A review of 2025's noteworthy advances in medical research, including GLP-1 receptor agonists as anti-aging drugs, tumor-agnostic therapies and xenotransplants.
Huayao Jiyuan (Shenzhen) Pharmaceutical Co. Ltd. has described hypoxia inducible factor-2α (HIF-2α; EPAS1) inhibitors reported to be useful for the treatment of cancer, infections, cardiovascular disorders, anemia, chronic kidney disease, disorders of hematopoiesis, inflammatory disorders and acute respiratory distress syndrome (ARDS).
Chengdu Easton Biopharmaceuticals Co. Ltd. has divulged sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, cough, urinary incontinence and multiple sclerosis.
Insilico Medicine Inc. has identified serine/threonine-protein salt-inducible kinases (SIK) inhibitors reported to be useful for the treatment of rheumatoid arthritis, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), giant cell arteritis, primary sclerosing cholangitis, inflammatory bowel disease, atherosclerosis, type 2 diabetes and glomerulonephritis, among others.
Neurodawn Pharmaceutical Co. Ltd. has synthesized phosphodiesterase PDE3A inhibitors reported to be useful for the treatment of dementia and cerebrovascular disorders.
Guangzhou Institutes of Biomedicine and Health has disclosed bromodomain-containing protein 4 (BD2 domain) (BRD4 BD2) inhibitors reported to be useful for the treatment of diabetes, autoimmune diseases, cardiovascular disorders, male contraception, chronic obstructive pulmonary disease, viral and parasitic infections and cancer.
Alagille syndrome (ALGS) is a rare, multisystem genetic disorder most commonly caused by haploinsufficiency of the JAG1 gene, leading to reduced JAG1 protein function and impaired development of intrahepatic bile ducts. Researchers from Arnatar Therapeutics Inc. described the development of antisense oligonucleotides (ASOs) engineered using their proprietary ACT‑UP1 platform to upregulate endogenous JAG1 expression and thereby address the underlying genetic deficiency.
Multiple sclerosis (MS) is a chronic immune-mediated disease characterized by the destruction of myelin sheaths, neuroaxonal damage, glial cell activation and formation of demyelinated plaques in the CNS. Since MS is considered a prototypic antigen-specific autoimmune disease, restoring immune tolerance to self-antigens is being explored as a therapeutic strategy.
Venetoclax has shown good results for adult acute myeloid leukemia (AML) in combination with azacitidine, but there is increasing evidence of inherent and acquired resistance. High expression of nicotinamide phosphoribosyltransferase (NAMPT) has been associated with cancer aggressiveness and poor prognosis due to increased nicotinamide metabolism.
Previous work showed that histone ‘readers’, which bind to post-translational modifications on histones, may be upregulated in melanoma and thereby upregulate oncogenes. Given that the histone reader ATAD2 is known to be overexpressed in several types of cancer, researchers at the University of Alabama at Birmingham asked whether the same is true in melanoma.
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