Voltage-gated sodium channels, particularly Nav1.7, are highly expressed in peripheral sensory neurons and are crucial for pain signal conduction and signal transmission in the spinal dorsal horn. Because of their role in triggering pain, these channels are considered critical targets for analgesics. Systemic inhibition of Nav1.7 has been shown to abolish pain perception.
The serine/threonine kinase glycogen synthase kinase-3β (GSK-3β) plays a multifunctional role through its involvement in multiple signaling pathways. Because of its relevant role in Alzheimer’s disease (AD) pathogenesis, regulating GSK-3β activity has been proposed as a potential approach to target AD-related pathology.
Suzhou Raymon Pharmaceuticals Co. Ltd. has identified nitric oxide (NO) donor-containing compounds reported to be useful for the treatment of glaucoma, age-related macular degeneration, diabetic retinopathy, cataract, uveitis, keratitis and ocular hypertension.
Mair Therapeutics BV has established a scientific collaboration with Radboud University to accelerate the discovery of small-molecule agonists of TMEM175, a lysosomal ion channel genetically linked to Parkinson’s disease.
U.S. Department of Health and Human Services (HHS) has discovered dopamine D2 receptor antagonists potentially useful for the treatment of Tourette disease, bipolar disorder, tardive dyskinesia, Huntington’s disease, schizophrenia, depression, postoperative nausea and vomiting and gastroesophageal reflux disease.
About 10% of amyotrophic lateral sclerosis (ALS) cases result from inherited genetic mutations, with about 20% of them attributed to mutations in the gene encoding the ubiquitous cytoplasmic copper/zinc superoxide dismutase 1 (SOD1).
Insilico Medicine Cayman Topco has obtained IND approval from the FDA for ISM-8969 for the treatment of Parkinson’s disease, enabling initiation of a phase I trial in healthy volunteers.
Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant skeletal muscle disorder with a prevalence of approximately 1 in 8,000. The disorder is driven by aberrant expression of double homeodomain protein 4 (DUX4) within the D4Z4 macrosatellite array. Currently, effective treatments for FSHD are lacking. Strategies aimed at reducing DUX4 expression could hold promise as potential therapeutic approaches for FSHD.
Chengdu Kanghong Pharmaceutical Co. Ltd. has disclosed leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of cancer, type 1 diabetes, glaucoma, inflammatory bowel disease, multiple sclerosis, neurodegeneration, psoriasis and rheumatoid arthritis, among others.
Nippon Zoki Pharmaceutical Co. Ltd. has discovered alkyne derivatives acting as GABA-A receptor subunit α1β2γ2 positive allosteric modulators. As such, they are believed to be useful for the treatment of sleep disorders.
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