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News from AvigenAAV Vectors Employment Nasdaq - AVGN Corporate Partners  Table of Contents

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.


Our Mission

To be a leader in the field of Gene Therapy by developing simple practical approaches to treating major human diseases with our technologies.


Company Profile

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.


Contact Information

Telephone
510-748-7150
FAX
510-748-7155
 
Postal address
1201 Harbor Bay Pkwy #100
Alameda, CA 94502 USA
 
Electronic mail
CEO: monahan@avigen.com
CFO: paulson@avigen.com
VP R&D: kurtzman@avigen.com
VP Clinical Affairs & Operations: devlaminck@avigen.com
VP Business Development: maurer@avigen.com
 
Webmaster: monahan@avigen.com

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Last modified: October 29, 1997