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.
Researchers from the University of Pittsburgh presented preclinical data for novel messenger RNA (mRNA) therapy consisting of human very long chain acyl-CoA dehydrogenase (hVLCAD) mRNA; the product is being developed as a potential treatment of VLCAD deficiency.
The FDA has cleared Immusoft Corp.'s IND for ISP-001 (iduronicrin genleukocel-T) for the treatment of mucopolysaccharidosis type I (MPS I), allowing initiation of a phase I trial.
Glucagon-like peptide 1 (GLP-1) signaling to intraepithelial lymphocyte (IEL) cells in the gut, where most GLP-1 is made, was important for inflammation in the gut itself, but less so for systemic inflammation.
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