Enigma Biomedical USA Inc. has selected two four-repeat tau (4R tau) protein PET imaging biomarkers to advance into phase I studies. These imaging biomarkers show promise as important new tools in advancing understanding of neurodegenerative diseases in which the misfolded 4R tau protein is implicated.
Jiangsu Hengrui Medicine Co. Ltd. and Shanghai Hengrui Pharmaceutical Co. Ltd. have disclosed Toll-like receptor 7 (TLR7) and/or TLR8 and/or TLR9 antagonists reported to be useful for the treatment of rheumatoid arthritis, multiple sclerosis, systemic lupus erythematosus and Sjögren’s syndrome.
Investigators from Insmed Inc. have presented new preclinical data on the efficacy of their adenoviral vector (AAV9)-based gene therapy INS-1201 for the treatment of Duchenne muscular dystrophy (DMD).
Nitrase Therapeutics Inc. has unveiled a protein named glyoxalase domain-containing protein 4 (GLOD4) responsible for catalyzing selective protein nitration. This reflects a new class of enzymatic activity discovered by the company that had not been previously characterized.
At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X (ss.AAV9.oTAM and ss.AAV9.hE50-sgRNA).
Quiver Bioscience Inc. is collaborating with the Dup15q Alliance to advance an antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome.
Domain Therapeutics SA has nominated PAR2 antagonist DT-9046 as a drug candidate with potential to treat various inflammatory diseases, including atopic dermatitis, inflammatory bowel disease and arthritis, as well as neuroinflammatory conditions such as migraine.
Scientists at St. John’s University, The University of Pennsylvania and Université de Montréal have identified poly(ADP-ribose) polymerase 1 (PARP-1; ARTD1) inhibitors reported to be useful for the treatment of cancer, Lewy body dementia, amyotrophic lateral sclerosis, Alzheimer’s disease and Parkinson’s disease.
In work conducted by Jining Medical University investigators, synthesis and optimization of a previously reported ATP-competitive pyrrolo[2,3-b]pyridine-based glycogen synthase kinase 3β (GSK-3β) inhibitor led to the discovery of compound [I], a candidate with significant inhibitory activity on GSK-3β (IC50=0.35 nM), with its inhibitory effect being approximately 12-fold greater than that of broad-spectrum GSK-3β inhibitor staurosporine and 189-fold greater than that of the parent compound.
Scientists at Medical University of Vienna, The University of Queensland and Universität Innsbruck have identified cysteine stapled dynorphin A (CSD) analogues acting as κ-opioid receptor antagonists reported to be useful for the treatment of anxiety disorders, depression, substance abuse and dependence, and stress disorders.
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