Long-chain fatty acid beta-oxidation disorders (lcFAOD) are a group of inherited metabolic diseases, characterized by the accumulation of disease-specific acyl-CoAs reflecting the enzyme deficiency. The exact consequence of accumulating lcFAO-intermediates and their influence on cellular lipid homeostasis in patients with these diseases is unknown.
An Italian group of researchers has used zinc finger editing to silence the PCSK9 gene and improve blood cholesterol levels in mice by applying a single dose of their modifier. The epigenetic-based method could be an alternative to genome editing.
Max Biopharma Inc. and Metaba LLC are collaborating to study the effects of oxysterol drug candidates that target metabolic dysfunction-associated steatohepatitis (MASH, formerly nonalcoholic steatohepatitis or NASH), idiopathic pulmonary fibrosis, chronic inflammation, and atherosclerosis on metabolic processes.
Neomorph Inc. has entered into a collaboration and licensing agreement with Novo Nordisk A/S to discover, develop and commercialize molecular glue degraders.
Transthera Sciences (Nanjing) Inc. has announced the initiation of IND-enabling studies for TT-02332, a potent, selective and highly CNS-penetrating NLRP3 inflammasome inhibitor.
1910 Genetics Inc. has divulged non-receptor tyrosine-protein kinase TYK2 (JH2 domain) inhibitors reported to be useful for the treatment of allergy, cancer, inflammation, autoimmune disease, metabolic diseases, endocrine and neurological disorders.
Novel pyrimidinyl and triazinyl sulfonamide derivatives acting as uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-like) antagonists have been reported in an F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. patent.
A Nanjing Suzhong Pharmaceutical Research Co. Ltd. and Suzhong Pharmaceutical Group Co. Ltd. patent describes phenyl-substituted dihydronaphthyridine compounds acting as mineralocorticoid receptor (MR) antagonists potentially useful for the treatment of aldosteronism, cirrhosis, diabetic nephropathy, heart failure, hypertension, myocardial infarction, renal failure and stroke.
Rpxds Co. Ltd. has identified new α-ketoglutarate-dependent dioxygenase FTO inhibitors acting as serum LDL-, cholesterol- and triglyceride-lowering agents and thus reported to be useful for the treatment of obesity, Alzheimer’s disease and obesity-related disorders.
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