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.
It has been previously demonstrated that noise exposure leads to activation of the mitogen-activated protein kinase (MAPK) pathway, and that inhibition of this pathway protects from hearing loss. Tizaterkib (formerly AZD-0364) is a novel, highly selective, orally bioavailable ERK1/2 inhibitor that is currently in early clinical development for the treatment of cancer. In recent work, investigators from Creighton University aimed to assess the potential of tizaterkib for the treatment of noise-induced hearing loss (NIHL).
Bleeding of unknown cause (BUC) is a diagnosis of exclusion, and it is common for these patients to have congenital platelet function disorders. Whole-exome sequencing may help reach a more accurate diagnosis in these cases.
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.
Stroke is the main neurologic cause of global morbidity and mortality with a global prevalence of more than 100 million last year. Under neuropathological conditions such as excitotoxicity associated with stroke, the pro-glycolytic enzyme 6-phosphofructo-2-kinase/fructose-2,6-bisphosphatase-3 (PFKFB3) enhances glucose consumption which leads to redox stress and apoptotic neuronal death.
At the ongoing WORLDSymposium meeting in San Diego, Gain Therapeutics Inc. presented preclinical data on the company’s GCase enhancer GT-02287 as a potential approach to rescue motor function in Parkinson’s disease (PD).
4DMT Molecular Therapeutics Inc. is looking ahead to phase III as positive data continue to roll out for gene therapy candidate 4D-150 in wet age-related macular degeneration (AMD), with the results from the phase II portion of the phase I/II Prism trial showing the single-shot treatment significantly reduced the need for chronic anti-VEGF injections. Presented over the weekend at the Angiogenesis, Exudation, and Degeneration 2024 Conference, results from the 24-week dose-expansion cohort, which comprised wet AMD patients with severe disease and high treatment burdens, showed patients receiving the high dose (3E10 vg/eye) had a 90% reduction in annualized anti-VEGF injection rates.
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