Veritas Bio, LLC, a privately held biotechnology company is pleased to announce that it has received a Notice of Allowance from the USPTO for Patent Application No. 12/514,237, “In Vivo Delivery Of Double Stranded RNA To a Target Cell”.
Dr. Pachuk, the inventor said, “The allowed claims cover transfecting double-stranded RNA into skin or muscle cells under conditions wherein double-stranded RNA such as siRNA is delivered to the liver for the purpose of down regulating liver pathogens. This proprietary technology is applicable for the rapid development of RNA therapeutics for the treatment of infectious liver diseases including Hepatitis B, C and others.”
“Our technology solves one of the biggest hurdles for the development of RNAi and other nucleic acid-based drugs by providing a safe and effective delivery system for effector RNA molecules. This technology enables the utilization of the body’s innate delivery system to transport effector RNA molecules rather than relying on expensive synthetic, toxic, immunogenic and inefficient delivery systems, nanoparticles or viral particles for delivery.”
Humans, naturally extrude exovesicles (also called exosomes) from multiple cell types including muscle and skin cells. Exovesicles are natural nanoparticles the body produces that transport molecules from one cell to another and from one tissue or organ to another. Veritas Bio’s technology enables utilization of this delivery system to organs, tissues and cells.
This new technology enables local administration to the skin or muscle, of either the effector molecules or a DNA encoding the effector molecules. The molecules are then loaded by the transfected cell into exovesicles, which are extruded from the cell and distributed to other cells in the body, to deliver RNA to target cells is a natural, safe and effective method of systemic delivery of RNA, without invoking the innate and adaptive immune responses to the delivery system. The exosomes can be targeted to specific cell types and tissues through the use of ligands as described in the patent.
A corresponding application has also been filed in Europe. Veritas is in the process of filing continuing applications to cover various other indications including delivery to non-liver tissues including immune cells, various types of therapeutic nucleic acid molecules and compositions in which RNA containing exovesicles are manufactured in producer cells in culture.
The Allowed Patent Application and the continuing applications are available for licensing.