Cell Genesys receives patent for adeno-associated virus gene therapy
Cell Genesys, Inc. (Foster City, CA; 650-425-4542) announced that the company has received a notice of allowance from the United States Patent and Trademark Office The United States Patent and Trademark Office (PTO or USPTO) is an agency in the United States Department of Commerce that provides patent protection to inventors and businesses for their inventions, and trademark registration for product and intellectual property for a broad patent covering all gene therapy products that utilize adeno-associated virus adeno-associated virus
a replication-defective, single-stranded DNA virus classifed in the genus Dependovirus of the family Parvoviridae. They depend on help provided by coinfection with adenoviruses for their replication. Not known to cause disease. vectors to deliver genes as therapeutic agents. The allowed patent includes composition of matter claims which cover the essential component of any adeno-associated virus gene delivery system, specifically any recombinant adeno-associated virus vector products, free of contaminating wild-type adeno-associated virus, regardless of the method of production. In addition, Cell Genesys has an exclusive license to issued United States Patent 5,436,146 which covers methods of producing recombinant adeno-associated virus vectors as well as additional composition of matter claims.
"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 Howard Hughes Medical Institute, (HHMI), nonprofit medical research organization founded in 1953 by Howard Hughes and largly funded from proceeds of the 1984–85 sale of Hughes Aircraft. Headquartered in Chevy Chase, Md. 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 preclinical studies,
n.pl a term used to describe research done before a clinical study. May be laboratory or epidemiologic research. , Cell Genesys has demonstrated the potential utility of these adeno-associated virus gene therapy vectors to deliver therapeutic genes to treat cardiovascular disease Cardiovascular disease
Disease that affects the heart and blood vessels.
Mentioned in: Lipoproteins Test
cardiovascular disease , anemia, hemophilia and central nervous system disorders Nervous system disorders
A satisfactory classification of diseases of the nervous system should include not only the type of reaction (congenital malformation, infection, trauma, neoplasm, vascular diseases, and degenerative, metabolic, toxic, or deficiency such as Parkinson's disease Parkinson's disease or Parkinsonism, degenerative brain disorder first described by the English surgeon James Parkinson in 1817. When there is no known cause, the disease usually appears after age 40 and is referred to as Parkinson's disease. . Recently, Cell Genesys published findings in Nature Genetics demonstrating the successful in vivo in vivo /in vi·vo/ (ve´vo) [L.] within the living body.
Within a living organism.
in vivo adv. 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 blood clotting, process by which the blood coagulates to form solid masses, or clots. In minor injuries, small oval bodies called platelets, or thrombocytes, tend to collect and form plugs in blood vessel openings. 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 recombination, process of "shuffling" of genes by which new combinations can be generated. In recombination through sexual reproduction, the offspring's complete set of genes differs from that of either parent, being rather a combination of genes from both parents. of DNA DNA: see nucleic acid.
or deoxyribonucleic acid
One of two types of nucleic acid (the other is RNA); a complex organic compound found in all living cells and many viruses. It is the chemical substance of genes. 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 ex vivo /ex vi·vo/ (eks´ ve´vo) outside the living body; denoting removal of an organ (e.g., the kidney) for reparative surgery, after which it is returned to the original site. 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 Autoimmune Disorders Definition
Autoimmune disorders are conditions in which a person's immune system attacks the body's own cells, causing tissue destruction. and cancer, as well as the company's licensing program in gene activation technology.