It has been previously demonstrated that the two coding variants in the APOL1 gene (G1 and G2) are associated with a greater risk of progressive, proteinuric kidney disease; however, there currently are no therapies to address the causal genetic drivers of this disease. Researchers from Maze Therapeutics Inc. presented the discovery and preclinical characterization of a novel small-molecule inhibitor of APOL1, MZ-302, and they evaluated its efficacy in a new transgenic model of APOL1-mediated kidney disease (AKD).
Researchers from the University of Queensland discovered that senolytic therapies can suppress long-COVID neuropathology and long-term disorders caused by viral infections by reducing senescent cells, thereby reducing inflammation. Published Nov. 13, 2023, in Nature, the study examined the use of human pluripotent stem cells to generate small mini human brain organoids to screen for antiaging interventions called senolytics that selectively eliminate senescent cells that accumulate with age, lead author Julio Aguado told BioWorld.
The GOBACK study analyzed germline susceptibility in children with birth defects and a cancer diagnosis, including a total of 47 probands. Whole-genome sequencing was performed, and researchers evaluated single-nucleotide variants, insertion/deletion variants, as well as copy number variations.
Fundamental Pharma GmbH has described TRPM4/NMDA interaction inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis (ALS).
Aligos Therapeutics Inc. has divulged bicyclic compounds acting as viral replication inhibitors reported to be useful for the treatment of hepatitis B (HBV) and hepatitis D virus infections.
The outcomes from two investigational CRISPR-based therapies were presented by researchers from Crispr Therapeutics at the recent American Heart Association’s scientific sessions.
Nuevolution A/S has synthesized bromodomain-containing protein 4 (Brd4, HUNK1) inhibitors reported to be useful for the treatment of cancer, hypoxia, fibrosis, autoimmune disease, inflammatory disorders and viral infections.
Shenzhen Forward Pharmaceuticals Co. Ltd. has disclosed fibroblast growth factor receptor 2 (FGFR2) inhibitors reported to be useful for the treatment of cancer.
CLDN18 is a cell surface membrane protein involved in the formation of tight junctions, and the expression of its CLDN18.2 isoform has been observed in pancreatic, gastric and esophageal tumors.
Sirpant Immunotherapeutics Inc.’s lead product candidate, Sirpant-M (SI-101), has been awarded U.S. orphan drug designation for the treatment of T-cell lymphoma.