Injectable glucagon-like peptide 1 receptor (GLP-1R) agonists have shown efficacy in weight management and glycemic control. Semaglutide is available as an oral formulation, but it requires specific timing and conditions. Carmot Therapeutics Inc. has presented data for its oral GLP-1R agonist CT-996 from preclinical studies in obesity; the drug is currently in early clinical development.
There is growing evidence of the role of soluble endoglin in the biology of platelets, including thrombosis. French researchers have investigated the role of genetic variants in the gene encoding endoglin, ENG, and the risk of venous thrombosis development.
CAR T-cell therapy that targets BCMA is an effective option for treating relapsed/refractory multiple myeloma, but there is a lack of persistence due to the inability to develop a memory phenotype.
The generation of anti-citrullinated protein autoantibodies (ACPA) are known key drivers in the pathogenesis of rheumatoid arthritis (RA) and are generated by peptidyl arginine deiminases (PADs).
Transmembrane serine protease 6 (TMPRSS6) is a negative regulator of hepcidin, which is the main iron homeostasis-regulating hormone. Regeneron Pharmaceuticals Inc. has recently presented preclinical data for the monoclonal antibody targeting TMPRSS6, REGN-7999, which is being developed for the treatment of iron overload.
Flamingo Therapeutics BV and Ionis Pharmaceuticals Inc.’s FTX-001 (also known as FLM-7523) is a potential first-in-class antisense oligonucleotide (ASO) targeting the human metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) long noncoding RNA (lncRNA); it is being developed for the treatment of cancer.
A recent paper has identified the enhancer ETS2, located in a so-called gene desert, as a contributor to five separate immune disorders. It also showed that one of ETS2’s target genes mediating this inflammation was the eminently druggable MEK, a kinase that is the target of the FDA-approved inhibitors Mekinist (trametinib, GSK plc), Mektovi (binimetinib, Array Biopharma Inc.), Cotellic (cobimetinib, Roche Holding AG) and Koselugo (selumetinib, Astrazeneca plc/Merck & Co. Inc.).
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