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In cell-basedassays, pegozafermin had a similar receptor engagement profile as native FGF21, with approximately 8-fold higher potency at FGF receptor 1 (FGFR1). These findings provide evidence that pegozafermin is a promising candidate molecule for the treatment of patients with NASH or SHTG.
Mirikizumab effectively inhibits the interaction of IL-23 with its receptor, and potently blocks IL-23-induced IL-17 production in cell-basedassays while preserving the function of IL-12. In both local and systemic in vivo mouse models, mirikizumab blocked IL-23-induced keratin mRNA or IL-17 production, respectively.
What was the scientific breakthrough in the treatment of sickle cell anaemia, and how did combining GWAS with CRISPR lead to the identification of causal variants and innovative therapies? Most people carrying two copies of the sickle mutation in the adult haemoglobin gene will get sickle-cell anaemia. This is a great question.
This approach capitalizes on prior investments in R&D, mitigates risk by leveraging established safety and pharmacokinetic profiles, and accelerates the delivery of treatments to patients. For example, researchers utilized gene expression data to determine the anti-ulcer drug cimetidine as a possible treatment for colorectal cancer.
allowing researchers to use “certain alternatives to animal testing, including cell-basedassays and computer models, to obtain an exemption from the FDA to investigate the safety and effectiveness of a drug.”
As the complexity of these therapeutics increases, so must the sophistication of the bioanalytical assays designed to either quantify them or measure their impact on the patient. Selecting the right assay depends on the specific immunomodulator and the clinical trial’s endpoints.
ASLAN003 is a highly selective and potent inhibitor of human DHODH (IC 50 = 35 nM) and has been shown to be more than 30 times more potent at inhibiting the DHODH enzyme in cell free and cell-basedassays than the first generation inhibitor teriflunomide. .
SINGAPORE, Oct.
Recombinant monoclonal antibody (mAB) basedtreatments have proven their efficacy. mRNA vaccines are developing very quickly with dozens of ongoing clinical trials against cancers or infectious diseases (e.g. HIV or more recently SARS-Cov-2).
For example, we might examine the structure-activity relationship in a cell-basedassay for consistency with the structure-activity relationship that we’ve observed in the enzyme inhibition assay. We have to make assumptions in these situations and we also need to check that these assumptions are reasonable.
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