Vanishing white matter disease (VWM) is a rare and progressive leukoencephalopathy caused by loss-of-function mutations, in a recessive pattern of inheritance, in any of the genes encoding eIF2B, a guanine nucleotide exchange factor for eIF2 and an effector of the integrated stress response (ISR). At last week’s American Academy of Neurology meeting, Calico Life Sciences LLC and Abbvie Inc. presented preclinical results for their brain-penetrant compound ABBV-CLS-7262 (fosigotifator sodium tromethamine) in VWM.
Wista Laboratories Ltd. has disclosed thiazole-containing compounds acting as microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of corticobasal syndrome, Alzheimer’s disease, Down syndrome and more.
Biointervene Inc. has described adenosine analogues acting as adenosine A3 receptor (ADORA3) agonists reported to be useful for the treatment of mild cognitive impairment, diabetic neuropathy, irritable bowel syndrome, neurodegeneration, ototoxicity, neuropathic pain, chemotherapy-induced peripheral neuropathy and spinocerebellar ataxia, among others.
Neurocrine Biosciences Inc. has synthesized vesicular monoamine transporter 2 (VMAT2) inhibitors reported to be useful for the treatment of hyperkinesia and psychosis.
University of California Oakland has described potassium channel subfamily K member 2 (TREK-1; KCNK2) and/or potassium channel subfamily K member 10 (TREK2; KCNK10) activators reported to be useful for the treatment of Caisson disease (decompression sickness), depression, headache, pulmonary hypertension, insomnia, chronic pain, lung and neuronal Injury.
Trace amine-associated receptor 1 (TAAR1) is a member of the G protein-coupled receptor (GPCR) TAAR family, which has been shown to be enriched in the central nervous system and periphery. TAAR1 can couple to diverse G protein subtypes, including Gs, Gq and Gi. Shandong University investigators have recently reported the discovery of a novel TAAR1 agonist as an antipsychotic drug candidate.
Voyager Therapeutics Inc. has announced the selection of a lead development candidate in the GBA1 gene therapy program for the treatment of Parkinson’s disease and other GBA1-mediated diseases under its collaboration with Neurocrine Biosciences Inc.
Shandong Ruzhi Biomedical Technology Co. Ltd. has synthesized compounds reported to be useful for the treatment of neurodegeneration, cardiovascular disorders and cerebrovascular disorders.
Researchers from Hefei Industrial Pharmaceutical Institute Co. Ltd. published data detailing the design and biological evaluation of novel NMDAR-GluN2B antagonists as potential candidates for the treatment of ischemic stroke.
Bioage Labs Inc. has synthesized NLRP3 inflammasome inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, Alzheimer’s disease, Parkinson’s disease and traumatic brain injury.