Neolaia Inc. has divulged new quinoline- and quinazoline-carboxamide derivatives acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors potentially useful for the treatment of atherosclerosis, cancer, inflammation, obesity, Alzheimer’s, autoimmune, cardiovascular and metabolic disorders.
Insilico Medicine Inc. has reported new NLRP3 inflammasome inhibitors potentially useful for the treatment of obesity, autoimmune and autoinflammatory diseases.
The National Center of Neurology and Tokyo University of Science in Japan have jointly developed new μ-opioid receptor antagonists potentially useful for the treatment of opioid dependence.
Mayo Foundation for Medical Education Research (MFMER) and the University of Minnesota have disclosed new bromodomain-containing protein 4 (BRD4; HUNK1) inhibitors potentially useful for the treatment of cancer, inflammatory and cardiovascular disorders.
Entos Pharmaceuticals Inc. has established a collaboration with the L-CMD Research Foundation with the aim of developing a curative therapy for LMNA-related congenital muscular dystrophy (L-CMD).
Selective inhibition of Werner syndrome helicase (WRN) has been shown to trigger extensive DNA damage and cell death specifically in microsatellite instability-high (MSI-H) cancers, highlighting WRN as a tumor-selective target with potential for precision oncology approaches beyond immunotherapy. Researchers from Eikon Therapeutics Inc. presented the preclinical profile of EIK-1005, a WRN inhibitor.
Researchers from Xenon Pharmaceuticals Inc. described the preclinical efficacy of XPC-837 in models of Dravet syndrome, a severe developmental and epileptic encephalopathy most commonly caused by de novo loss of function mutations in the SCN1A gene.
Addex Therapeutics Ltd. has announced findings from the evaluation of its γ-aminobutyric acid subtype B receptor (GABA[B]) positive allosteric modulator (PAM) in a nonhuman primate (NHP) model of chronic cough. In the study, the GABA(B) PAM drug candidate significantly reduced citric acid-induced cough frequency.
Vanda Pharmaceuticals Inc. presented data this week at the annual American Academy of Neurology conference regarding allele-specific antisense oligonucleotides (ASOs) that specifically target the mutant p.A53T allele from the SNCA gene while preserving the expression of the wild-type allele. The mutant allele is associated with increased risk of early-onset Parkinson’s disease (PD) and current ASOs target SNCA regardless of its mutation status.
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