Company Overview
Targeted Genetics Corporation is a biotechnology company committed to the development and commercialization of innovative targeted molecular therapies for the prevention and treatment of inflammatory arthritis and other acquired and inherited diseases with significant unmet medical need. For more than a decade the Company has been at the forefront of innovation in the development of multiple gene delivery systems. As a result, Targeted Genetics has significant expertise in the preclinical development, clinical and regulatory affairs and manufacturing of its innovative gene-based product candidates. The Company has amassed a robust portfolio of intellectual property covering scientific and commercial aspects of its technologies and product development programs. Targeted Genetics believes that gene-based therapies will become a proven class of therapeutics that will find important use as a new weapon in the war against disease.
We use our considerable knowledge and capabilities in the development and manufacturing of gene delivery technologies to advance a diverse product development pipeline. Our clinical development programs target potential therapies for inflammatory arthritis and a vaccine for HIV/AIDS, as well as preclinical programs for congestive heart failure, Huntington's disease and hyperlipidemia. In addition to these programs, Targeted Genetics has generated important proof-of-concept data that support the utility of multiple gene delivery systems and the potential of several additional product candidates. These delivery systems and product candidates provide a broad spectrum of potential future development programs, as well as partnering or out-licensing opportunities.
Targeted Genetics is a Seattle, Washington based company with approximately 100 employees. The Company has been public since May 1994 and its stock trades on the NASDAQ Capital Market under the ticker symbol TGEN.
Clinical Development Programs
Inflammatory Arthritis
tgAAC94 is currently being evaluated in a Phase I clinical trial for the treatment of inflammatory arthritis including rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis. tgAAC94 uses a rAAV vector to deliver a DNA sequence encoding a potent inhibitor of tumor necrosis factor-a (TNF-a) directly to affected joints in patients with RA. TNF-a inhibition has been validated as an approach to treating inflammatory autoimmune diseases through the successful commercialization and wide use of several anti-TNF-a therapies. It is estimated that the market for anti-TNF-a therapies will exceed $7 billion by 2011. Unlike approved anti-TNF-a therapies, tgAAC94 is designed for local administration to affected joints and may have the potential to address the unmet medical needs of patients who do not fully respond to systemic therapy, estimated to be between 25 and 40 percent of the patients on anti-TNF therapies, as well as those patients who have just a few affected joints and do not want to be, or are not advised to be, on a systemic therapy.
HIV/AIDS Vaccine
tgAAC09, a vaccine candidate designed to prevent the development of AIDS in people uninfected with HIV, is in a Phase I clinical trial. This rAAV-based vaccine candidate is designed to deliver selected HIV genes in order to stimulate an immune response against the virus that causes AIDS. In preclinical studies, rAAV vaccines have elicited robust and durable B-cell and T-cell responses in animal models.
Preclinical Development Programs
Congestive Heart Failure
Targeted Genetics Corporation and Celladon Corporation are working to develop adeno associated virus (AAV)-based gene therapies for the treatment of congestive heart failure. We believe the combination of Targeted Genetics’ expertise in the development, manufacture and clinical evaluation of AAV-based therapies with Celladon’s portfolio of genes with potential in the treatment of congestive heart failure holds significant promise.
Hyperlipidemia
Targeted Genetics is also exploring the field of cardiovascular disease by applying its AAV vector technology to the treatment of hyperlipidemia, the elevation of lipids in the bloodstream.
Huntington's Disease
Through its partnership with Sirna Therapeutics, Inc., Targeted Genetics is working to develop novel therapies for the treatment of Huntington’s Disease (HD), an incurable neurodegenerative disorder. The collaboration combines Targeted Genetics’ adeno-associated virus (AAV) delivery platform with Sirna’s HD program and expertise in RNA silencing technologies.
Additional Product Development Opportunities
Proof-of-concept data have been generated in a variety of other disease indications, including hemophilia and cancer. Although Targeted Genetics is not currently pursuing development of these programs, they provide partnering or out-licensing opportunities. These programs also have the potential to become internal core development programs in the future, providing Targeted Genetics with diverse opportunities for growth.
Leadership in AAV
Targeted Genetics’ current clinical development programs are based on the Company’s AAV technology. The Company has the longest and broadest experience in the development, manufacture and clinical study of AAV vectors. The Company was the first to bring AAV vectors into clinical trials and more than 200 trial participants have received Targeted Genetics’ AAV-based product candidates. AAV is a naturally occurring virus that is not associated with any disease in humans. The vector efficiently delivers genetic information to numerous cell types and can be engineered to carry a variety of DNA sequences. AAV vectors are highly stable, persist in cells for extended periods of time, and do not integrate into host-cell DNA.
This combination of attributes makes AAV vectors particularly useful in the treatment of chronic diseases such as arthritis and a number of genetic diseases. AAV-based delivery of genes encoding therapeutic proteins may present an attractive alternative or complement to systemic therapy. Additionally, AAV vectors can be used for the development of vaccines that induce robust and durable B-cell and T-cell responses.
Targeted Genetics is the leading manufacturer of AAV vectors. The Company has developed a proprietary manufacturing process that utilizes the same technology used to produce monoclonal antibodies and therapeutic proteins, and has patents and patent applications related to the preparation, manufacture and purification of its AAV vectors. Current manufacturing is being conducted in 100-liter bioreactors, and the process can be scaled to meet commercial needs. Targeted Genetics’ expertise in biologics manufacturing has enabled the Company to leverage its existing manufacturing infrastructure, including its Regulatory Affairs and Quality expertise, to provide contract manufacturing services to other companies at times when its manufacturing capacity is not fully utilized by its own manufacturing needs. This approach enables the Company to derive near-term revenue from its state-of-the-art manufacturing facilities as it continues development of its promising product pipeline.
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