Therapeutics for peripheral neuropathies, such as Charcot-Marie-Tooth (CMT) disease, are still quite limited and focus on managing symptoms. Augustine Therapeutics NV has presented an orally available histone deacetylase 6 (HDAC6) inhibitor, AGT-100216, for the treatment of CMT. AGT-100216 was designed to work through improving axonal regeneration and nerve functional restoration.
Caregen Co. Ltd. has described peptides reported to be useful for the treatment of cartilage injury, fracture, intervertebral disc herniation, degenerative intervertebral disc disorder, osteoarthritis, osteomalacia and muscular injury.
Researchers at Risen (Shanghai) Pharma Eng Co. Ltd. and Risen (Suzhou) Pharma Tech Co. Ltd. have divulged GTPase KRAS inhibitors reported to be useful for the treatment of cancer.
Scientists at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified NLRP3 inflammasome inhibitors reported to be useful for the treatment of cardiovascular disorders.
Sanofi SA has synthesized interleukin-17A (IL-17A)/interleukin-17 receptor A (IL-17RA) interaction inhibitors reported to be useful for the treatment of psoriasis, rheumatoid arthritis, radiographic axial spondyloarthritis (ankylosing spondylitis), multiple sclerosis, asthma, chronic obstructive pulmonary disease, atopic dermatitis and systemic lupus erythematosus, among others.
Praxis Precision Medicines Inc. has disclosed inhibitors of potassium channel subfamily T member 1 (KCNT1) and its mutants reported to be useful for the treatment of Brugada syndrome, heart failure, muscle disorders, myocardial infarction, neurological disorders, pain, pruritus and psychiatric disorders.
Cannabidiol (CBD) is a nonpsychoactive cannabinoid with potential therapeutic use in several diseases such as epilepsy or anxiety, but shows poor solubility, erratic absorption and structural polymorphisms that limit its bioavailability. Researchers from Artelo Biosciences Inc. presented the pharmacokinetic profile of ART12.11, a proprietary co-crystallization of CBD and co-former tetramethylpyrazine (TMP), formulated to achieve improved bioavailability and stability and to overcome polymorphism of CBD.
Cystic fibrosis (CF) is characterized by lack of hydration in the airways by impaired functioning of cystic fibrosis transmembrane conductance regulator (CFTR), leading to infection, inflammation and lung tissue damage. It is hypothesized that inhibiting the epithelial sodium channel (ENaC) in the airways in CF may enhance the mucociliary clearance (MCC) and provide clinical benefit, but numerous inhaled ENaC blockers have failed in clinical trials. Enterprise Therapeutics Ltd. is developing an inhaled ENaC blocker compound, ETD-001, for the treatment of CF.
Researchers from the National Cancer Institute presented data from a study that aimed to explore the role of the major nuclear exporter protein exportin 1 (XPO1) in pancreatic neuroendocrine tumor (pNET) subsistence and therapy resistance.