huntington's disease

MIND4-19 is an inhibitor of SIRT2 with antitumor activity.

Selisistat (EX-527) is a potent and specific inhibitor of the deacetylase SIRT1 (IC50 = 38 nM) and can be utilized in the study of neurological disorders such as Huntington's chorea.

Dalzanemdor (SAGE-718) is an NMDA receptor-positive modulator of metabolism that can be used to study Huntington's chorea, Alzheimer's disease, and cognitive dysfunction.

BN-82451 2HCl is a dual inhibitor of COX-1 and COX-2 and can be used to study Huntington's chorea and Parkinson's disease.

Rosolutamide (ALZ-003) is a curcumin analog, an orally active Nrf1 and Nrf2 activator.Rosolutamide modulates oxidative homeostasis, improves mitochondrial function and promotes autophagy, reduces mutant protein aggregation, and decreases intracellular mitochondrial reactive oxygen species (ROS) levels.Rosolutamide has been used in the study of neurodegeneration such as spinal cerebellar ataxia and Huntington disease. neurodegenerative diseases such as Huntington's disease. Regulates oxidative homeostasis.

HTT-D3 is an orally active, highly efficient Huntington splicing modulator. It induces HTT mRNA degradation, reduces HTT protein levels, and inhibits P-glycoprotein (P-gp) efflux. This compound can be used in Huntington's disease research.

Tetrabenazine (Ro 1-9569) is a former antipsychotic drug used to treat various movement disorders. It inhibits neurotransmitter uptake into adrenergic storage vesicles and serves as a high-affinity label for the vesicle transport system.

Dantrolene is a postsynaptic muscle relaxant by inhibiting Ca2+ ions release from sarcoplasmic reticulum stores by antagonizing ryanodine receptors.

CHDI 00484077 is a highly selective, central nervous system (CNS) permeable Class IIa HDAC inhibitor with inhibitory effects on HDAC4, HDAC5, HDAC7, and HDAC9, which improves neuronal function, and can be used to study Huntington's disease.

Tetrabenazine Metabolite ((-)-β-HTBZ), a vesicles monoamine transporter 2 (VMAT2) inhibitor, is an active metabolite of Tetrabenazine. Tetrabenazine Metabolite has a high affinity for VMAT2 with a Ki of 13.4 nM. Tetrabenazine Metabolite is often used to study chorea associated with Huntington's disease and other hyperactivity disorders.

B2 (Linazolamide intermediate B impurity 2) promotes inclusion formation in cellular models of Huntington's disease and Parkinson's disease

NCT-504, a selective allosteric inhibitor of PIP4Kγ, exhibits an IC50 value of 15.8 μM. It holds promise for the investigation of Huntington's disease.

PBT-1033 (PBT-2) is a potential neuroprotective agent for the treatment of Alzheimer's disease and Huntington's disease.

1-phenyl-3,4-dihydroisochinoline (3,4-Dihydro-1-phenylisoquinoline) is a structural analog of the neurotransmitter dopamine that interacts with dopamine receptors in the central nervous system. It has been studied as a potential therapeutic agent for the treatment of neurological disorders such as Parkinson's disease, Alzheimer's disease and Huntington's chorea.

1-(Anilinocarbonyl)proline is a compound utilized for identifying dual-action probes within a cell model of Huntington's disease.

Chloroguanabenz is an antiprion agent and a derivative of the α2-adrenergic receptor agonist guanabenz . It inhibits prion formation in yeast and mammalian cell-based assays. Chloroguanabenz reduces the levels of both soluble and aggregated forms of the truncated Huntingtin derivative Htt48 in an HEK293T cellular model of Huntington's disease.

Votoplam functions as a gene splicing modulator, employed in the inhibition of Huntington's disease [1].

SEN177 shows the potential in the research of Huntington's disease that is a potent inhibitor of glutaminyl cyclase (QPCT) with an IC 50 of 0.013μM for glutaminyl-peptide cyclotransferase-like (QPCTL). The Ki value of SEN177 for human glutaminyl cyclase (hQC) is 20 nM. SEN177 significantly reduces the early stages of mutant HTT oligomerisation and reduces the percentage of neurons with Q80 aggregates [1].

Rho-Kinase-IN-2 (Compound 23) is an orally active and selective inhibitor of Rho Kinase (ROCK) with CNS penetration, exhibiting a high affinity for ROCK2 with an IC50 of 3 nM. This compound is of potential interest for further investigations in Huntington's disease research [1].

Votoplam HCl (PTC-518 HCl) is the hydrochloride form of Votoplam.Votoplam is a systemically acting gene splicing regulator that down-regulates the levels of huntingtin protein, which is primarily used to inhibit Huntington's disease (HD).

CAY10786 (GPR52 antagonist-1) is an antagonist of G protein-coupled receptor 52 (GPR52, IC50 = 0.63 μM).

Compound P110 (Drp1 peptide inhibitor P110) is a selective Drp1 peptide inhibitor with neuroprotective effects. It inhibits the activation of Drp1, prevents Drp1 mitochondrial translocation induced by MPTP, and alleviates dopamine neuron loss, dopaminergic terminal damage, and behavioral deficits caused by MPTP. This compound is useful for Alzheimer's disease research. Additionally, Compound P110 reduces mitochondrial and organ damage in animal models of Huntington's disease, cerebral ischemia, and myocardial infarction.

HDAC4 CHDI Degrader 11 is a potent, selective degrader of HDAC4 (PROTAC) with DC_50 values of 4 nM and 6 nM in Jurkat E6-1 and Jurkat cells, respectively. This compound integrates the class IIa HDAC inhibitor trifluoromethyloxadiazole with a ligand for the Von Hippel-Lindau (VHL) protein through a linker. Demonstrating significant efficacy, HDAC4 CHDI Degrader 11 achieves a DC_50 value of 1 nM in a mouse cell model of Huntington's disease. For enhanced degradation in neuroblastoma cell lines, it can be administered alongside the P-glycoprotein inhibitor, Elacridar.

PMX-53, a macrocyclic complement 5a (C5a) peptidomimetic and C5a receptor antagonist (IC50 = 0.3 µM), effectively inhibits the C5a-induced secretion of myeloperoxidase (MPO) in isolated human polymorphonuclear leukocytes (PMNs). Administered orally at 10 mg kg, PMX-53 mitigates vascular leakage, PMN infiltration, and the production of TNF-α and IL-6 in a rat peritoneal Arthus reaction model. Additionally, in a 3-nitropropionic acid (3-NP)-induced Huntington's disease rat model, it alleviates body weight loss, anorexia, and striatal degeneration. Furthermore, at a dose of 3 mg kg, PMX-53 decreases atherosclerotic lesion size and lipid content in ApoE- - mice.