AAV delivery of gene encoding ENBREL shows promise in reducing inflammation in preclinical studies
Seattle, WA-June 5, 2000-Targeted Genetics Corporation (Nasdaq:TGEN) presented data from its gene therapy program for rheumatoid arthritis (RA) this weekend at the American Society of Gene Therapy Third Annual Meeting in Denver, Colorado. The Company is utilizing its adeno-associated virus (AAV) vector technology to deliver the DNA sequence encoding tumor necrosis factor receptor-immunoglobulin Fc fusion protein (TNFR:Fc) as a treatment for RA and other inflammatory diseases. Recombinant TNFR:Fc, ENBREL (etanercept), currently marketed for the treatment of several forms of RA, was developed by Immunex Corporation. Targeted Genetics obtained rights to the gene therapy applications of numerous genes and gene delivery technologies when it was spun out of Immunex in 1992.
Dr. Haim Burstein, Senior Scientist in the Department of Research at Targeted Genetics, presented the data in an abstract titled "Treatment of Experimental Arthritis Using Recombinant AAV-TNFR:Fc Vector Gene Therapy." The studies were conducted in collaboration with Dr. Sharon M. Wahl at the National Institute of Dental and Craniofacial Research, an institute of the National Institutes of Health. In these studies, recombinant AAV vectors containing the TNFR:Fc fusion genes were evaluated in an experimental rat model of arthritis. A single intra-articular (joint) administration of AAV-rTNFR:Fc into both rear ankle joints of arthritic rats resulted in a significant reduction of ankle and hind paw swelling as measured by arthritis index scores. A single intramuscular administration produced a similar effect. Bioactive rat TNFR:Fc protein was readily detectable in the serum of treated rats 33 days after intramuscular administration of AAV-rTNFR:Fc compared to control animals.
Injection of AAV-rTNFR:Fc into a single joint led to a reduction in hind paw swelling in the c
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