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.

We focus on disorders with unmet treatment needs, with our pilot study designing Parkinson's disease therapeutics. Currently, only symptomatic relief is possible, with no effective treatments available to prevent, slow or halt disease progression. As such, novel combinational mechanisms of pharmacological action are desperately needed. Our pilot  miRNA targets modify entire networks of related therapeutics, including PTBP2 and the majority of drug targets currently in clinical trials —simultaneously and at innately determined ratios.