Skip to main content

Advertisement

Table 1 Human clinical trials of arthritis gene therapy

From: Gene therapy of the rheumatic diseases: 1998 to 2008

Transgene Vector Ex vivo/In vivo Phase PI, Institution or sponsor OBA protocol number Status Number of subjects in study
IL-1Ra Retrovirus Ex vivo I Christopher H Evans and Paul D Robbins, University of Pittsburgh (PA, USA) 9406-074 Closed 9
IL-1Ra Retrovirus Ex vivo I Peter Wehling, University of Düsseldorf (Germany) N/A Closed 2
HSV-tk Plasmid In vivo I Blake Roessler, University of Michigan (Ann Arbor, MI, USA) 9802-237 Closed 1
TNFR:Fc fusion protein (etanercept) AAV In vivo I Philip Mease, Targeted Genetics Corporation (Seattle, WA, USA) 0307-588 Closed 15
TGF-β1 Retrovirus Ex vivo I Chal-Won Ha, Kolon Life Science (Gwacheon, Korea) N/A Open 12
TGF-β1 Retrovirus Ex vivo I Michael Mont, TissueGene Inc. (Gaithersburg, MD, USA) 0307-594 Open 4
TNFR:Fc fusion protein (etanercept) AAV In vivo I/II Philip Mease, Targeted Genetics Corporation 0504-705 Enrolled Clinical hold lifted by FDA in December 2007 127
  1. All of these target rheumatoid arthritis, except for the TissueGene Inc. and Kolon Life Science trials that target osteoarthritis. The Targeted Genetics Corporation trial can also recruit subjects with psoriatic arthritis and ankylosing spondylitis. AAV, adeno-associated virus; FDA, US Food and Drug Administration; HSV-tk, herpes simplex virus thymidine kinase; IL-1Ra, interleukin-1 receptor antagonist; N/A, not applicable; OBA, Office of Biotechnology Activities; PI, principal investigator; TGF-β1, transforming growth factor-beta-1; TNFR, tumor necrosis factor receptor. Reprinted with permission [23].