An Italian group of researchers has used zinc finger editing to silence the PCSK9 gene and improve blood cholesterol levels in mice by applying a single dose of their modifier. The epigenetic-based method could be an alternative to genome editing.
Separate teams of investigators have reported new insights into how the brain disposes of metabolic waste via the glia-based lymphatic system, or glymph system. In two papers published in Nature on Feb. 28, 2024, scientists from Washington University in St. Louis described how in sleeping animals, the synchronized activity of neurons drove ionic gradients that facilitated the movement of fluid through brain tissue. And researchers from the Massachusetts Institute of Technology showed that, in a mouse model of Alzheimer’s disease (AD), the glymphatic system mediated clearance of amyloid-β after sensory stimulation at a 40-Hertz rhythm.
Hangzhou Zhongmei Huadong Pharmaceutical Co. Ltd. has described GTPase KRAS (G12D mutant) inhibitors reported to be useful for the treatment of cancer.
Heptares Therapeutics Ltd. has divulged pyrrolidine-2-carboxamide derivatives and morpholine-3-carboxamide derivatives acting as prostaglandin E2 receptor EP4 subtype (PTGER4; EP4) agonists reported to be useful for the treatment of gastrointestinal and respiratory disorders.
Scientists at Johns Hopkins University and The Lieber Institute for Brain Development have identified peripherally restricted GABA(A) receptor subunits α3β2γ2S (GABRA3) positive allosteric modulators reported to be useful for the treatment of inflammatory bowel disease, lactose intolerance and abdominal pain.
Arvinas Operations Inc. has synthesized proteolysis targeting chimera (PROTAC) compounds comprising a cereblon (CRBN)-binding moiety covalently linked to a non-receptor tyrosine-protein kinase TYK2-targeting moiety via linker.
Iambic Therapeutics Inc. has disclosed quinazoline compounds acting as cyclin-dependent kinase (CDK) inhibitors, particularly CDK2 and/or CDK4 and/or CDK6 inhibitors reported to be useful for the treatment of ovarian and breast cancer.
Pulsesight Therapeutics SAS has launched with seed financing, with the aim of developing disruptive non-viral gene therapies with minimally invasive delivery technology.
PARP inhibitors such as olaparib are used for the treatment of metastatic castration-resistant prostate cancer (mCRPC) harboring homologous recombination deficiency (HRD), but a proportion of these patients do not respond to therapy or eventually develop resistance.
RSS
