Drug Discovery
Mirna’s drug discovery engine leverages cutting-edge RNA technologies developed at Ambion and Asuragen, and is positioned in a unique environment that enables the rapid identification and validation of therapeutic candidates. Mirna Therapeutics has access to the latest miRNA expression analysis tools and a series of established in vitro and in vivo disease model systems allowing Mirna to rigorously identify the key miRNAs that play direct roles in the development of diseases.
In many cases, Mirna scientists - while at Ambion - were the researchers who developed the first commercial products for studying miRNAs. Experience and expertise with these tools has enabled a comprehensive search for therapeutically relevant miRNAs. For instance, to identify miRNAs with therapeutic value in oncology applications, Mirna scientists evaluated samples from cancer patients to identify those miRNAs that are most consistently and significantly expressed at different levels between a diseased tissue and a normal tissue. In parallel, Mirna scientists have used numerous functional assays, including proliferation, viability, cell cycle progression, apoptotic response, angiogenesis and oncogenic transformation, to identify miRNAs that inhibit cancer cells. The preferred miRNAs for therapeutic development are the ones with good efficacy in both cell culture and animal models.
To date, Mirna Therapeutics has identified a series of miRNAs with therapeutic activity for many human diseases including cancer. The therapeutic application of these has been called out in numerous patent applications, claiming a total of more than 300 miRNAs. These patent applications are among the earliest in the field, putting Mirna Therapeutics into a dominant IP position. Mirna’s main focus is the development of miRNA therapeutics for solid cancers. Mirna has established proof of concept for the systemic delivery of therapeutic miRNAs in pre-clinical mouse models of cancer and is moving its lead candidates toward the clinic.
