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.”
Sialidosis is a lysosomal storage disease caused by mutations in the NEU1 gene, which encodes sialidase neuraminidase 1. These mutations lead to enzyme deficiency and subsequently accumulation of oligosaccharides and sialylated glycopeptides in tissues and body fluids, which in turn lead to cell and organ dysfunction. There are no approved therapies. Three different AAV9 vectors encoding NEU1 were developed and tested by UMass Chan Medical School researchers in the preclinical setting in mice.
NGGT (Suzhou) Biotechnology Co. Ltd. has presented preclinical data on an AAV vector approach that expresses human PAH, rAAV8-PAH, also known as NGGT-002. NGGT-002 has liver tropism and it was codon-optimized for expressing PAH in the liver.
Neuronal ceroid lipofuscinosis, commonly known as Batten disease, is an inherited pediatric neurodegenerative lysosomal storage disease caused by mutations in the CLN5 gene. The disease is incurable and there is an urgent medical need for novel therapies. A murine model of Batten disease was developed to study a novel AAV vector that expresses CLN5, AAV9-CLN5. In the study by University College London investigators, the gene therapy, driven by the synapsin promoter, was intracerebroventricularly administered into neonatal Cln5-knockout mice.
Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the GLA gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage.
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