Mirus Bio Corporation announced the grant of US Patent No.7,148,205 entitled "Intravascular Delivery of Non-Viral Nucleic Acid." The patent broadly covers administration of RNAi-inducing molecules via hydrodynamic intravascular injection.
A key bottleneck impeding the progress of the groundbreaking field of RNA interference (RNAi) has been the lack of effective delivery methods. This delivery breakthrough combined with RNAi creates a powerful discovery research tool for studying gene function in animal models, and in the long term might be used for certain human therapeutic tissues.
"Mirus Bio is increasingly being recognized for its world class expertise in nucleic acid chemistry and delivery," commented Russell Smestad, Mirus Bio's President. "Hydrodynamic injection has already been widely adopted in the RNAi research field as the most effective method for in vivo delivery to liver, where it is a unique tool for target identification and validation studies. In the future we anticipate that our proprietary Pathway IV ™ hydrodynamic protocol for delivery of nucleic acids to limb skeletal muscle will similarly be recognized as an enabling platform for human therapeutics, both for RNAi as well as DNA based products. We are actively pursuing strategic alliances and licensees to apply this technology as widely as possible."
Hydrodynamic intravascular injection is a method to deliver nucleic acids through the bloodstream to surrounding cells and tissues. Normally, standard injection of DNA or RNA into a vein or artery would result in the nucleic acids being retained within the blood vessel until degraded and filtered out of the body. However, researchers at Mirus Bio together with collaborators at the University of Wisconsin-Madison discovered that rapid injection of a large volume nucleic acid-saline solution combined with simultaneous mechanical or biological alteration of the permeability of the vessel wall enabled the DNA/RNA to migrate into the surrounding tissue cells. This enables regional delivery throughout an entire limb or other tissue rather than being localized to a single point of injection as happens with a needle and syringe.
RNA interference (RNAi) is a natural cellular process wherein short nucleic acids known as small interfering RNA (siRNA) regulate gene expression and protein production. In normal cells, DNA is copied to messenger RNA (mRNA) which directs the synthesis of protein. The RNAi gene silencing process involves the introduction of double-stranded RNA molecules into a cell, after which a multistep cellular process creates single-stranded siRNA molecules that interfere with the translation of mRNA into the protein it encodes. Blocking production of disease causing proteins in this manner represents a fundamentally new approach for innovative medicines. The significance of this biological pathway was highlighted in October when the two researchers credited with discovering this powerful biological phenomenon were jointly awarded "The Nobel Prize in Physiology or Medicine for 2006".
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 labelling products to researchers worldwide. In addition, the company is developing novel human therapeutics enabled by its proprietary Pathway IV™ delivery platform.