Targeted Genetics Announces Publication of Inflammatory Arthritis Study in Molecular Therapy

Data Demonstrate Complete and Long-Term Suppression of Recurrent Joint Inflammation in a Preclinical Model of Arthritis

SEATTLE, WA, USA | Jan 18, 2007 |
Targeted Genetics Corporation (NASDAQ: TGEN) today announced publication of positive results from a preclinical study of intramuscular administration of a pseudotyped AAV2/1-TNFR:Fc vector, in the February issue of Molecular Therapy. The study, led by Dr. Haim Burstein, Senior Director, Product Discovery for Targeted Genetics, establishes a proof of concept for administration of AAV-TNFR:Fc vector to the muscle for the purpose of achieving long-term, sustained and therapeutically relevant levels of TNFR:Fc protein to treat chronic systemic inflammatory joint disease. The article is titled "Long-term Suppression of Experimental Arthritis Following Intramuscular Administration of a Pseudotyped AAV2/1-TNFR:Fc Vector." Molecular Therapy is the official publication of the American Society of Gene Therapy.

"We are very encouraged by the important findings of this study that the delivery of therapeutic proteins using gene therapy has the potential to improve the efficacy, duration, convenience, and cost-effectiveness of chronic disease treatment, by replacing frequent and often invasive administrations of recombinant proteins with infrequent delivery of corresponding therapeutic genes," said H. Stewart Parker, President and CEO of Targeted Genetics. "These results provide support for further investigation of intra-muscular gene therapeutic drug candidates in inflammatory arthritis and broaden the potential of our inflammatory arthritis program."

In this study, a rat model was employed to compare the serum levels and duration of TNFR:Fc protein expression following intramuscular administration of pseudotyped AAV-TNFR:Fc vectors based on serotypes 1,2, and 5. The data demonstrated that all three vectors led to the sustained expression of serum TNFR:Fc protein for at least 1 year. However, Serum TNFR:Fc protein levels in rats administered intramucularly with the serotype 1-based vector were 10- and 100-fold higher compared with animals treated with the serotype 2 and 5-based vectors, respectively. Additionally, a single intramuscular administration of AAV2/1-TNFR:Fc vector to rats inflicted with recurrent ("flare-up") arthritis resulted in complete and long-term suppression of recurrent joint inflammation for at least 150 days. Furthermore, serum TNFR:Fc levels were sustained, and correlated with vector dose. The study is available at:

About Tumor Necrosis Factor Alpha

Tumor necrosis factor alpha (TNF-alpha) is a major proinflammatory cytokine that is overproduce in rheumatoid arthritis joints by activated moncytes, macrophages, and T cells, and promotes inflammatory responses that are important in the pathogenesis of rheumatoid arthritis (RA). In RA, TNF-alpha high concentrations of TNF-alpha in synovial fluid are associated with the erosion of cartilage and bone. TNFR:Fc fusion proteins have been developed for the therapeutic neutralization of TNF-alpha and are effective in treating inflammatory joint disease include RA, juvenile RA, psoriatic arthritis, and ankylosing spondilytis. However, effective control of these chronic inflammatory joint diseases using protein therapeutics raises at least one difficult challenge: the need for frequent repeat dosing as effective levels of the therapeutic protein cannot be maintained for extended periods.

About Targeted Genetics

Targeted Genetics Corporation is a biotechnology company committed to the development of innovative targeted molecular therapies for the prevention and treatment of acquired and inherited diseases with significant unmet medical need. Targeted Genetics' proprietary Adeno-Associated Virus (AAV) technology platform allows it to deliver gene coding proteins to increase gene function, as well as RNAi to decrease or silence gene function. Targeted Genetics' product development efforts target inflammatory arthritis, AIDS prophylaxis, congestive heart failure and Huntington's disease. To learn more about Targeted Genetics, visit Targeted Genetics' website at

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:

This release contains forward-looking statements regarding the results from this pre-clinical study, the efficacy of tgAAC94 with respect to the treatment of chronic systemic inflammatory joint disease and Targeted Genetics' ability to commercialize tgAAC94 and other statements about Targeted Genetics' plans, objectives, intentions and expectations. These statements, involve current expectations, forecasts of future events and other statements that are not historical facts. Inaccurate assumptions and known and unknown risks and uncertainties can affect the accuracy of forward-looking statements. Factors that could affect Targeted Genetics' actual results include, but are not limited to, Targeted Genetics' ability to obtain, maintain and protect its intellectual property, Targeted Genetics' ability to raise capital when needed, Targeted Genetics' ability to recruit and enroll suitable trial participants, the timing, nature and results of research and clinical trials, potential development of alternative technologies or more effective processes by competitors, and, Targeted Genetics' ability to obtain and maintain regulatory or institutional approvals, as well as other risk factors described in "Part I, Item 1A. Risk Factors" in Targeted Genetics' most recent annual report on Form 10-K or "Part II, Item 1A. Risk Factors" in Targeted Genetics' most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission. You should not rely unduly on these forward-looking statements, which apply only as of the date of this release. Targeted Genetics undertakes no duty to publicly announce or report revisions to these statements as new information becomes available that may change its expectations.

Investor and Media Contact:
Stacie D. Byars
Director, Communications
Targeted Genetics Corporation
(206) 521-7392

SOURCE: Targeted Genetics Corporation

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