D3 Bio Inc. has obtained IND clearance from the FDA for D3S‑003, enabling initiation of a first‑in‑human phase I trial in patients with advanced solid tumors harboring KRAS G12D mutations.
Mainly expressed in the liver, microRNA‑122‑5p (miR‑122) exhibits increased circulating levels in the context of obesity. When elevated in the bloodstream, miR-122 can act on extrahepatic tissues, including vascular endothelial cells, where it contributes to endothelial dysfunction and may promote the development of diabetic vascular complications.
Brain-derived tau, a protein that is exclusive to the brain and detectable in the blood, could serve as an indicator of brain damage after an ischemic stroke. The analysis of this special form of tau has revealed a relationship between high levels of the protein and extensive brain injury, a higher risk of complications, and poorer outcomes.
Idorsia Pharmaceuticals Ltd. and Shanghai Institute of Materia Medica of The Chinese Academy of Sciences have discovered 1,2,3-triazole derivatives acting as L-amino acid oxidase (IL4I1; LAAO) inhibitors designed for use in the treatment of cancer.
Latigo Biotherapeutics Inc. has synthesized sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers. They are reported to be useful for the treatment of pain, cough and pruritus.
Alexion Pharmaceuticals Inc. has reported complement C1s subcomponent (C1S) inhibitors. They are described as potentially useful for the treatment of amyotrophic lateral sclerosis, rheumatoid arthritis, lupus nephritis and more.
Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous sensorimotor peripheral neuropathies. It is the most frequent inherited neuromuscular disorder affecting 9.7-82.3 patients per 100,000 individuals. Over 100 genes with all patterns of inheritance have been linked to CMT. These genes encode proteins involved in nerve-specific processes, such as axonal transport, myelination and synaptic transmission, and in general housekeeping pathways. However, the reason underlying why defects in such ubiquitous proteins predominantly affect peripheral nerves remains unclear.
About one-third of patients with major depressive disorder (MDD) are treatment resistant. Ketamine is very effective in treatment-resistant depression, but it is associated with psychotomimetic effects. Metabotropic glutamate mGlu2 and mGlu3 receptors negatively regulate the release of glutamate and are involved in the pathogenesis of depression.
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