Ventus Therapeutics Inc. has nominated a potential first-in-class cyclic GMP-AMP synthase (cGAS) inhibitor, VENT-03, as the company’s first development candidate directed against cGAS.
A model of tauopathy developed in the worm nematode Caenorhabditis elegans was developed for investigating the mechanisms behind tauopathy development.
Satellos Bioscience Inc. has designated SAT-3153 as a development candidate for Duchenne muscular dystrophy and will move the candidate into pre-IND-enabling studies and toward an IND filing. SAT-3153 is a small molecule designed to inhibit a particular kinase protein which the company believes controls Notch polarity within muscle stem cells.
Umecrine Cognition AB has announced results showing that its GABA-A receptor-modulating steroid antagonist golexanolone reversed fatigue, anxiety, depression, and some cognitive and motor alterations in a preclinical model of Parkinson's disease. Compared to a control-operated group, models with Parkinson's disease showed increased fatigue in the treadmill test and anxiety in the open field test.
Vertex Pharmaceuticals Inc. has divulged N-(hydroxyalkyl (hetero)aryl) tetrahydrofuran carboxamides acting as sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, Charcot-Marie-Tooth disease, cough, urinary incontinence and multiple sclerosis.
Rewind Therapeutics NV has described 3-pyrrolylsulfonamide compounds acting as uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-like) antagonists reported to be useful for diagnosis and treatment of neurodegeneration, leukodystrophies, optic neuropathy, Charcot-Marie-Tooth disease, demyelinating, psychiatric and genetic disorders, among others.
Researchers from Yale University and affiliated organizations published data from a study that aimed to assess the mechanistic and pathophysiological importance of plaque-associated axonal spheroids (PAASs) in Alzheimer’s disease (AD).
Orexin OX2 receptor agonists have been described in a recent Sumitomo Pharma Co. Ltd. patent as potentially useful for the treatment of hypersomnia, narcolepsy and sleep apnea, among others.
Several recent patents from Vertex Pharmaceuticals Inc. have disclosed sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain, arrhythmia, Charcot-Marie-tooth disease, cough, urinary incontinence and multiple sclerosis.
2021 was the year Aduhelm (aducanumab, Biogen Inc.) was approved as the first amyloid-β-busting drug for Alzheimer’s disease. And in 2022, there was as much need for an effective AD drug as ever. Aduhelm’s commercial fate was sealed with the decision of the Center for Medicare & Medicaid Services (CMS) that the drug would only be reimbursed in clinical trials approved by the CMS or the NIH.