Cell Genesys receives patent for adeno-associated virus gene therapy
"We believe that this patent provides Cell Genesys with a dominant proprietary position for adeno-associated virus gene therapy products," said Stephen A. Sherwin, MD, chairman and chief executive officer of Cell Genesys. "Adeno-associated virus vector technology and intellectual property are among the key assets obtained through our recent acquisition of Somatix Therapy Corporation, which will enable Cell Genesys to aggressively pursue the commercialization of gene therapy."
"The development of helper-free recombinant adeno-associated virus vectors is a significant step towards the commercialization of this important gene delivery system," said Thomas E. Shenk, PhD, Investigator, Howard Hughes Medical Institute and Professor, Princeton University (Princeton, NJ) and co-inventor with Richard J. Samulski, PhD and Long-Sheng Chang, PhD on the patent. "Research conducted by Cell Genesys as well as others working in the field supports the commercial value of this invention in the develop-ment of adeno-associated virus gene therapy products for human therapeutics." Research scientists led by Dr. Shenk have pioneered the early development of recombinant adeno-associated virus vectors. Cell Genesys is the exclusive licensee of this patent which is based on the work of Dr. Shenk and his group at Princeton University.
In preclinical studies, Cell Genesys has demonstrated the potential utility of these adeno-associated virus gene therapy vectors to deliver therapeutic genes to treat cardiovascular disease, anemia, hemophilia and central nervous system disorders such as Parkinson's disease. Recently, Cell Genesys published findings in Nature Genetics demonstrating the successful in vivo gene delivery of the factor IX gene utilizing the company's proprietary gene delivery adeno-associated virus vectors to transfer the factor IX gene into targeted cells of the liver in normal mice. Through just a single injection, therapeutic levels of the factor IX protein were produced for at least nine months showing long-term production of a medically-relevant protein. The factor IX gene encodes the factor IX protein essential for blood clotting and is deficient in certain hemophiliacs.
The novel composition claims included in the notice of allowance cover all recombinant adeno-associated virus gene therapy products which are not contamin-ated with wild-type adeno-associated virus virus, or virus potentially capable of replicating, regardless of method of production. The crux of the invention and the key to the commercial utility of this vector system lies in the ability to prevent recombination of DNA elements used in manufacturing, thereby avoiding the generation of undesirable replication-competent adeno-associated virus virus. This patent, along with the company's issued patent and clinical manufacturing and trial expertise, enables Cell Genesys to aggressively develop adeno-associated virus gene therapy products. Cell Genesys plans to license its proprietary adeno-associated virus vector technology for certain applications outside its own development programs.
Typically, recombinant adeno-associated virus vectors are produced in cells to which specific "helper DNA" (adeno-associated virus rep and cap genes) and the gene of interest (adeno-associated virus vector carrying therapeutic gene) have been added. The allowed patent pertains to a novel approach in which potential recombination of adeno-associated virus vector and helper DNA is eliminated and thereby avoids potential generation of replication-competent adeno-associated virus virus containing the rep and cap genes. Adeno-associated virus vectors are derived from a non-pathogenic human virus.
Cell Genesys is focused on the development and commercialization of ex vivo and in vivo gene therapies to treat major, life-threatening diseases and disorders such as cancer and AIDS. The company's AIDS gene therapy is in Phase II human clinical testing and is being developed through a worldwide collaboration with Hoechst Marion Roussel, Inc. (Kansas City, MO). In the cancer area, where Cell Genesys retains worldwide rights, five human clinical trials are expected to be initiated by the end of the third quarter. Cell Genesys' assets outside gene therapy include its Abgenix, Inc. subsidiary, which is developing antibody therapies for transplantation-associated medical conditions, inflammation, autoimmune disorders and cancer, as well as the company's licensing program in gene activation technology.
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|Publication:||BIOTECH Patent News|
|Date:||Sep 1, 1997|
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