Although congenital aortic valve disease (AVD) is one of the most prevalent types of congenital heart disease, affecting 1% to 2% of the population, the understanding of the molecular genetics and mechanisms underlying the disease remains limited.
The Nipah virus (NiV) from the Henipavirus genus is a bat-borne zoonotic pathogen that causes encephalitis and acute respiratory distress syndrome in humans. NiV infections have a very high fatality rate. No approved vaccine or therapeutics against NiV infection exist for use in humans despite its dangerous potential for causing larger epidemics.
K36 Therapeutics Inc. has obtained IND clearance from the U.S. FDA for KTX-2001, a selective, oral, small-molecule inhibitor of nuclear receptor-binding SET domain protein 2 (NSD2), a histone methyltransferase and oncogene that activates gene expression in some cancers.
Methicillin-resistant Staphylococcus aureus (MRSA) is the leading cause of nosocomial infections, with an increasing prevalence worldwide. Researchers aimed to apply a new approach called “pseudo-natural product” (PNP) scaffolds for antibacterial development, and hypothesized that the indotropane class of PNPs might show potential activity against bacterial strains.
Proteinqure Inc. has received regulatory clearances from the U.FDA and Health Canada to initiate a phase I trial of lead candidate, PQ-203. The trial will begin in Canada and expand to U.S. sites later in 2025. The FDA also granted PQ-203 fast track designation for triple-negative breast cancer (TNBC).
Homeobox genes, a conserved family of transcription factors, are key regulators of embryogenesis, cell growth and differentiation, and have been linked to bone mass regulation and osteogenesis. However, their specific roles in postmenopausal osteoporosis and osteoclast development are still not well understood, with limited and fragmented knowledge on which genes are central to these processes.
Rivo Bio Inc. has disclosed compounds acting as 5-HT2A receptor agonists reported to be useful for the treatment of depression, schizophrenia and anxiety disorders.
Hefei Institutes of Physical Science Chinese Academy of Sciences has synthesized phosphoglycerate kinase 1 (PGK1) inhibitors reported to be useful for the treatment of inflammatory bowel disease.
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