There is evidence that NACHT, LRR and PYD domains-containing protein 3 (NLRP3)-driven mechanisms may drive both peripheral and hypothalamic inflammation in preclinical obesity. NLRP3 inflammasome activation has been tied to the pathogenesis of obesity-related metabolic syndrome and its progression.
Mwyngil Therapeutics Inc. has in-licensed several lead GPR75 modulator molecules and a proprietary cell-surface discovery platform from Expert Systems Inc.
Ascletis Pharma Inc. has selected ASC-36, a once-monthly, potentially best-in-class subcutaneously administered amylin receptor peptide agonist, as a clinical development candidate.
Genescience Pharmaceuticals Co. Ltd. has identified sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria (PKU).
Suzhou Spring-Sea Bio-Pharmaceuticals Co. Ltd. has described polypeptides acting as glucagon-like peptide 1 receptor (GLP-1R), glucagon receptor (GCGR) and gastric inhibitory polypeptide receptor (GIPR) agonists reported to be useful for the treatment of dyslipidemia, hepatic steatosis, metabolic syndrome, obesity and type 2 diabetes.
Neumora Therapeutics Inc. has reported preclinical data for NMRA-215, a brain-penetrant, oral NLRP3 inhibitor in development for obesity, suggesting potential utility as a monotherapy as well as in combination with a GLP-1 agonist.
Activating the glucagon-like peptide 1 receptor (GLP-1R) is the mechanism of action of most drugs currently used to treat obesity and type 2 diabetes. To enhance efficacy and reduce side effects, many groups have been developing double or triple agonists that, at the same time, also stimulate the receptors for glucagon (GCG) or glucose-dependent insulinotropic peptide (GIP).
Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by hepatocytes.
Genescience Pharmaceuticals Co. Ltd. has identified phenylalanine hydroxylase (PAH) (R408W mutant) stabilizers reported to be useful for the treatment of phenylketonuria (PKU).
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