Novartis AG has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of familial dysautonomia, Huntington’s disease and spinal muscular atrophy.
University of Sydney has synthesized translocator protein (TSPO; PBR) (A147T mutant) ligands reported to be useful for the diagnosis and treatment of neurological disorders.
Biogen Inc. has disclosed huntingtin (HTT; HD) (mutant) splicing modulators reported to be useful for the treatment of Huntington’s disease and spinal muscular atrophy (SMA).
Alzheimer’s disease (AD) progression involves microglial activation, and restoring or maintaining microglia homeostasis is a therapeutic approach to fight against AD.
Centessa Pharmaceuticals plc has obtained IND clearance from the FDA to initiate a phase I study of ORX-142 in healthy volunteers. ORX-142 is an investigational, novel, highly potent and selective OX2R agonist being developed for the treatment of select neurological and neurodegenerative disorders.
Nanjing Mingde New Drug Research Co. Ltd. has described potassium voltage-gated channel subfamily KQT member 2/3 (KCNQ2/3) activators reported to be useful for the treatment of epilepsy.
Newco Elkedonia SAS has raised €11 million (US$12.7 million) in a seed round, which will fund it to take a potentially first-in-class ELK-1 inhibitor into the clinic in the treatment of refractory depression. ELK-1 plays a pivotal role in neuroplasticity and in reward circuits in the brain, which when disrupted can lead to depression, addiction and post-traumatic stress disorder.
Maplight Therapeutics Inc. has disclosed G protein-coupled receptor GPR52 modulators reported to be useful for the treatment of neurological and psychiatric disorders.
Capsida Biotherapeutics Inc. has obtained IND clearance from the FDA for CAP-003, an intravenously administered gene therapy, for Parkinson’s disease associated with GBA mutations (PD-GBA). A phase I/II trial will begin dosing in the third quarter of this year.
Epilepsy affects more than 70 million worldwide and while several drugs have proven effective for controlling seizures, around 30% of patients do not respond to them and their side effects can be intolerable.