Insmed Inc. has synthesized cathepsin C (dipeptidyl peptidase I) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, asthma, cancer, chronic rhinosinusitis, heart failure, inflammatory bowel disease, psoriasis and thrombosis, among others.
Duchenne muscular dystrophy is the most frequent inherited muscle disease and no cure is available. The condition is currently treated with corticosteroids, which can cause substantial side effects.
Inhibiting emopamil binding protein (EBP), an enzyme involved in cholesterol biosynthesis, promotes the formation of oligodendrocytes, a potential approach for the management of multiple sclerosis (MS); Genentech Inc. has presented data regarding their oral and brain-penetrant EBP inhibitor GNE-3406.
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.
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