Synucleozid hydrochloride (NSC 377363 hydrochloride) is a potent inhibitor of the SNCA mRNA that encodes alpha-synuclein protein (IC50=1.5 uM). Synucleozid selectively targets the alpha-synuclein mRNA 5? UTR at the designed IRE site, decreases the amount of SNCA mRNA loaded into polysomes and thereby inhibits SNCA translation. Synucleozid has the potential for the investigation of Parkinson's disease[1]. IC50 & Target: IC50: 1.5 uM (SNCA IRE RNA)[1] In Vitro: Synucleozid (0.25-1 uM; 24 hours) abrogates cytotoxicity induced by alpha-synuclein preformed fibrils, which act as seeds and recruit endogenous alpha-synuclein to aggregate[1]. Synucleozid (0-1 uM; 24 hours) binds to the A bulge near the base of the IRE hairpin, reduced levels of alpha-synuclein in a dose-dependent manner with an IC50 of 500 nM, and inhibits alpha-synuclein protein expression in SH-SY5Y neuroblastoma cells[1]. Synucleozid (100 nM-100 uM; 24 hours) binds to 2-AP-labeled and native IRE RNA with similar affinities. It decreases 2-AP emission with an EC50 value of 2.7 uM, recovery of 2-AP emissions is observed as a function of unlabeled SNCA IRE RNA (RNA-0) concentration, affording a competitive Kd of 1.5 uM[1]. Synucleozid (0.25-1 uM; 24 hours) decreases alpha-synuclein and other proteins that have IREs in their mRNA's UTR including APP, PrP, Ferritin and TfR as a dose-dependent mannner. All panels is completed in SH-SY5Y cells, except for PrP protein which is assessed in Neuro-2A cells[1].

MK-8591 (Islatravir, MK8591, EFdA) is a nucleoside analog that exhibits remarkable potency against wild-type and drug-resistant HIVs, inhibits HIV-1 reverse transcriptase with multiple mechanisms.

Nacubactam (OP 0595, RG 6080, RO 7079901) is a diazabicyclooctane that inhibits class A and C β-lactamases with IC50 of <1 uM, also acts as a PBP2-active antibacterial.

2BAct (eIF2B activator 2BAct) is a novel, highly selective, CNS-permeable small molecule eIF2B activator with EC50 of 33 nM in cell-based reporter assays.

Pamiparib (BGB-290, BGB290) is a highly potent, selective and brain penetrant PARP1/2 inhibitor with potent anti-tumor activity.

MYLS22 is a first-in-class and selective optic atrophy 1 (OPA1) inhibitor. MYLS22 can target endothelial OPA1 to curtail tumor growth and inhibits angiogenesis by impinging on NFkB activity and on angiogenic gene expression.

SHIN1 is a potent dual inhibitor of human serine hydroxymethyltransferase 1/2 (SHMT1/2).

Novel FABP4/5 inhibitor, inhibiting lipolysis in 3T3-L1 adipocytes and in primary human adipocytes

Novel potent and cell-active PARG inhibitor, selectively killing BRCA-mutant and PARP inhibitor-resistant cancer cells

TX1-85-1 is a potent, selective, ATP-competitive Her3 (ErbB3) inhibitor with IC50 of 23 nM, forms a covalent bond with Cys721 located in the ATP-binding site of Her3.

A potent, selective, orally active S1P1 antagonist with IC50 of 2.5 nM Ca2+ mobilization assays, without activity on S1P2, S1P3 and S1P4 (IC50>10 uM).

ML299 is a potent, CNS penetrant, dual phospholipase PLD1/PLD2 probe with IC50 of 6 nM/20 nM, respectively.

A BIX-01294 derivative that inhibit malaria parasite histone methyltransferases, resulting in rapid and irreversible parasite death.

CLK inhibitor T3 (T3) is a highly potent, selective, and cell-based stable CDC-like kinase (CLK) inhibitor with 0.67, 15 and 110 nM for CLK1,2 and 3, respectively.

Novel Orally Available Polymerase Inhibitor, Showing Efficacy Against a Lethal Morbillivirus Infection in a Large Animal Model

R243 is a potent, small molecule CCR8 antagonist that inhibits CCR8 signaling and chemotaxis, inhibits CCL1-induced Ca2+ flux and CCL1-driven peritoneal macrophages aggregation at 0.2 uM.

Ribocil is a synthetic mimic of riboflavin 5’-monophosphate (FMN) that competes with the natural ligand to inhibit FMN riboswitch-mediated expression of ribB.

ONO-8130 is an orally available EP1 receptor antagonist.

PS 210 is a potent, specific activator of PDK1 that target the PIF-binding pocket of PDK1, does not alter the activity of any of the 121 kinases (S6K, PKB/Akt or GSK3)..

ATP competitive tyrosine kinase inhibitor

Novel cyclin dependent kinase inhibitor

MC180295 (MC-180295, MC 180295) is a novel potent, highly selective CDK9 inhibitor with IC50 of 5 nM, displays >22-fold selectivity over other CDKs.

Cedazuridine (E7727) is a novel cytidine deaminase inhibitor, and a componet of ASTX727 for treatment of hematologic malignancies..

3-Hydroxy-4-methoxyacetophenone(Acetoisovanillone; Isoacetovanillone) is an active compound isolated from P. spinosa. Isoacetovanillone possesses anti-inflammatory activity and prevented injuries due to administration of acetic acid in the colon.

Lubiprostone, also known as RU-0211, is a medication used in the management of chronic idiopathic constipation, predominantly irritable bowel syndrome-associated constipation in women and opioid-induced constipation. It was initially approved by the U.S. Food and Drug Administration (FDA) in 2006. Lubiprostone is a bicyclic fatty acid derived from prostaglandin E1 that acts by specifically activating ClC-2 chloride channels on the apical aspect of gastrointestinal epithelial cells, producing a chloride-rich fluid secretion. These secretions soften the stool, increase motility, and promote spontaneous bowel movements (SBM).

RU-TRAAK-2 is a completely reversible TRAAK inhibitor, shows no activity for non-K2P channels (Kv1.2, Slo1 and GIRK2).

LY2365109 is a potent and selective GlyT1 inhibitors with IC50 value of 15.8 nM.

Myelin Oligodendrocyte Glycoprotein 35-55, mouse, rat (MOG 35-55) is a minor component of CNS myelin that induces experimental autoimmune encephalomyelitis in C57BL/6 mice by an encephalitogenic T cell response.

An orally active, irreversible inhibitor of protein disulfide isomerase (PDI), forming a covalent bond with the active site cysteines of PDI.

SI-2 (EPH-116) is a potent small-molecule inhibitor of steroid receptor coactivator-3 (SRC-3 or AIB1) that can selectively inhibit the intrinsic transcriptional activities of SRC-3, also inhibits SRC-1 and SRC-2.