A new method for accelerating the maturation of neuronal cell models and brain organoids is poised to make it possible to track the etiology of neurodegenerative diseases that develop over decades. The non-invasive technique uses graphene to convert light into electrical cues that prompt neurons to connect and communicate in vitro.
UCB SA has divulged compounds acting as NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of inflammation, injury, neurodegeneration, and eye, autoimmune and neurological disorders, among others.
Loss-of-function mutations in the CTNNB1 gene cause β-catenin deficiency, leading to CTNNB1 syndrome, a rare neurodevelopmental disorder marked by motor and cognitive impairments. Because the disease stems from single-allele mutations that vary widely, a broadly applicable corrective strategy is needed. Since CTNNB1 is dosage-sensitive, therapies must preserve normal regulation, and in some cases may also need to suppress harmful mutant transcripts.
Jazz Pharmaceuticals plc and Saniona AB have entered into a global license agreement for Jazz to obtain exclusive worldwide rights to develop and commercialize SAN-2355 for epilepsy and other potential indications.
Lario Therapeutics Ltd. has synthesized voltage-dependent R-type calcium channel subunit α-1E (Cav2.3) blockers reported to be useful for the treatment of epilepsy, neurodegeneration, neurodevelopmental disorders, Parkinson’s disease, cerebral vasospasm and endocrine disorders.
Receptor-interacting protein kinase 1 (RIPK1) plays a pivotal role in ischemic stroke pathology by mediating necroptosis and promoting neuroinflammation, both of which contribute to secondary brain injury and worsen clinical outcomes.
Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion.
One of the major obstacles to treating neurological disorders such as Alzheimer’s disease is the blood-brain barrier: drugs that are injected into the circulation usually do not enter the brain effectively. Researchers at Denali Therapeutics Inc., Biogen Inc. and the University of Minnesota have devised a vehicle for transporting antibodies against amyloid-β that can bypass the blood-brain barrier by binding to the transferrin receptor, which is expressed much more abundantly in capillaries than in arteries.
Maplight Therapeutics Inc. has synthesized G protein-coupled receptor GPR6 inverse agonists reported to be useful for the treatment of anxiety, bipolar disorder, eating disorders, depression, schizophrenia, Huntington’s disease, Parkinson’s disease and Alzheimer’s disease, among others.
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