At this week’s WORLDSymposium meeting, researchers from M6P Therapeutics Inc. reported on the preclinical efficacy of M-021, a novel enzyme replacement therapy (ERT) that co-expresses recombinant GAA with a bicistronic vector encoding N-acetylglucosamine-1-phosphotransferase (PTase; S1-S3).
Researchers from JCR Pharmaceuticals Co. Ltd. have presented new data for JR-171, a novel enzyme replacement therapy currently in early clinical development for the treatment of mucopolysaccharidosis type I (MPS I), also known as Hurler syndrome.
Tetra Pharm Technologies ApS has announced it is advancing its cannabinoid CB1 receptor antagonist, TPT-0701, into preclinical testing for appetite suppression.
Agonists of the glucagon-like peptide-1 receptor (GLP-1RAs) have emerged as effective treatments for obesity, but have a negative impact on lean mass during weight loss.
Diabetic retinopathy and diabetic kidney disease are frequent microvascular complications of diabetes, both related to exacerbated vascular permeability coming from microvascular barrier malfunctioning.
Graviton Bioscience Corp. has announced a strategic investment from Sanofi SA. Under the agreement, Sanofi receives a right of first negotiation to license compounds across various indications, including immunological and metabolic syndrome indications.
Vivet Therapeutics SAS has been awarded financing of €4.9 million (US$5.3 million) from the French government to advance the development of a gene therapy for the treatment of the neurodegenerative disease cerebrotendinous xanthomatosis (CTX).
Kupffer cells are macrophages located in the liver that play a key role in liver pathology, concretely in metabolic dysfunction-associated steatotic liver disease (MASLD; formerly nonalcoholic fatty liver disease or NAFLD), but the mechanisms behind this are poorly understood.
Researchers from Maze Therapeutics Inc. presented the discovery and preclinical characterization of MZ-101 as a potential candidate for the treatment of Pompe disease and other glycogen storage disorders.
Kissei Pharmaceutical Co. Ltd. has described 3,4-dihydroquinolin-2(1H)-one compounds acting as thyroid-stimulating hormone (thyrotropin) receptor (TSHR) antagonists reported to be useful for the treatment of hyperthyroidism, Graves’ disease and thyroid-associated ophthalmopathy.