Targeted Genetics is focused on the development and commercialization of targeted molecular therapies using our proprietary gene delivery technology. We have industry-leading capabilities in the development and manufacture of Adeno-Associated Viral (AAV) based products that we are leveraging to build a diverse product development pipeline across multiple diseases, including inflammatory arthritis and other serious diseases. We have significant expertise in researching and developing gene delivery technologies, including expertise in quality assurance, quality control, general research, regulatory affairs and clinical affairs. We believe that gene-based therapeutics provide a means to treat diseases not fully addressed by current biologic and pharmaceutical drugs, and have the potential to address significant unmet medical needs in multiple disease areas. Our business strategy is to leverage our proprietary intellectual property through product commercialization efforts and collaborations, and ultimately, to create shareholder value by developing innovative and proprietary therapies in significant markets.
Our business strategy includes:
Advancing tgAAC94: A unique therapeutic approach to treating inflammatory arthritis
tgAAC94 utilizes our adeno-associated viral (AAV) vector platform to deliver a DNA sequence encoding an inhibitor of TNF-α, a potent proinflammatory cytokine that plays a major role in inflammatory arthritis. The product candidate is designed for local administration into inflamed joints for use in patients who, despite being treated with systemic TNF-α antagonists, have one or more joints that continue to be inflamed. The product candidate is also being developed for patients who may benefit from localized rather than systemic treatment of their disease. According to the American College of Rheumatology (ACR) and the Centers for Disease Control and Prevention (CDC), 2-3 million people in the United States are living with inflammatory arthritis, and the cost of treating arthritis and other rheumatic conditions was $86.2 billion in 1997, the most recent year for which these data are available. Although there have been important advances in the treatment of a variety of inflammatory diseases, 15 to 40 percent of patients suffering from rheumatoid arthritis only partially respond to the TNF-α inhibitor drugs. We believe that Targeted Genetics' approach to treating inflammatory arthritis has significant potential. Our targeted, localized product candidate in conjunction with systemic therapy may come to play an important role in helping patients better manage their disease. Advancing tgAAC94 through clinical development and toward the market is our primary objective.
Leveraging the safety and utility of our AAV platform to expand our product and partnership opportunities
We have development collaborations with leading companies that provide us with additional product development opportunities. These partnerships are the result of our industry leading capabilities in AAV development and manufacturing, and typically provide us with research and development funding as well as significant revenue sharing and development milestones. Our expertise and capabilities enable us to form strategic partnerships with leading pharmaceutical and biotechnology companies, non-profit organizations and academic institutions, from early stage proof-of-concept studies to extensive development and commercial collaborations. These collaborations have created product opportunities for us in areas with significant clinical and commercial potential, such as treatment and prevention of HIV/AIDS, congestive heart failure, LCA (eye disease), and Huntington's disease.
Establishing strategic relationships that provide revenue, R&D funding, potential royalties and novel product opportunities
Partnerships are a key component of both our development and financial strategies. Leveraging our unique leadership position in the development of AAV-based products to establish partnerships that provide us with near- and mid-term payments and R&D funding allows us to participate in novel product opportunities without detracting resources from our lead program in inflammatory arthritis. These partnerships also may potentially provide royalty-based revenue over the long term. Additionally, by combining our expertise in AAV-based product development with the leadership of our partners in specific therapeutic or technology areas, we create synergies that support the development of innovative and transformative therapies. |
