Jnana Therapeutics Inc. has identified 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, phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism spectrum disorders, among others.
Synlogic Inc. jolted Wall Street with news that the firm is scrapping for futility Synpheny-3, its pivotal study with labafenogene marselecobac (SYNB-1934) for phenylketonuria, and will evaluate strategic options. Shares of the Cambridge, Mass.-based firm (NASDAQ:SYBX) fell 48.7%, or $1.68, to end Feb. 9 at $1.77. Synlogic will cease operations and reduce its workforce by more than 90%, retaining only certain employees to help with the wind-down.
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.
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