Scientists at Osaka University and Tokyo University of Science have described compounds targeting Claudin-5 (CLDN5) acting as blood-brain barrier (BBB) permeability regulators reported to be useful for the treatment of sepsis, cerebral edema, infections, epilepsy, multiple sclerosis, psychiatric disorders, Alzheimer’s disease and Parkinson’s disease, among others.
Researchers at Meiji Seika Pharma Co. Ltd., National Center of Neurology & Psychiatry and Tokyo University of Pharmacy & Life Sciences have synthesized NAD(+) H\hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of neurodegeneration.
Pheno Therapeutics Ltd. has disclosed uracil nucleotide/cysteinyl leukotriene receptor (GPR17; P2Y-Like) antagonists reported to be useful for the treatment of multiple sclerosis, amyotrophic lateral sclerosis, Alzheimer’s disease and Parkinson’s disease.
Tonix Pharmaceuticals Holding Corp. has licensed exclusive worldwide rights to TNX-4900 (formerly PW-507) from Rutgers University. TNX-4900 is a highly selective, small-molecule sigma-1 receptor (S1R) antagonist, which has demonstrated analgesic activity in multiple models of neuropathic pain.
ADEL Inc. closed a year-end licensing deal worth up to $1.04 billion with Sanofi SA for ADEL-Y01, a specific tau-targeting Alzheimer’s disease drug candidate in a U.S. phase I study.
Formation Bio Inc. acquired ex-China rights to Lynk Pharmaceuticals Co. Ltd.’s oral TYK2-inhibitor, LNK-01006, for up to $605 million. The phase I-ready central nervous system (CNS) candidate will be developed at Formation’s newly formed subsidiary, Bleecker Bio.
Superoxide dismutase 1 (SOD1) mutations were among the first genetic causes identified in familial amyotrophic lateral sclerosis (ALS) and confer a toxic gain-of-function that drives motor neuron degeneration via protein misfolding, oxidative stress, mitochondrial dysfunction and neuroinflammation.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive degeneration of upper and lower motor neurons, resulting in paralysis and death typically within 3-5 years of symptom onset. Historically, treatment options have been extremely limited. However, the identification of genetic contributors to ALS pathogenesis has enabled the application of antisense oligonucleotides (ASOs) to selectively modify or reduce the expression of disease-associated genes at the RNA level.
Sometimes the darkest products (names) bring a bright spot of news to their developers, as the U.S. FDA clearance for Galway, Ireland-based Medtronic plc's Onyx liquid embolic system demonstrates. Onyx gained the indication for embolization of the middle meningeal artery as an adjunct to surgery for the treatment of symptomatic subacute or chronic subdural hematoma based on the EMBOLISE trial results. The approval represents a significant advancement in treating this common neurosurgical condition.
Researchers from Wuhan Institute of Biomedical Sciences, Jianghan University and collaborators recently showed that activating the endogenous transcription factors Ngn2 and Isl1 via CRISPRa can directly reprogram astrocytes into functional motor neurons in the mouse spinal cord.
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