Separate teams of investigators have reported new insights into how the brain disposes of metabolic waste via the glia-based lymphatic system, or glymph system. In two papers published in Nature on Feb. 28, 2024, scientists from Washington University in St. Louis described how in sleeping animals, the synchronized activity of neurons drove ionic gradients that facilitated the movement of fluid through brain tissue. And researchers from the Massachusetts Institute of Technology showed that, in a mouse model of Alzheimer’s disease (AD), the glymphatic system mediated clearance of amyloid-β after sensory stimulation at a 40-Hertz rhythm.
Arthex Biotech SL has received FDA clearance to initiate the phase I/IIa Arthemir study of ATX-01 for the treatment of myotonic dystrophy type 1 (DM1).
Researchers from Macquarie University have detailed the discovery of a novel gene therapy vector targeting pathological TAR-binding protein 43 (TDP-43), CTx-1000, as a potential therapeutic candidate for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) – two diseases characterized by cytoplasmic deposition of the nuclear TDP-43.
Transthera Sciences (Nanjing) Inc. has announced the initiation of IND-enabling studies for TT-02332, a potent, selective and highly CNS-penetrating NLRP3 inflammasome inhibitor.
Vandria SA has disclosed urolithin and fluorene derivatives acting as ferroptosis inhibitors and reported to be useful for the treatment of neurodegeneration and inflammatory disorders.
Voyager Therapeutics Inc. has announced the selection of a lead development candidate from its Friedreich’s ataxia program with collaborator Neurocrine Biosciences Inc.
Open Source Therapeutics has patented new aromatic substituent-containing (hetero) aryl-imidazole compounds acting as bifunctional epoxide hydrolase 2 (EPHX2; sEH) inhibitors.