Azor Biotek is developing RNA-targeting small molecule therapeutics for the safe and curative treatment of neurodegenerative and pulmonary diseases. Our flagship therapeutics are designed to target miR-29 with exquisite specificity and bioavailability dwarfing that of comparable oligonucleotides.
microRNA (miR)
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.
Live zebrafish screens
Early screening in zebrafish further selects for therapeutics with high bioavailability, and allow for in vivo quantification of concrete pathological hallmarks while providing approximations of the effective dose, toxicity and off-target effects.
GFP expression in dopaminergic neurons allows for absolute quantification of neurodegeneration. Fish are not an ideal model organism for modeling lung pathology, however, the ability to visualize the developing cardiovascular and mesenchyme systems in vivo makes them well suited to evaluate anti-fibrotic, anti-angiogenic and anti-metastatic properties.
Indicated Markets
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 prognoses. Other medical conditions indicated for miR-29 have a combined (though less readily addressable) market of >$300B.
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.
Funding
Funding for our pilot project has been provided in part by the National Research Council of Canada Industrial Research Assistance Program
Recognitions
2022 Invited Speaker — RNA Targeted Drug Discovery
2022 CEO Award Nomination — Business Worldwide
2022 Invited guest — Worldwide Business with Kathy Ireland
2022 Top 100 Lifescience Innovators — YoungStartup Ventures
2021 Sweet 16 Most Promising Canadian Biotech — GIIANT PHARMA
