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.

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.