Gene Therapy For Rheumatoid Arthritis
(Dr. Barrie Carter, Executive Vice President and Director)
Rheumatoid Arthritis is a disease characterized by chronic inflammation, thickened joint lining and destruction of cartilage. It is believed to be an autoimmune disease, although some current theories point to a possible viral or bacterial origin. The disease generally strikes young adults, and can lead to painful, deformed joints. At the annual meeting of the American Society of gene Therapy held on the weekend of June 4-5, Targeted Genetics Corporation presented an abstract entitled, "Treatment of Experimental Arthritis Using Recombinant AAV-TNFR:Fc Vector Gene Therapy," describing its adeno-associated virus (AAV) vector technology to deliver the genes encoding TNFR:Fc, to treat the inflammation of rheumatoid arthritis. TNFR:Fc stands for tumor necrosis factor receptor-immunoglobulin Fc, a protein antagonist of tumor necrosis factor-alpha, which is involved in the inflammatory response. The researchers used a gene delivery system consisting of the injection of a genetically modified virus. The virus is able to deliver a copy of the gene encoding the protein into the genetic material of the targeted cells. The rats experienced significantly less inflammatory symptoms in areas receiving only a single injection. The researchers noted that blood levels of the TNFR:Fc protein did not rise significantly, leading them to observe that the effect appeared local rather than systemic.
Dr. Barrie Carter, Executive Vice President and Director, Research and Development of Targeted Genetics stated, "We believe that the safety profile and long-term expression properties of our AAV vectors make them a logical approach to developing a gene-based delivery system for this important therapeutic protein. Local delivery of AAV-TNFR:Fc to arthritic joints may allow patients to be dosed every few months while maintaining therapeutic levels of the protein between treatments. We are moving forward with the preclinical development of the human version of this construct."
H. Stewart Parker, President and Chief Executive Officer of Targeted Genetics, commented, "These data demonstrate that gene delivery may have a number of applications beyond the treatment of cancer or single-gene defects."
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