misfolding

VAS 3947 is a specific inhibitor of NADPH oxidase (NOX).VAS 3947 has a potent anti-platelet effect, inducing apoptosis through UPR activation, mainly due to protein aggregation and misfolding, independent of anti-NOX activity.

Icerguastat (IFB-088) is a selective inhibitor of holophosphatase. Sephin1 safely and selectively inhibited a regulatory subunit of protein phosphatase 1 in vivo.

IFB-088 acetate, a benzyl guanidine derivative, is used to treat diseases and cancers associated with the PPP1R15A pathway and associated with protein misfolding stress, Examples include tau disease, synaptic riboprotein disease, polyglutamine and polyalanine diseases, leukodystrophy, cystic fibrosis, multiple sclerosis, lysosome storage disorders, amyloidosis diseases, inflammation, metabolic disorders, cardiovascular diseases, osteoporosis, neurological trauma, and more.

Sephin1 (IFB-088) is reportedly a selective inhibitor of GADD34 (PPP1R15A), which is a stress-induced regulatory subunit of protein phosphatase 1 complex that dephosphorylates eIF2α

Quinacrine is a compound that is commonly used as an anti-protozoal agent. It inhibits voltage-dependent sodium channels (IC50: 3.3 μM) and suppresses aldehyde oxidase (IC50: 3.3 μM). Quinacrine prevents misfolding of prion protein (EC50: 0.3 μM). As an e

Fe-TMPyP binds to the prion protein PrP and inhibits misfolding. Fe-TMPyP is also a peroxynitrite decomposition catalyst.

The metallo-protein Cu/Zn-superoxide dismutase (SOD1) is a ubiquitous enzyme responsible for scavenging superoxide radicals. Mutations in SOD1, which alter its metal binding capacity and can result in protein misfolding and aggregation, have been linked to familial amyotrophic lateral sclerosis (ALS). Cu-ATSM is an orally bioavailable, blood-brain barrier permeable complex that has traditionally been used in cellular imaging experiments to selectively label hypoxic tissue via its susceptibility to reduction by oxygen-depleted mitochondria. More recently, Cu-ATSM has been reported to improve locomotor function and survival in a transgenic ALS mouse model by delivering copper specifically to cells in the spinal cords of mice producing misfolded SOD1 proteins. Copper chaperone for SOD (CCS) is presumed to utilize the copper from Cu-ATSM to prevent misfolding of the SOD1 protein.

NPT200-11 is an orally bioavailable and brain penetrating inhibitor of accumulation of alpha-synuclein (ASYN) misfolding and aggregation. NPT200-11 can be used in studies about the underlying pathology of synucleinopathies including Parkinson's disease (PD), dementia with Lewy bodies (DLB) and Multiple Systems Atrophy (MSA).

Minzasolmin(DLX-313) is an alpha-synuclein misfolding inhibitor for the study of Parkinson's disease.

CFTRcorrector 14 (Compound SVQ26) functions as a Class 3 corrector targeting the cystic fibrosis transmembrane conductance regulator (CFTR), enhancing CFTR activity (improving activity in the presence of Class 1 corrector VX-809 with an EC50 of 3.08 μM). It addresses the misfolding and functional impairments of CFTR proteins caused by mutations. CFTRcorrector 14 is used in cystic fibrosis research.