Seelos Therapeutics Inc. has announced in vivo data demonstrating that a single dose of SLS-004 downregulated the production of α-synuclein in an established α-synuclein overexpressing animal model of Parkinson's disease.
The farnesoid X receptor (FXR), a bile acid receptor, plays a direct role regulating innate immune cells in the gut, and treating mice with an FXR agonist improved symptoms of inflammatory bowel disease (IBD). The findings provide a link between diet and innate immunity, and could lead to better ways to treat IBD. “The intestine is a major target for inflammation and inflammatory disease, particularly in the modern Western culture, where high-fat diets are becoming very prevalent,” Ronald Evans told BioWorld.
Repeat expansions of two or more base pairs cause dozens of neurological disorders – Huntington’s disease, which is caused by an expansion of the triplet CAG in the coding sequence for huntingtin, is perhaps the most famous one. Now, investigators at Stanford University have shown that cancer genomes, too, frequently feature repeat expansions.
Investigators from Cerevance Inc. have reported the discovery and preclinical characterization of a novel tandem pore domain halothane-inhibited K+ channel 1 (THIK-1) inhibitor, C-101248, being developed for the treatment of neuroinflammation in Alzheimer’s disease (AD). NETSseq and histological analysis revealed that THIK-1 expression was up-regulated in microglia from different cortical regions of AD donors compared with aged matched nondemented control brains.
Roche has synthesized pyruvate dehydrogenase kinase 1 (PDK1) inhibitors reported to be useful for the treatment of systemic lupus erythematosus, psoriasis, inflammatory bowel diseases, idiopathic pulmonary fibrosis, chronic obstructive pulmonary disease and cancer.
Blossomhill Therapeutics Inc. has identified macrocyclic compounds acting as GTPase KRAS (G12D mutant) inhibitors reported to be useful for the treatment of cancer.