Veritas Bio, LLC, a privately held biotechnology company is pleased to announce that the European Patent Office (EPO) has granted a patent to Veritas for European Patent Application No. 07 873 598.2 1401. The patent relates to the “In vivo Delivery of Nucleic Acids.”
Dr. Pachuk, the inventor, said, “The allowed claims cover transfecting nucleic acids into skin or muscle cells under conditions wherein nucleic acids and expression vectors encoding nucleic acids, such as, DNA siRNA, mRNA, oligonucleotides and other nucleic acid molecules are delivered to the liver. The delivery of the nucleic acid can be for treating any disease or condition of the liver. These include down regulating endogenous liver genes associated with a disease condition and the genes of liver pathogens and the promoters of these genes. The allowed claims also cover the delivery of DNA, expression vectors, mRNA and vectors encoding mRNA for gene therapy. This proprietary technology is applicable for the rapid development of RNA therapeutics for the treatment of infectious liver diseases and other liver diseases.”
“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 and other nucleic acid 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 them to target cells is a natural, safe and effective method of systemic delivery, 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 allowed in the US. 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 and the continuing applications are available for licensing.
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