miRNA are attractive drug targets that in turn target whole networks of related transcripts, providing innate combinational therapy. 13 miRNA-acting biologics were brought to clinical trial in 2019 alone. Such biologic therapeutics have significant limitations and challenges to clinical use that are avoided with small molecule therapeutics. Azor Biotek designs small molecules to amend miRNA misexpression using proprietary RNA compatible virtual screening and lead optimization tools.
Designed compounds are first screened for transcriptional modification efficacy in live zebrafish before evaluation in advanced reverse translation models of disease. Live screening selects for therapeutics with high bioavailability while providing approximations of the effective dose, toxicity and off-target effects.
Our pilot Parkinson's disease miRNA targets modify entire networks of related therapeutics, including PTBP2 and the vast majority of drug targets currently in clinical trials —simultaneously and at innately determined ratios. Compounds targeting this miRNA target has been brought to clinical trial as pulmonary fibrosis therapeutics — topical as a COVID sequella.
Funding for our pilot project has been provided in part by the National Research Council of Canada Industrial Research Assistance Program (NRC IRAP)
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