Targeted Genetics' Preclinical Results Demonstrate Complete Suppression of Rheumatoid Arthritis During Study
Targeted Genetics Corporation

MINNEAPOLIS, and SEATTLE, June 7 /PRNewswire-FirstCall/ -- Targeted Genetics Corporation (Nasdaq: TGEN) presented preclinical results on Saturday supporting utility of its product candidate to treat rheumatoid arthritis. Studies to evaluate the long-term effect of TNFR:Fc expression on disease resulted in a complete suppression of rheumatoid arthritis over a three-month observation period in animal models. These data were presented on Saturday at the 7th Annual American Society of Gene Therapy Meeting 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.

Abstract #880, titled "Suppression of Inflammation in a Rat Model of Arthritis Following Intramuscular Administration of AAV-TNFR:Fc Vector Pseudotyped with AAV Type 1 Capsid," was presented in a poster session on Saturday afternoon by Haim Burstein, Ph.D., senior director of product discovery at Targeted Genetics. In this study, rats with experimental arthritis were injected intramuscularly with AAV-TNFR:Fc vectors with AAV1 serotype capsids. The data demonstrated efficient, long-term and sustained secretion of soluble TNFR:Fc protein to the systemic circulation. More importantly, when recurring flares of inflammation were introduced again into joints of these rats, disease was suppressed.

"Today's presentations represent Targeted Genetics' growing body of data supporting development of its product candidate to treat rheumatoid arthritis," said Dr. Burstein. "We continue to demonstrate in preclinical studies, safety and efficacy of AAV-based treatment of inflammatory arthritis. In addition, we continue to observe positive preclinical results favoring use of our AAV technology to treat rheumatoid arthritis over sustained periods of time. The evidence of extensive suppression of disease in a preclinical setting suggests the potential utility of AAV vectors for long-term systemic delivery of secreted soluble TNFR:Fc protein."

A concurrent poster (Abstract #881) also was presented on Saturday discussing Targeted Genetics' rheumatoid arthritis program. The poster presentation, titled "Preclinical Safety Assessment of Intra-Articular Administration of rAAV-humanTNFR:Fc in Rats in Support of a Phase I Arthritis Trial," discussed preclinical safety of our product candidate currently in a Phase I clinical trial. Lewis rats received either vehicle control, rAAV-ratTNFR:Fc, or one of three different doses of rAAV-humanTNFR:Fc via intra-articular injection. Clinical observations were performed on a daily and weekly basis, along with observations at sacrifice. The results demonstrate that the product candidate was well tolerated and gene expression was confirmed. The highest dose tested did not result in toxicity. These data, in addition to other safety and efficacy data, support clinical evaluation of tgAAC94 currently underway.

"As we advance our tgAAC94 product development program, Targeted Genetics continues to further understand the potential of our AAV technology and its role in the treatment of inflammatory diseases," said H. Stewart Parker, president and chief executive officer of Targeted Genetics. "With several AAV-based product candidates currently in clinical development, we are leveraging AAV assets, such as long-term expression, safety and stability profile, and diverse product applicability, with the goal of product commercialization. As the first company to bring AAV-based products into the clinic, we continue to maintain our leadership role in AAV product development."

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 www.targetedgenetics.com.

This release contains forward-looking statements regarding our 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.

SOURCE: Targeted Genetics Corporation

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