Ono Pharmaceutical Co. Ltd. and Wuxi Apptec Co. Ltd. have jointly developed new ceramide glucosyltransferase (glucosylceramide synthase; GLCT-1) and lysosomal acid glucosylceramidase (GBA; β-glucocerebrosidase; β-glucosidase) dual inhibitors reported to be useful for the treatment of mild cognitive impairment, among others.
Investigators from Abzyme Therapeutics LLC have hypothesized that inhibiting this pathway in the CNS may prevent tissue damage and cease the progression of multiple sclerosis (MS).
Argenx SE’s ARGX-119 is a monoclonal antibody (MAb) targeting the muscle-specific kinase (MuSK) Frizzled (Fz)-like domain, and has entered early clinical development for the treatment of neuromuscular diseases.
Vanderbilt University has identified leucine-rich repeat kinase 2 (LRRK2; dardarin) and LRRK2 (G2019S mutant) inhibitors reported to be useful for the treatment of tauopathies, cancer, Crohn’s disease, type 1 diabetes, leprosy, rheumatoid arthritis, traumatic brain injury and Alzheimer’s disease, among others.
Researchers from the Chinese Academy of Medical Sciences reported the discovery of the 1,2,4-oxadiazole derivative FO-4-15 for the treatment of Alzheimer’s disease (AD).
Japan Tobacco Inc. has identified NLRP3 inflammasome inhibitors reported to be useful for the treatment of arteriosclerosis, gout, inflammatory bowel disease, amyotrophic lateral sclerosis, multiple sclerosis, metabolic dysfunction-associated steatohepatitis (MASH; formerly nonalcoholic steatohepatitis [NASH]), Alzheimer’s disease and Parkinson’s disease.
Secondary progressive multiple sclerosis (SPMS) is a chronic form of disease that occurs after relapsing-remitting MS, with a progressive disease course, and its pathogenesis remains unclear. CX3C chemokine receptor 1 (CX3CR1) is a G protein-coupled receptor that may be a useful marker of Eomes+ Th cells; the antigen has been shown to be expressed by cytotoxic Th cells and required for late-onset disease.
The Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) meeting in Copenhagen this week is celebrating its 40th edition. In recognition of this landmark, the plenary session and opening lecture were attended by Queen Margrethe of Denmark. Afterward, the hot topic session on neuroprotective therapies set the stage for the subsequent discussions on the latest trends in the management and treatment of multiple sclerosis (MS).
Papillon Therapeutics Inc.’s PPL-001 has been awarded orphan drug designation by the FDA for Friedreich’s ataxia. PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy.
2A Biosciences Inc. has divulged compounds acting as 5-HT2A and 5-HT2C receptor agonists reported to be useful for the treatment of atopic dermatitis, conjunctivitis, Crohn’s disease, type 2 diabetes, obsessive-compulsive disorder, rheumatoid arthritis, schizophrenia and traumatic brain injury, among others.