TGEN Tecogen Inc

Targeted Genetics Presents Preclinical Data From Rheumatoid Arthritis Program at the 7th Annual Meeting of the American Society of Gene Therapy - Results Support Efficacy, Safety and Broad Utility of AAV Technology - MINNEAPOLIS and SEATTLE, June 3 /PRNewswire-FirstCall/ -- Targeted Genetics Corporation (NASDAQ:TGEN) will present results of preclinical studies supporting its rheumatoid arthritis program today in two sessions during the 7th Annual Meeting of the American Society of Gene Therapy in Minneapolis, MN. Targeted Genetics is currently conducting a Phase I clinical trial in patients with rheumatoid arthritis. The product candidate, tgAAC94, utilizes the Company's recombinant adeno-associated viral (rAAV) vector technology platform to deliver the DNA sequence encoding a potent inhibitor of tumor necrosis factor (TNF-alpha), known as TNFR:Fc, directly into affected joints of patients suffering from rheumatoid arthritis. Data presented today will highlight data from studies evaluating multiple routes of delivery of tgAAC94. Haim Burstein, Ph.D., senior director of product discovery at Targeted Genetics, will present in two different sessions throughout the day. The first presentation is during the workshop session #WS224 titled, "Musculo-Skeletal: Designing and Planning Clinical Trials for Musculo-Skeletal Diseases." Dr. Burstein's presentation will include an overview of Targeted Genetics' product candidate, tgAAC94, encouraging preclinical results, and current clinical program status. Preclinical results to date testing tgAAC94 have demonstrated suppression of arthritis, as measured by arthritis index scores that include a decrease in cartilage and bone destruction and inflammatory cell infiltration. No safety issues related to the product candidate have been observed in preclinical studies. Dr. Burstein also will present Abstract #99, titled "Long-Term Expression of Soluble TNFR:Fc Protein Following Multiple Administrations of AAV-TNFR:Fc Vector Pseudotyped with Capsids of Alternate Serotypes" in a poster session during the afternoon. In this study, different routes of administration, as well as alternate capsid serotypes of AAV vectors, were tested. Both intramuscular and intravenous administration resulted in sustained, high levels of secreted soluble TNFR:Fc protein in the systemic circulation for over a year. Following pulmonary delivery of tgAAV-ratTNFR:Fc vector, soluble TNFR:Fc protein was observed in serum. Maximum expression of TNFR:Fc protein in the circulation was achieved within six weeks after administration and gradually declined over a period of eight months. Repeat administration via pulmonary delivery resulted in renewed expression. Pulmonary administration elicited serum anti-AAV capsid neutralizing antibody responses, but these did not prevent lung transduction and efficient secretion of TNFR:Fc protein to the circulation following repeat administration. "Data presented today demonstrate the diverse assets of Targeted Genetics' AAV technology, providing both a localized and systemic delivery approach to treatment of rheumatoid arthritis," said Dr. Burstein. "Use of AAV to deliver the DNA sequence encoding TNFR:Fc directly into affected joints has resulted in suppression of arthritis in animal models as evidenced by decreased articular index score, inflammatory cell infiltration, pannus formation and cartilage and bone destruction. Presentation of additional preclinical data today suggest that various routes of administration used to deliver AAV vectors, along with different capsid serotypes of AAV, yield long-term expression of TNFR:Fc protein. We believe these data continue to support the broad potential of Targeted Genetics' AAV gene delivery technology to treat systemic TNF-a mediated autoimmune diseases such as rheumatoid arthritis." Targeted Genetics' Current Phase I Clinical Trial Targeted Genetics' Phase I clinical trial is a multi-center, randomized, double-blind, placebo-controlled, dose escalation study designed to assess safety of intra-articular delivery of tgAAC94. Other secondary parameters assessed are the ability of intra-articular administration of tgAAC94 to reduce pain and swelling in the injected joint and overall disease activity. The amount of local and circulating TNFR:Fc protein also will be measured. The trial is being conducted in the U.S. and Canada, and will ultimately enroll up to 32 patients. Participants will receive a single injection of tgAAC94 or placebo directly into an affected joint and will be followed for 24 weeks following injection. This clinical trial is based on prior preclinical data testing intraarticular administration of the product candidate directly into affected joints. Results demonstrated localized expression of the protein in targeted joints without significantly elevated levels of circulating TNFR:Fc protein. About Targeted Genetics Targeted Genetics Corporation develops gene-based products for preventing and treating acquired and inherited diseases. The Company has three clinical product development programs targeting cystic fibrosis, AIDS prophylaxis and rheumatoid arthritis. The Company also has a promising pipeline of product candidates focused on hemophilia and cancer and a broad platform of gene delivery technologies, as well as a promising body of technology for cellular therapy under development by its subsidiary company, CellExSys. For more information about the Company visit its website at http://www.targetedgenetics.com/. This release contains forward-looking statements regarding our regulatory filings, research programs, clinical trials, product development and potential related to tgAAC94 and other statements about our 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 our actual results include, but are not limited to, the timing, nature and results of our research and our clinical trials, our preclinical results with animals are not necessarily indicative of results that will be obtained in humans, our ability to obtain and maintain regulatory or institutional approvals, our ability to obtain, maintain and protect our intellectual property related to tgAAC94, and our ability to raise capital when needed, as well as other risk factors described in the section entitled "Factors Affecting Our Operating Results, Our Business and Our Stock Price" in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2004. You should not rely unduly on these forward-looking statements, which apply only as of the date of this release. We undertake no duty to publicly announce or report revisions to these statements as new information becomes available that may change our expectations. DATASOURCE: Targeted Genetics Corporation CONTACT: Courtney Self of Targeted Genetics Corporation, +1-206-521-7392 Web site: http://www.targetedgenetics.com/

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