Bayer AG has synthesized new furoindazole derivatives acting as G protein-coupled receptor 84 (GPR84) antagonists reported to be useful for the treatment of inflammation, metabolic disorders, autoimmune and lung diseases, among others.
Sex differences at the cellular level could explain why men respond less well to glioblastoma (GBM) treatments, according to a study led by the Washington University School of Medicine in St. Louis (WUSTL). The researchers found that male and female GBM tumor cells had different metabolic needs. GBM cells from male surgical samples absorbed more glutamine and had different nutritional preferences for amino acids.
Researchers at Northwestern University and COUR Pharmaceutical Development Company Inc. have shown that biodegradable carboxylated poly(D,L-lactic-co-glycolic acid) (PLGA) nanoparticles containing encapsulated Ag (tolerance-inducing microparticles [TIMPs] or COUR nanoparticles [CNPs]) are able to prevent and treat type 1 diabetes (T1D) induced by transfer of monospecific diabetogenic CD4 and CD8 transgenic T cells to NOD.scid mice.
Proof of concept has been achieved for a therapy for argininosuccinic aciduria consisting of human argininosuccinic lyase (ASL) mRNA encapsulated in lipid nanoparticles (ASL-LNPs).
Quince Therapeutics Inc. (formerly Novosteo Inc.) has presented data on NOV-004, a construct that delivers abaloparatide therapeutic agent into the fracture site, designed for the treatment of osteogenesis imperfecta.
Cerebral creatine (Cr) deficiency syndromes are caused by AGAT, GAMT or SLC6A8 deficiencies that may cause severe neurodevelopmental delay and intellectual disability.
Communication between adipose tissue and the brain increases the risk of cognitive impairment in patients with insulin resistance through extracellular vesicles (EVs) containing microRNAs (miRNAs). Neurons could be damaged when these nucleotides reach the hippocampus guided by membrane proteins in prediabetic overweight people.
Argininosuccinic aciduria (ASA) is a disorder caused by argininosuccinate lyase (ASL) deficiency and is the second most common inherited urea cycle defect.
The FDA has granted orphan drug designation to Iecure Inc.'s lead product candidate GTP-506, an investigational product for the treatment of ornithine transcarbamylase (OTC) deficiency.