Therapeutics Programs : Overview
Non-Small Cell Lung Cancer | Metastic Prostate Cancer | Acute Myeloid Leukemia
Mirna Therapeutics has identified a series of miRNAs with therapeutic activity for various human diseases including cancer. Our main efforts support the development of miRNA therapeutics for non-small cell lung carcinoma, metastatic prostate cancer, and acute myeloid leukemia. The selection of therapeutic leads is based on their efficacy and biology in the diseased tissue. To this end, Mirna Therapeutics has taken a combinatorial approach, using expression analysis tools and functional assays to ensure the identification of miRNAs that play a direct role in the development of the disease (see figure below).
For expression analysis, Mirna scientists have 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. For the miRNA function studies, Mirna scientists have used a number of different cell and animal models to identify miRNAs that affect cell proliferation, viability, cell cycle progression, apoptotic response, angiogenesis, transformation, and other cancer-related cell processes. Those miRNAs that are mis-regulated in human cancers and also affect cancer-related cell processes have been evaluated for therapeutic potential in a number of cell and animal models of cancer. As noted in the table below, six therapeutic miRNAs have exhibited an ability to induce a response in at least two of the three cancers that we have tested, suggesting that these miRNAs are key tumor suppressors with broad therapeutic potential.
Using animal and cell models of cancer, Mirna scientists have validated the concept of “miRNA Replacement Therapy” which involves introducing synthetic miRNAs into diseased tissues with the goal of restoring normal proliferation, apoptosis, cell cycle, and other pathways that have been affected by down-regulation of one or more miRNAs. In many cases, reactivation of these miRNA-regulated pathways leads to a therapeutic response.
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