Info

Mirus Bio Corporation is a biopharmaceutical company focused on discovering, developing and commercializing innovative nucleic acid based technologies and products. Under development are RNAi gene silencing therapies based on Mirus' proprietary Dynamic PolyConjugatesÔ delivery platform and gene therapies utilizing its Pathway IVÔ delivery protocol. Commercially, Mirus markets a range of innovative transfection and labeling research reagents worldwide. Dynamic PolyConjugates ("DPC") is a targeted systemic siRNA delivery platform. Our proprietary polymer based formulation chemistry efficiently targets gene silencing complexes to specific cells. As the DPCs attach to and enter the target cells, they respond to the environmental cues provided by the cell to disassemble and release the active siRNA molecule. This unique chemistry mimics the natural viral targeting and disassembly process. This technology is currently being optimized for liver and cancer tissues, and is actively being evaluated by select partners for research and clinical indications. Pathway IV is a clinically practical procedure based upon intravenous ("IV") delivery of plasmid DNA ("pDNA") to targeted limb muscle. With blood flow temporarily occluded by a tourniquet, a DNA solution is rapidly injected into a suitable vein. This elevates the pressure within the occlusion zone, making the blood vessel wall more permeable and allowing the pDNA to migrate into the adjoining muscle cells. Blood flow is then restored to normal within a few minutes, with only mild transient affects to the vasculature. This quick, robust procedure is being used as an enabling platform that will provide first-in-class therapeutic treatments for muscular dystrophy, peripheral ischemia and other diseases. Patients with the most common muscular dystrophies, Duchenne and Becker, suffer from a genetic defect in which dystrophin protein is either poor or inadequate in their muscle cells. It affects roughly 1 of every 3500 male births. MyoDys® will treat this disease by inserting a gene encoding active dystrophin into affected muscles to delay or prevent loss of muscle function. Peripheral ischemia is a condition in which reduced blood supply to limbs causes claudication (cramping), chronic pain, and potentially loss of the limb. It is common in diabetics and the elderly, where up to 20% of those over age 70 experience intermittent claudication. Mirus' treatment is based upon delivery of a gene encoding an angiogenic factor to the affected limb, which induces revascularization of the limb. In the future, the company envisions using muscle as the host site for gene therapies that induce secretion of therapeutic proteins to treat a range of diseases, including anemia, arthritis, ALS, and cancer immunotherapeutics. Mirus R&D uniquely integrates nucleic acid delivery and chemistry, a core competency which is unmatched in the industry. This serves as the foundation for both its novel research reagents and emerging therapeutics. The company maintains advanced research programs focused on synthetic siRNA delivery, and on gene delivery to liver and other target tissues. Access to Mirus' technologies is available through strategic alliances as well as licensing in nonstrategic fields. Mirus is well positioned to be a partner of choice due to its consistent record of profitability; driven by the sales growth of its research reagents.