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Mirus Announces Breakthrough Intravenous Gene Delivery Method
FOR IMMEDIATE RELEASE: June 3, 2004
MADISON, WI, USA — Mirus Bio Corporation today formally announced the development of Pathway IV ™ gene delivery, a novel method to efficiently deliver therapeutic genes to muscle via the blood stream. This technique represents an important advance in the gene therapy field that could lead to novel therapies for such diseases as muscular dystrophy, peripheral vascular ischemia, arthritis, anemia, and cancer. Dr. Jon A. Wolff, Mirus co-founder and Chief Scientific Officer, presented the breakthrough progress at the annual meeting of the American Society of Gene Therapy (ASGT). Complete data will be reported in Molecular Therapy1, the official journal of the ASGT.
The technique is elegant in its simplicity and clinically practical. With blood flow in an arm or leg temporarily occluded by a tourniquet or blood pressure cuff, a plasmid DNA (pDNA) solution is rapidly injected intravenously. This elevates the pressure within the occlusion zone, transiently 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 no adverse affects to the vasculature.
“Earlier methods of gene delivery focused on intramuscular injection of plasmid DNA or the injection of viruses or gold particles that ferry genes of interest into target cells,” commented Dr. Wolff. “These methods suffer from limitations related to the level of gene expression achievable, duration of expression, and/or ability to administer repeat doses. With Pathway IV ™ we have been able to overcome these hurdles and demonstrate high level, long term gene expression with either single or multiple doses – a truly robust platform for treating chronic diseases. Moreover, we’ve also shown that this protocol is effective for delivering siRNA molecules, another type of nucleic acid that allows one to suppress unwanted gene expression in a target tissue.”
The company’s lead product utilizing this platform technology is a gene therapy for muscular dystrophy. Patients with the most common muscular dystrophies, Duchenne and Becker, suffer from a genetic defect in which dystrophin protein is either absent or inadequate in their muscle cells. It affects roughly 1 of every 3500 male births. The overall goal of the clinical development program is to treat this disease by inserting a gene encoding dystrophin into affected muscles to delay or prevent loss of muscle function and enhance patients’ quality of life. Based on this new method, Mirus and its strategic partner, Transgene S.A. (Strasbourg, France) intend to carry out a Phase I/II human clinical study. Selected aspects of the companies’ muscular dystrophy research have been supported directly or indirectly by the Muscular Dystrophy Association and the Association Francaise contre les Myopathies.
The research team responsible for this outstanding advance in delivery technology was led by Dr. Jim Hagstrom and Ms. Julia Hegge, together with their colleagues at Mirus and collaborators at the University of Wisconsin – Madison. This team demonstrated the robustness and versatility of the technique in a range of disease models and species, including the local expression of dystrophin in muscle and the systemic production of erythropoietin to treat anemia.
About Mirus:
Mirus Bio Corporation is a leader in the emerging fields of gene therapy and RNA interference, based upon its expertise in nucleic acid chemistry and delivery. The company currently markets state-of-the-art DNA and siRNA transfection and labeling products to researchers worldwide. In addition, the company is developing novel human therapeutics enabled by its proprietary Pathway IV™ delivery platform.
1 Hagstrom, J. et al. 2004. A Facile Non-Viral Method for Delivering Genes and siRNAs to Skeletal Muscle of Mammalian limbs. Molecular Therapy, In Press
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