Hepatocellular carcinoma (HCC) is a complex disease where both the activation of the cyclic GMP-AMP synthase-stimulator of the interferon gene (cGAS-STING) pathway and the induction of immunogenic cell death (ICD) have proven effective immunotherapeutic strategies in some instances.
Ariceum Therapeutics GmbH has received approval from the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) to conduct a phase I trial (CITADEL-123) of 123I-ATT-001, its iodine-123 labeled PARP inhibitor, in patients with recurrent glioblastoma. The study is expected to begin in the U.K. in June of 2024.
Dyadic International Inc.'s Dutch subsidiary, Dyadic Nederland BV, has entered into a strategic partnership agreement and collaboration with Rabian BV to develop efficacious, scalable, and affordable rabies prophylactics and vaccines utilizing Dyadic's C1 protein production platform.
Long-chain fatty acid beta-oxidation disorders (lcFAOD) are a group of inherited metabolic diseases, characterized by the accumulation of disease-specific acyl-CoAs reflecting the enzyme deficiency. The exact consequence of accumulating lcFAO-intermediates and their influence on cellular lipid homeostasis in patients with these diseases is unknown.
Researchers from Capital Medical University have presented preclinical data for the novel PDZ-binding kinase (PBK) inhibitor, HI-TOPK-032, which is being developed for the treatment of cancer. The study aimed to assess the effects of the candidate on NK-92MI cell infiltration into ovarian tumors.
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.
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