Targeted Genetics continues to believe that the optimal delivery system for individual gene-based products will be dictated by the characteristics of the gene to be delivered, the type of cell to which the gene must be delivered and the duration of expression needed to elicit the desired therapeutic effect. Targeted Genetics is committed to maintaining and building its position in multiple delivery systems, in order to retain the flexibility needed to develop the safest and most efficacious products possible.
Adeno-Associated Virus (AAV) Vectors
AAV is a naturally occurring virus that is not associated with any disease in humans. AAV-based vectors efficiently deliver genetic information to numerous cell types and can be engineered to carry a variety of DNA sequences. AAV vector particles are stable, the vector genome persist in cells for extended periods of time, and do not integrate into chromosomal 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.
Although AAV vectors were originally developed for use in the treatment of diseases resulting from gene mutations, Targeted Genetics has leveraged its AAV technology platform into additional indications with significant market potential. AAV-based delivery of genes encoding therapeutic proteins may present an attractive alternative or complement to systemic protein therapy. For example, our program in inflammatory arthritis exemplifies how the delivery of DNA sequences using AAV vectors may have broad utility in developing the next generation of a variety of approved protein therapies. We believe that our targeted, localized gene-therapy solution has significant market potential and may come to play an important role in helping patients better manage their disease. AAV vectors can also be used for the development of vaccines that induce robust and durable B-cell and T-cell responses. Our HIV/AIDS vaccine program highlights the potential of AAV vectors as a novel approach to vaccine development. The great need for development of vaccines to treat diseases in pandemic proportions worldwide underscores our committment to develop novel vaccine technologies.
Targeted Genetics has extensive 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 250 trial participants have received Targeted Genetics’ AAV-based product candidates. Clinical trials in inflammatory arthritis and for an HIV/AIDS prophylaxis vaccine are ongoing, and additional preclinical data are being generated in the areas of congestive heart failure, Huntington’s disease and hyperlipidemia.
Manufacturing
The manufacturer of AAV vectors is one of Targeted Genetics' core strengths. We have 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. 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 assurance 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.
Additional Technology Assets
Targeted Genetics has amassed a significant body of technology, intellectual property and practical expertise that set the Company apart from its competitors. In addition to its platform in AAV vectors, Targeted Genetics has access to additional gene delivery technologies:
Viral technologies include:
- Adenovirus-AAV hybrid vectors that enable more rapid, small scale vector production. This system may have particular utility in evaluating multiple genes or multiple gene constructs for target validation and drug development initiatives
- Adenovirus vectors for use in the treatment of cancer and other disease
Non-viral technologies include:
- DC Cholesterol (DCC), a non-viral delivery system designed for local administration directly to tumors or to enclosed cavities within the body. DCC-based product candidates have been evaluated in Phase II trials of head and neck cancer and in Phase I trials in ovarian cancer and has demonstrated effective gene transfer to cancer cells. Product candidates using the DCC delivery system have been well tolerated, with a good safety profile
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