New research shows base and prime editing can correct some forms of phenylketonuria (PKU) in mice and human cell lines, raising the prospect that this gene-editing approach could allow children born with the inherited metabolic disorder to have a treatment that would avoid the need for dietary restrictions and medication.
Disc Medicine Inc.’s positive phase II data from an ongoing, open-label trial called Beacon with oral bitopertin in erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) whetted investor appetite for the results of the other mid-stage Disc experiment known as Aurora with the compound, due early next year.
Cerevance Inc. has described N-(4-aminocyclohexyl)pyrimidine-4-carboxamide derivatives acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors. As such, they are reported to be useful for the treatment of aging, diabetic nephropathy, glaucoma, inflammatory disorders, metabolic syndrome, obesity and rheumatoid arthritis.
Research led by Duke University and the German Center for Diabetes Research shows the glucose-dependent insulinotropic polypeptide (GIP) receptor plays an essential role in the action of the type 2 diabetes drug Mounjaro (tirzepatide; Eli Lilly and Co.) in human pancreatic islets.
A nutritional supplement to reduce the effects of aging might not be a pill of eternal youth, but it could reduce many of the problems of getting old while maintaining good health. The first step to achieve this is included in a study led by scientists from Columbia University. They have set their sights on the amino acid taurine.
Novartis AG has identified diaminocyclopentylpyridine derivatives acting as proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitors and thus reported to be useful for the treatment of hypercholesterolemia, hyperlipidemia, hypertriglyceridemia, sitosterolemia, vascular inflammation, atherosclerosis, peripheral vascular and coronary heart disease, among others.
An Agios Pharmaceuticals Inc. patent describes branched-chain-amino-acid aminotransferase, mitochondrial (BCAT2) inhibitors reported to be useful for the treatment of isovaleric acidemia, maple syrup urine disease, methylmalonic acidemia and propionic acidemia.
Research at Umecrine AB has led to the identification of a 3α-substituted 3β-hydroxy 17-oximated androstane compound acting as a GABA-A receptor antagonist.
By interfering with mitochondrial plasticity, researchers have succeeded in attenuating brain metastases of HER2-expressing breast tumors. The authors wrote that their findings “highlight targeting mitochondrial dynamics is a viable therapeutic opportunity to limit both brain tumors and metastasis.”
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