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Avigen is a publicly held company with its headquarters in Alameda, California. The company is listed on NASDAQ as AVGN. Avigen is a leader in the development of gene therapy products derived from adeno-associated virus ("AAV") for the treatment of inherited and acquired diseases. Our proposed gene therapy products are designed for in vivo administration to achieve the production of therapeutic proteins within the body. We are developing two broad-based proprietary gene delivery technologies: AAV vectors and the Targeted Vector Integration ("TVI") system. Avigen believes AAV vectors can be used to deliver genes for the treatment of brain, liver and prostate cancer, anemia, hemophilia, hyperlipidemia and metabolic storage diseases. We also believe that our TVI system will allow us to pursue more effective treatments for blood cell-related diseases, including sickle cell anemia, beta-thalassemia and human immunodeficiency virus ("HIV") infection.
To be a leader in the field of Gene Therapy by developing simple practical approaches to treating major human diseases with our technologies.
AAV Vectors. Avigen's gene therapy products are based on gene delivery systems
called vectors. AAV vectors are derived from AAV, a
common non-pathogenic human virus, and take advantage of the natural efficiency with which
viruses deliver genes to cells. To produce an AAV vector, the virus is modified by
removing the viral genes and replacing them with genes for therapeutic proteins. We
believe that 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,
in vivo administration to patients, higher levels of gene expression and improved
stability allowing AAV vectors to be manufactured, stored and handled like more
traditional pharmaceutical products. Avigen believes that its proprietary manufacturing
process will simplify manufacturing and purification and achieve increased yield of high
purity AAV vectors.
TVI System. Avigen's proprietary 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. We believe gene therapy vectors
that integrate at a specific site, such as the site on chromosome 19 where the
non-pathogenic AAV normally integrates, will have an increased safety profile relative to
vectors that integrate randomly. We also believe that the ability to integrate large
segments of DNA could lead to gene therapy applications involving the delivery of multiple
genes or requiring precise or controllable gene regulation.
Product Development. Based on encouraging results in animal models, Avigen has
initiated two preclinical development programs using AAV vectors for the treatment of
hemophilia and brain tumors. Additionally, we have a number of research programs and
collaborations intended to generate product development candidates for liver and prostate
cancer, hyperlipidemia, metabolic storage diseases, hemoglobin disorders and HIV
infection. We believe that the number of potential applications for gene therapy will
increase significantly as advances are made in the area of genomics. These advances are
enabling scientists to link diseases to specific gene defects. As more genes are
discovered, the need for improved gene delivery technologies is expected to increase. With
the identification of new disease-related genes, we believe that our AAV vectors and TVI
system will provide significant opportunities for new gene therapy products.
Corporate Partnering Opportunities. Avigen is actively seeking to develop long-term strategic collaborations with pharmaceutical companies that can provide funding for research and development activities for potential products of the sponsor. We also are looking for new applications to disease, and for new gene sequences to deliver. We and our collaborators have delivered over thirty different genes, so we can do custom development programs on applications that would fit the product lines and/or research interests of most pharmaceutical companies.
Finance History. Avigen was established as a private company in October 1992 with a focus on gene therapy. Avigen commenced an initial public offering in May 1996, raising $20 million on 2.5 million shares. So far, the company has raised a total of $32.9 million in equity financing through public and private sources from U.S. and Japanese investors. The company is listed on NASDAQ as AVGN.
Product Development
Programs
The company has selected its product
development programs based on experimental data that demonstrate the feasibility of gene
delivery to specific target cells. We believe that our technologies may be used with
several different genes, giving rise to multiple product and corporate partnering
opportunities, and that further advances in genomics, including the sequencing, mapping
and identification of genes linked to diseases, may offer other product opportunities. The
following table summarizes Avigen's current product development programs:-
Avigen AAV Vector-Based Gene Therapy Programs
Program | Indication | Target Cell | Status |
---|---|---|---|
Blood Diseases | Hemophilia | Muscle | Preclinical |
Anemia | Muscle | Research | |
Cancer | Brain Tumors | Tumor | Preclinical |
Liver Cancer | Tumor | Research | |
Prostate Cancer | Tumor | Research | |
Neural Diseases | Parkinsons Disease | Brain | Research |
Metabolic Diseases | Hyperlipidemia | Muscle | Research |
Storage Diseases | Muscle | Research |
Avigen TVI-Based Gene Therapy Programs
Program | Indication | Target Cell | Status |
---|---|---|---|
Blood Diseases | Anemia | BoneMarrow | Research |
Infectious Diseases | HIV | Bone Marrow | Research |
Management
NAME | POSITION |
---|---|
Philip J. Whitcome, Ph.D. | Chairman of the Board |
John Monahan, Ph.D. | President, Chief Executive Officer and Director |
Thomas J. Paulson | Vice President, Finance and Chief Financial Officer |
Gary J. Kurtzman, M.D. | Vice President, Research and Development |
Wanda deVlaminck | Vice President, Clinical and Regulatory Affairs |
Robert H. Maurer | Vice President Business Development |
Zola Horovitz, Ph.D. | Director |
Yuichi Iwaki, M.D., Ph.D. | Director |
Richard T. Pratt | Director |
John K.A. Prendergast, Ph.D. | Director |
Lindsay A. Rosenwald, M.D. | Director |
Leonard P. Shaykin | Director |
Scientific Advisory Board
The Company has established a Scientific
Advisory Board, consisting of experts in the field of medicine, genetics and
molecular biology, which reviews and evaluates the Company's research programs and advises
the Company with respect to technical matters in fields in which the Company is involved.
The members of the Scientific Advisory Board are prominent scholars in their field and, as
a result, may serve as consultants to a wide variety of companies.
Employment Opportunities
We provide exciting career
opportunities to creative, highly motivated individuals, and are an equal opportunity
employer. Submit resumes to: Human Resoures, Avigen, Inc., 1201 Harbor Bay Parkway, Suite
1000, Alameda, CA 94502.