Multiple sclerosis (MS) is a chronic immune-mediated disease characterized by the destruction of myelin sheaths, neuroaxonal damage, glial cell activation and formation of demyelinated plaques in the CNS. Since MS is considered a prototypic antigen-specific autoimmune disease, restoring immune tolerance to self-antigens is being explored as a therapeutic strategy.
Neushen Therapeutics Inc. has disclosed N-acetyl-β-D-glucosaminidase (O-GlcNAcase; OGA) inhibitors reported to be useful for the treatment of Alzheimer’s disease.
Gene editing technologies are moving forward in preclinical development with innovative strategies designed to treat diseases at their root and even reverse them. However, many approaches still struggle to reach target cells or tissues – either they fail to arrive, or their efficacy is low. In vivo therapies face numerous challenges, but despite these hurdles, 2025 has marked a year of remarkable progress.
Researchers at the University of Sydney have uncovered a mechanism that may explain why glioblastoma returns after treatment, and the world-first discovery offers new clues for future therapies. Glioblastoma is one of the deadliest brain cancers, accounting for about half of all brain tumors, with a median survival rate of just 15 months. Despite surgery and chemotherapy, more than 1,250 clinical trials over the past 20 years have struggled to improve survival rates.
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.
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