Inhibition of receptor-interacting serine/threonine-protein kinase 2 (RIPK2) has been previously described as a promising strategy for the treatment of inflammatory bowel disease, as RIPK2 is a key player in the signaling leading to bacterial peptidoglycan (PGN)-induced inflammation and it amplifies pro-inflammatory responses in the intestine.
Researchers from Lerna Biopharma Pte. Ltd. (formerly Cargene Therapeutics Pte. Ltd.) recently presented the discovery and preclinical evaluation of a first-in-class GalNAc-siRNA therapeutic, CG-LR1, being developed for the treatment of liver diseases.
CVI Pharmaceuticals has presented preclinical data on their thyroid hormone receptor beta (THRB) selective agonist CVI-2742 for the potential treatment of NASH. Selective activation of the THRB contributes to ameliorating the symptoms of nonalcoholic steatohepatitis (NASH), such as liver inflammation and fibrosis, hepatocyte ballooning and liver steatosis.
REV-ERBα (NR1D1) is a circadian transcriptional repressor that plays a role in the regulation of lipid metabolism and macrophage function, and the global deletion of REV-ERBα has been previously linked to increased microglial activation and mitigation of amyloid plaque formation. In the current study, researchers from Washington University in St Louis and affiliated organizations aimed to explore the cell-autonomous effects of microglial REV-ERBα on tau pathology.
Amyotrophic lateral sclerosis (ALS) is a disorder that leads to progressive muscle weakness and loss of muscle control due to affectation of the motor neurons. Increasing evidence points to defects in the nuclear envelope, which leads to disease progression.
Researchers from Ipsen Ltd. and affiliated organizations presented the discovery and preclinical characterization of a novel NTCP inhibitor, A-7387, being developed for the treatment of HBV and HDV infections.
Researchers from The Brigham and Women's Hospital presented data from a study that aimed to evaluate the role of somatic variants in drug-resistant mesial temporal lobe epilepsy (MTLE). High-coverage whole-exome sequencing was conducted using DNA samples derived from the hippocampus and paired brain tissue and/or blood samples.
Omega Therapeutics Inc. has presented preclinical data on hepatocyte nuclear factor 4-alpha (HNF4A) modulation in fibrotic liver disease models to enhance its key role and suggest it as a potent therapeutic target.