA vaccines and mRNA transfection, such as protein production, gene therapy and anti-cancer immunization.

PEG(2000)-C-DMG represents a PEGylated derivative synthesized from 1,2-dimyristoyl-sn-glycerol (1,2-DMG). This compound is instrumental in the formulation of lipid nanoparticles (LNPs), particularly when combined with other lipidic compounds, for the efficient delivery of small interfering RNA (siRNA). Specifically, LNPs incorporating PEG(2000)-C-DMG and encapsulating siRNA designed to target the genes responsible for encoding programmed cell death protein ligand 1 (PD-L1) and PD-L2 have demonstrated efficacy in reducing the expression levels of PD-L1 and PD-L2 in monocyte-derived dendritic cells. Furthermore, formulations that include PEG(2000)-C-DMG are being actively explored in the advancement of LNPs aimed at the delivery of siRNA-based vaccines, highlighting its critical role in the development of therapeutic and prophylactic strategies.

Rovanersen (WVE-120101) is an antisense oligonucleotide that can be used for huntington’s disease research.

DPPE-MPEG(2000) is a PEGylated form of 1,2-dipalmitoyl-rac-glycero-3-PE (DPPE). It has been used in the synthesis of anionic liposomes for drug redistribution and toxicity prevention.

DC-Chol is a cationic cholesterol derivative. DC-Chol, as a component of lipoplexes with DOPE, has been used for transfection of mRNA into A549 cells without affecting cell viability. Incubation of DC-chol/DOPE liposomes or lipoplexes with human whole blood has no effect on neutrophil elastase or β-thromboglobulin levels or the number of platelets and red and white blood cells, indicating hemocompatibility. DC-Chol has also been widely used in the synthesis of liposomes for the delivery of siRNA, DNA, and chemotherapeutic agents into cells and mice.

Azido-acetal linker is stable at physiological pH (7.4) but rapidly hydrolyzes in the acidic environment of endosomes useful as a fragment of lipid synthesis.

C14-4 Core (Core 4) is the core structure of C14-4.

DMAP-BLP is a lipid for RNA and vaccine delivery.DMAP-BLP exhibits optimized bilayer destabilizing and pKa properties leading to highly potent gene silencing in hepatocytes following IV administration that is similar to “gold standard” lipids such as DLinMC3-DMA.

KRIBB3 is an Hsp27 and microtubule inhibitor that inhibits migration and invasion of MDA-MB-231 cells in vitro in an Hsp27-dependent manner.

E7090 (E 7090) is a highly potent and selective orally bioavailable inhibitor targeting FGFR1, FGFR2, and FGFR3, with IC50 values of 0.71 nM, 0.50 nM, and 1.2 nM, respectively. It exhibits significantly weaker inhibition of FGFR4, with an IC50 of 120 nM. This distinct selectivity profile positions E7090 as a promising therapeutic candidate for diseases driven by aberrant FGFR1-3 signaling, offering a targeted approach to inhibit these receptors while minimizing off-target effects on FGFR4. Its oral availability further enhances its potential as a convenient and effective treatment option for patients with FGFR-dependent conditions.