Since things have been pretty dormant here on this thread, here's some additional company information to perhaps stir some life (from the Avigen Investor Fact Sheet, Spring 1997):
Avigen, Inc. is a leader in the development of gene therapy products derived from adeno-associated-virus (AAV) for the treatment of inherited and acquired diseases. The company's proposed gene therapy products are designed for in vivo administration to achieve the production of therapeutic proteins within the body. Avigen is developing two broad-based proprietary gene technologies: AAV vectors and the Targeted Vector Integration (TVI) system. AAV vectors are being used to deliver genes for the treatment of brain, liver and prostate cancer, anemia, hemophilia, hyperlipidemia and metabolic storage diseases. The TVI system is a proprietary technology for achieving site-specific integration of DNA.
AAV Vectors. Avigen has derived its AAV vectors from AAV, a common non-pathogenic human virus, to take advantage of the natural efficiency with which viruses deliver genes to cells. The company produces AAV vectors by removing the virus's own genes and replacing them with genes for therapeutic products. AAV vectors combine desirable properties of viral and non-viral vectors and may offer several potential advantages over other gene therapy vectors. These advantages include efficient delivery of genes to both dividing and non-dividing target cells, absence of viral genes that may be responsible for causing an undesirable immune response, suitability for in vivo administration to patients, high levels of gene expression, and improved stability allowing AAV vectors to be manufactured, stored and handled like more traditional pharmaceutical products. Avigen's scientists have developed a proprietary manufacturing process that will simplify AAV vector manufacturing and purification and achieve increased yields of high purity.
TVI System. Avigen's TVI system utilizes components of AAV to integrate large segments of DNA at a specific location on human chromosome 19. Integration is essential for certain gene therapy applications where the genes must be passed on to the progeny of the cell. Avigen anticipates that gene therapy vectors that integrate at a specific site, such as the site of chromosome 19 where the non-pathogenic AAV normally integrates, will have an increased safety profile relative to vectors that integrate their genetic payloads randomly. Avigen is currently designing gene therapy vectors incorporating TVI technology. The company believes that these vectors will allow it to pursue more effective treatments for blood-cell related diseases including sickle-cell anemia, beta-thalassemia and human immunodeficiency virus (HIV) infection.
Product Development. Based on encouraging results in animal models, Avigen has initiated two preclinical development programs using AAV vectors for the treatment of brain tumors and hemophilia. Additionally, the company has research programs and collaborations intended to generate gene therapies for other cancers (liver, prostate), blood diseases (hemoglobin disorders), metabolic diseases (hyperlipidemia and metabolic storage diseases), and infectious disese (HIV). With the identification by genomics researchers of new disease-related genes, the company believes is AAV vectors and TVI system will provide additional significant opportunities for the development of new gene therapy products.
Recent Accomplishments:
April, 1997. Avigen receives U.S. patent on novel methods for the production of commercially significant amounts of AAV vectors. Avigen believes that this patent will give the Company a competitive advantage to overcome many of the limitations related to vector production which have hindered clinical applications in the gene therapy field.
November, 1996. Avigen publishes in PNAS a report of the sustained production of human erythropoietin (Epo) in mice following the single injection of AAV vectors containing the Epo gene. The company has now observed Epo expression in mice for over fifteen months.
November, 1996. Avigen and collaborators at Johns Hopkins School of Medicine presented research at the American Heart Association showing in experimental models the successful delivery and sustained expression of a gene associated with an inherited fatal heart disease called Pompe's disease. Characterized by an enlarged and weakened heart muscle in infants, Pompe's disease is caused by a genetic mutation that results in the defective production of a key enzyme in heart muscle.
November, 1996. Avigen reported in Gene Therapy the successful demonstration of gene therapy using AAV vectors to treat human brain tumors transplanted into mice.
May, 1996. Avigen completes successful $20 million initial public offering [2.75 million shares at $8.00 on 5/22/96] |
