RNA GUIDed Drug Design

miR are inhibitory post-transcriptional regulators. Pathological miR misexpression is often inversely correlated with pathological miR-target-transcript misexpression — as such many miR have been indicated as innately combinational therapeutic drug targets, regulating networks of related misexpressed transcripts. 

6 companies have brought miR-targeting biologics to clinical trial, including a miR-29 oligonucleotide mimic (Remlarsen). The intrinsically poor bioavailability of oligonucleotides has hampered further clinical development.  In contrast, Azor Biotek's miR-29-targeted small molecules are selected for optimized bioavailability and specificity.

We are developing our miR-29 targeting therapeutics for Parkinson's disease, pulmonary fibrosis (a COVID sequella), and lung cancer.

If a curative therapeutic is developed for Parkinson's it would see widespread adoption, completely disrupting the $7B market and improve patient outcomes of >5% of senior citizens globally. Similarly, a more effective pulmonary fibrosis therapeutic would capture that $5B market, and expand it in proportion with improved prognosis. Other medical conditions indicated for miR-29 have a combined (though less readily addressable) market of >$500B. 

Other miR species have been indicated as targets for a multitude of common conditions, and our platform is not limited to miR — the total potential market for RNA-targeting therapeutics designed with our technology is inconceivably vast.