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The Novel GlycoPEGylated FGF21 Analog Pegozafermin Activates Human FGF Receptors and Improves Metabolic and Liver Outcomes in Diabetic Monkeys and Healthy Human Volunteers [Drug Discovery and Translational Medicine]

ASPET

In cell-based assays, 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.

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Generation and characterization of mirikizumab, a humanized monoclonal antibody targeting the p19 subunit of IL-23 [Drug Discovery and Translational Medicine]

ASPET

Mirikizumab effectively inhibits the interaction of IL-23 with its receptor, and potently blocks IL-23-induced IL-17 production in cell-based assays 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.

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Genetic variant identification: unveiling the power of STING-seq

Drug Target Review

Co-led by Dr Sanjana, the research study combines CRISPR gene editing and single-cell sequencing to identify causal genetic variants and enhance our understanding of the noncoding genome. Published in Science , the study offers new possibilities for identifying therapeutic targets in genetically influenced diseases.

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Exploring alternatives to animal testing in drug discovery

Drug Target Review

allowing researchers to use “certain alternatives to animal testing, including cell-based assays and computer models, to obtain an exemption from the FDA to investigate the safety and effectiveness of a drug.” Three-dimensional organoids have been a popular choice in testing drugs to treat cancer and disease.

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Accelerating Drug Discovery Through Repurposing

DrugBank

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.

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Inside The Altascientist Issue 41: Immunomodulation Assessments for Clinical Trials–Sophisticated Bioanalytical Approaches to Support Complex Modalities

Alta Sciences

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.

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How are mRNA vaccines changing drug discovery?

Tebu Bio: Drug Discovery

mRNA vaccines are developing very quickly with dozens of ongoing clinical trials against cancers or infectious diseases (e.g. Recombinant monoclonal antibody (mAB) based treatments have proven their efficacy. HIV or more recently SARS-Cov-2).

Vaccine 52