The pharmaceutical and biotech industries are targeting various RNA classes to develop new drugs. MicroRNAs (miRNAs) participate in widespread regulation of cell function and contribute to a spectrum of human diseases, especially cancer, making miRNAs a drug-target class of particular interest. Due to delivery issues and poor bioavailability, oligonucleotides-based therapies have proven unsuccessful in widespread miRNA targeting. As such, pharmaceutical and biotech companies are pursuing miRNA-targeted small molecule therapies. However, to efficiently identify drug-like miRNA-targeting small molecules that therapeutically alter biological activity, new tools are in demand. To meet this demand, Nymirum is developing the first ever platform to rapidly, accurately, and cost-efficiently identify and optimize small molecules that therapeutically target cancer-causing microRNAs.
Public Health Relevance Statement: Project Narrative miRNAs represent the largest RNA drug-target class with significant implications in cancer. Nymirum is developing the first ever platform to rapidly, accurately, and cost-efficiently identify and optimize small molecules that therapeutically target cancer-causing microRNAs.
Project Terms: base; Binding; Biological; Biological Assay; Biological Availability; Biology; Biotechnology; Cancer Etiology; Cell physiology; Cells; Computer software; cost; cytotoxicity; Data; Data Collection; Data Set; Databases; Disease; drug discovery; Drug Targeting; Exhibits; Goals; graphical user interface; human disease; In Vitro; Individual; Industry; inhibitor/antagonist; innovation; interest; Isotope Labeling; Knowledge; Legal patent; malignant breast neoplasm; Malignant neoplasm of liver; Malignant Neoplasms; Mediating; Methods; MicroRNAs; Modeling; Monitor; Muscle; Mutation; NMR Spectroscopy; novel therapeutics; Oligonucleotides; Pharmaceutical Preparations; Pharmacologic Substance; Pharmacology; Phase; Phase II Clinical Trials; prevent; Primary carcinoma of the liver cells; programs; Regulation; Reporting; Resolution; RNA; RNA Splicing; small molecule; small molecule libraries; small molecule therapeutics; Spinal; Structure; success; Technology; Testing; therapeutic target; three dimensional structure; tool; Untranslated RNA; virtual