Genescience Pharmaceuticals Co. Ltd. has identified sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of phenylketonuria (PKU).
Suzhou Spring-Sea Bio-Pharmaceuticals Co. Ltd. has described polypeptides acting as glucagon-like peptide 1 receptor (GLP-1R), glucagon receptor (GCGR) and gastric inhibitory polypeptide receptor (GIPR) agonists reported to be useful for the treatment of dyslipidemia, hepatic steatosis, metabolic syndrome, obesity and type 2 diabetes.
Neumora Therapeutics Inc. has reported preclinical data for NMRA-215, a brain-penetrant, oral NLRP3 inhibitor in development for obesity, suggesting potential utility as a monotherapy as well as in combination with a GLP-1 agonist.
Activating the glucagon-like peptide 1 receptor (GLP-1R) is the mechanism of action of most drugs currently used to treat obesity and type 2 diabetes. To enhance efficacy and reduce side effects, many groups have been developing double or triple agonists that, at the same time, also stimulate the receptors for glucagon (GCG) or glucose-dependent insulinotropic peptide (GIP).
Alpha-1 antitrypsin deficiency (AATD) is a monogenic disease caused by mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a serine protease inhibitor mainly produced by hepatocytes.
Genescience Pharmaceuticals Co. Ltd. has identified phenylalanine hydroxylase (PAH) (R408W mutant) stabilizers reported to be useful for the treatment of phenylketonuria (PKU).
A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.
When the levels of LDL cholesterol are elevated, there is increased risk for atherosclerotic cardiovascular disease. Lowering these levels contributes to decreasing the risk of major adverse cardiovascular events. EDIT-401 is a CRISPR-based LDL cholesterol-lowering gene-editing approach developed by Editas Medicine Inc.
Ascletis Pharma Inc. has selected ASC-35, a once-monthly, subcutaneously administered GLP-1 receptor (GLP-1R)/GIP receptor (GIPR) dual peptide agonist, as a clinical development candidate. Ascletis expects to submit an IND application to the FDA for ASC-35 for the treatment of obesity in the second quarter of next year.
While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.
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