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Table 2 Human clinical trials of arthritis gene therapy

From: Arthritis gene therapy's first death

Transgene Vector, ex vivo/in vivo Phase Principal investigator, institution/sponsor OBA Protocol Number Status n
IL-1 receptor antagonist Retrovirus, ex vivo I Evans and Robbins, University of Pittsburgh, USA 9406-074 Closed 9
IL-1 receptor antagonist Retrovirus, ex vivo I Wehling, University of Düsseldorf, Germany Not applicable Closed 2
HSV-tka Plasmid, in vivo I Roessler, University of Michigan, USA 9802-237 Closed 1
TNFR:Fc fusion protein (etanercept) AAV, in vivo I Mease, Targeted Genetics Corp., USA 0307-588 Closed 15
TGFβ1 Retrovirus, ex vivo I Ha, Kolon Life Sciences, Korea Not applicable Open 12
TGFβ1 Retrovirus, ex vivo I Mont, TissueGene Inc., USA 0307-594 Open 4
TNFR:Fc fusion protein (etanercept) AAV, in vivo I/II Mease, Targeted Genetics Corp., USA 0504-705 Enrolled. Clinical hold lifted by FDA in December 2007 127
  1. All of these trials target rheumatoid arthritis except for the TissueGene and Kolon trials, which target osteoarthritis. The Targeted Genetics Corp. trial can also recruit subjects with psoriatic arthritis and ankylosing spondylitis. A phase I study injecting NF-κB decoy oligonucleotides is underway at the University of Osaka in Japan (principal investigator: Tomita). This study is not included because it is not strictly gene therapy. Also omitted for the same reason are two trials using TNF antisense RNA [14]. aWhen expressed in conjunction with ganciclivir administration, herpes simplex virus thymidine kinase (HSV-tk) kills synovial cells and produces a synovectomy. AAV, adeno-associated virus; FDA, Food and Drug Administration; n, number of subjects in study; OBA, Office of Biotechnology Activities; TGFβ1, transforming growth factor beta 1; TNFR:Fc, tumor necrosis factor receptor:Fc domain of immunoglobulin fusion protein.