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.
The obesity market is hot with Novo Nordisk A/S generating $6.3 billion in sales from its semaglutide-containing drugs, Ozempic, Saxenda, Rybelsus and Wegovy, in the first quarter of 2024 and Eli Lilly and Co. bringing in $2.3 billion for its tirzepatide-containing drugs, Zepbound and Mounjaro, in the same quarter. It appears unlikely the market will be a two-horse race for much longer though.
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.
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