Work at Jnana Therapeutics Inc. has led to the identification of sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, metabolic dysfunction-associated steatohepatitis (MASH), phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism spectrum disorders, among others.
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.
Regenxbio Inc. plans to file a BLA this year seeking accelerated approval for gene therapy candidate RGX-121 to treat young children with mucopolysaccharidosis type II, also known as Hunter syndrome, based on positive data from the phase I/II/III Campsiite trial, which not only hit the biomarker endpoint but also indicated potential systemic benefits.
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.
A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.
Sangamo Therapeutics Inc.’s gene therapy for Fabry disease, isaralgagene civaparvovec (ST-920), is continuing to show promising efficacy and safety in the phase I/II study, Staar, but further ahead, the company needs to either attract a partner or secure financing to move to a registrational trial.
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.