Gene therapy of the rheumatic diseases: 1998 to 2008

Table 1 Human clinical trials of arthritis gene therapy

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

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].

ISSN: 1478-6362