In a recent study published in PNAS, researchers from the University of Texas Southwestern Medical Center investigated cholesterol-mimetic compounds specifically binding and inhibiting Scap. Their final goal was to understand cholesterol regulation by the Scap/SREBP system and identify potential therapeutics for dysregulated lipid metabolism.
The National Institute of Neurological Disorders and Stroke (NINDS) has awarded a 3-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children’s Hospital to fund completion of preclinical studies, manufacturing and preparation of an IND application for a first-in-human trial to advance adeno-associated virus (AAV)-ATP7A gene therapy for the treatment of Menkes disease.
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.
The enticing prospect – and proven worth – of dually agonizing the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors gained more evidence in a big way from Viking Therapeutics Inc. with VK-2735 in a phase II weight loss study. Shares of San Diego-based Viking Therapeutics Inc. (NASDAQ:VKTX) closed Feb. 27 at $85.05, up $46.57, or 121%, as investors learned that the drug achieved the primary and all secondary endpoints in the phase II study called Venture, with significant body-weight drops at all doses compared to placebo.
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.