amyloidosis

PD146176 (NSC-168807) is an inhibitor of 15-Lipoxygenase (15-LO) , it inhibits rabbit reticulocyte 15-LO with Ki of 197 nM and IC50 of 0.54 μM. PD146176 reverses cognitive impairment, brain amyloidosis, and tau pathology by stimulating autophagy in aged triple transgenic mice.

Tauroursodeoxycholate (UR 906), also known as ursodoxicoltaurine, is a highly hydrophilic tertiary bile acid that is produced in humans at a low concentration. Tauroursodeoxycholate is the more hydrophilic form of ursodeoxycholic acid, which is the more abundant naturally produced bile acid in humans.Tauroursodeoxycholate is being investigated for use in several conditions such as Primary Biliary Cirrhosis (PBC), insulin resistance, amyloidosis, Cystic Fibrosis, Cholestasis, and Amyotrophic Lateral Sclerosis.

Eprodisate disodium (NC-503) is a negatively charged sulfonated inhibitor of fibrillogenesis, used in the treatment of amyloid A (AA) amyloidosis.

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.

amyloid P-IN-1 can be used in studies about diseases with depletion of serum amyloid P components such as Alzheimer's disease, amyloidosis, osteoarthritis, and type 2 diabetes mellitus.

Dichlorophenyl-ABA is a transthyretin (TTR) amyloid fibril formation inhibitor that effectively suppresses pathological protein aggregation, inhibiting more than 80% of aggregate formation in TTR L55P-expressing cells, Dichlorophenyl-ABA is widely employed in amyloidosis research to study TTR stabilization, aggregation mechanisms, and therapeutic intervention strategies.

3-Demethylthiocolchicine, a colchicine analog, exhibits broad-spectrum antitumor activity and shares similar properties and activities with colchicine in inhibiting casein-induced amyloidosis, microtubule binding, and anti-inflammatory effects. Notably, it has significantly lower toxicity compared to colchicine.

(9R)-RO7185876 (Compound example 16) is a γ-secretase inhibitor. It reduces the secretion of Αβ42. This compound can be employed in the research of Alzheimer's disease, cerebral amyloid angiopathy, hereditary cerebral hemorrhage with amyloidosis, multi-infarct dementia, senile dementia, or Down syndrome.

Inotersen (ISIS-420915) sodium is a 2′-O-methoxyethyl–modified antisense oligonucleotide that selectively targets transthyretin RNA, reduces TTR transcript and protein levels. Inotersen serves as a validated molecular tool for mechanistic and translational research into hereditary transthyretin amyloidosis polyneuropathy.

Transthyretin-IN-4 (Compound B26) is a bivalent inhibitor of transthyretin (TTR) amyloidosis, with a bIC50 of 0.09 µM and a pIC50 of 1.4 µM. It is applicable for research in heart failure with preserved ejection fraction (HFpEF) and life-threatening arrhythmias.

Patisiran sodium is a double-stranded small interfering RNA (siRNA) that selectively binds a defined sequence within transthyretin (TTR) messenger RNA. Patisiran sodium possess potent and specific suppression of hepatic synthesis of both mutant and wild-type TTR protein, and its mechanism-based activity underpins its significant application in hereditary TTR amyloidosis research, gene silencing therapeutics development, and RNA interference–based translational medicine.

Revusiran (ALN-TTRSC) is a first-generation short interfering RNA (siRNA) that specifically targets transthyretin (TTR) mRNA and is utilized in TTR-mediated amyloidosis research.

Vutrisiran (ALN-TTRsc02) is an investigational, liver-directed small interfering RNA compound designed to selectively silence transthyretin gene expression, Vutrisiran is extensively used in research on transthyretin-mediated amyloidosis to evaluate RNA interference–based therapeutic strategies, disease-modifying mechanisms, and long-term suppression of pathogenic protein production.

Eprodisate is a novel compound specifically developed to disrupt the interactions between amyloidogenic proteins and glycosaminoglycans, effectively impeding the polymerization of amyloid fibrils and their deposition in tissues. This compound shows potential for delaying the advancement of AA amyloidosis-related renal disease and could apply to various forms of amyloidosis.

Acoramidis hydrochloride (Alxn2060 hydrochloride) is an orally active and selective kinetic stabilizer of WT and V122I- TTR (transthyretin) . Acoramidis hydrochloride is used in the study for transthyretin amyloidosis.

Poloxamer 188 is a nonionic triblock copolymer surfactant and bioactive excipient used as a medical excipient to exacerbate cerebral amyloidosis, presynaptic dystrophy, and pathogenic microglial cell activation in 5XFAD mice.

Eplontersen is a triantennary N-acetylgalactosamine–conjugated antisense oligonucleotide that selectively inhibits both wild-type and mutant transthyretin mRNA, thereby reducing pathogenic TTR protein production and amyloid fibril formation associated with ATTR amyloidosis affecting cardiac and peripheral nerve tissues.

Birtamimab (NEOD001) is a monoclonal antibody selectively targeting and depleting amyloid protein, used in research on light chain amyloidosis.

Dezamizumab (GSK 2398852) is a fully humanized recombinant monoclonal immunoglobulin G1 (IgG1) antibody specifically targeting the serum amyloid P component (SAP), a protein universally found in all types of amyloid deposits, and it possesses potential anti-amyloid activity by triggering an immunotherapeutic mechanism for the clearance of amyloid fibrils through macrophage-mediated phagocytosis, which can be applied in the research of amyloid light-chain (AL) amyloidosis and other systemic amyloid diseases.

Anti-amyloid agent-1 is a potent compound that inhibits amyloid aggregation, offering a promising approach for research into the treatment of amyloidosis [1].

PITB, a selective and orally active transthyretin (TTR) aggregation inhibitor, is utilized in transthyretin amyloidosis (ATTR) disease research [1].

WAY-612453 is an active compound utilized in researching amyloid diseases and synucleinopathies.

Anselamimab is a humanized IgG1κ monoclonal antibody targeting serum amyloid A1 (SAA1). It removes amyloid deposits by specifically binding to amyloid fibrils and triggering phagocytosis by macrophages and neutrophils.Anselamimab was used as an APP(β-amyloid A4 Protein Precursor ) inhibitor in the clinical phase for the treatment of light chain (AL) amyloidosis.

Transthyretin-IN-2 (Compound 36), a transthyretin (TTR) amyloidosis inhibitor, exhibits IC50 and pIC50 values of 1.31 μM and 5.88 μM, respectively. It is useful for researching TTR amyloidosis diseases [1].