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Neurology/psychiatric

HDAC inhibitor improves Charcot-Marie-Tooth disease in mice

July 8, 2024
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.
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Other news to note for July 5, 2024

July 5, 2024
Additional early-stage research and drug discovery news in brief, from: Pneumagen.
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Musculoskeletal

Caregen discovers new peptides with cartilage regenerative effects

July 5, 2024
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.
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Cancer

New GTPase KRAS inhibitors disclosed in Risen Pharma patent

July 5, 2024
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.
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Cardiovascular

Roche scientists describe new NLRP3 inflammasome inhibitors

July 5, 2024
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.
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Skin exam with dermatoscope
Dermatologic

Sanofi divulges new IL-17A/IL-17RA interaction inhibitors

July 5, 2024
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.
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Cardiovascular

Praxis Precision Medicines patents new KCNT1 inhibitors

July 5, 2024
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.
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Cannabidiol
Neurology/psychiatric

Pharmacokinetic evaluation of novel cannabidiol formulation presented

July 5, 2024
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.
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Lungs
Respiratory

New studies support ETD-001 and other ENaC blockers for cystic fibrosis

July 5, 2024
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.
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Illustration of cancer tumor on pancreas
Cancer

XPO1 as a therapeutic target in pancreatic neuroendocrine tumors

July 5, 2024
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.
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