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.
Roivant Sciences Ltd. CEO Matt Cline said the firm’s unit Immunovant Inc. with FcRn blocker batoclimab has established “frankly a new bar” in myasthenia gravis (MG) as the New York-based firm reported top-line results from its phase III study and first data from period 1 of the phase IIb study with the same drug in chronic inflammatory demyelinating polyneuropathy. The data look promising, and Immunovant intends to use the findings to help advance second-generation FcRn prospect IMVT-1402 in both indications. Potentially registrational trials are planned. The U.S. FDA has granted IND clearance.
Wall Street was weighing the gravity of the death from acute liver failure of a patient who was treated for Duchenne muscular dystrophy (DMD) with Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec). Liver injury is a known possible side effect of the product, first approved by the U.S. FDA in June 2023 for DMD, as well as other AAV-mediated gene therapies, and the potential problem is highlighted in Elevidys’ prescribing information.
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.
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