Proof-of-concept murine data demonstrated that delivery of anticodon engineered tRNAs to muscle cells restored production of full-length, native dystrophin.
Understanding psychiatric disorders at a cellular and molecular level could provide a different perspective to design diagnostic and therapeutic tools searching for the origin of these disorders and the alterations they cause. Fourteen simultaneous studies from the PsychENCODE Consortium have delved into the cellular atlases of human neurodevelopment, reporting the broadest view of neuropsychiatry to date.
Boehringer Ingelheim Pharma GmbH & Co. KG has disclosed dioxane derivatives acting as mGluR4 negative allosteric modulators reported to be useful for the treatment of neurodegeneration, metabolic disease, cancer, psychiatric disorders and neurological disorders.
Degron Therapeutics Inc. has entered into a collaboration and exclusive license agreement with Takeda Pharmaceutical Co. Ltd. to discover and develop novel molecular glue degraders for multiple targets in oncology, neuroscience and inflammation.
The potential of psychedelic substances such as lysergic acid diethylamide (LSD) and psilocybin in treating various neuropsychiatric disorders has received increased attention in recent times. These compounds are believed to exert their effects through the serotonin 5-HT2A receptor.
Cybin Irl Ltd. has described phenethylamine compounds acting as 5-HT2 receptor agonists reported to be useful for the treatment of lung diseases, cardiovascular disorders, inflammatory disorders, psychiatric disorders and neurological disorders.
Neurocrine Biosciences Inc. has divulged G protein-coupled receptor GPR52 modulators reported to be useful for the treatment of schizophrenia, obsessive-compulsive disorder, attention deficit hyperactivity disorder, bipolar disorder, vascular cognitive impairment, and Alzheimer’s, Parkinson’s and Huntington’s diseases, among other neurological disorders.
Astellas Pharma Inc. and Kotobuki Pharmaceutical Co. Ltd. have disclosed NLRP3 inflammasome inhibitors reported to be useful for the treatment of neurodegeneration and inflammatory disorders.
CTNNB1 syndrome is a rare neurodevelopmental disorder that is caused by mutations in the gene encoding β-catenin, CTNNB1, which plays a critical role in neuronal development, synapse formation and brain maturation.
Neuroninn Biosciences Co. Ltd. has disclosed G protein-coupled receptor GPR139 agonists reported to be useful for the treatment of anxiety, depression, Parkinson’s disease and more.