Targeted Genetics Corp (SEATTLE, Washington) announced that dosing has been completed for the phase I/II clinical trial of tgAAC94 administered directly to affected joints of subjects with inflammatory arthritis to reduce inflammation locally in the joints and to avoid the potential for systemic side effects. The ongoing trial will assess the safety and potential efficacy of different doses of the agent. Subjects enrolled in the study will continue to be followed and monitored.

Since the trial began in October 2005, a total of 127 subjects have been enrolled. In July 2007, the study was placed on clinical hold when a patient experienced a serious adverse event and subsequently died. An independent and in-depth review of all available product and clinical data was conducted to determine whether tgAAC94 contributed in any way to the patient's death. Evidence found and presented at the September and December 2007 meetings of the National Institutes of Health recombinant DNA advisory committee supported the position that tgAAC94 did not contribute to nor cause the patient's death. The committee determined that the patient died of an invasive fungal infection and, in November 2007, the US FDA removed the hold on the study.

Targeted Genetics reported interim data on the agent at the American College of Rheumatology (ACR) meeting in 2007. The data indicate that tgAAC94 is well-tolerated at the highest dose tested and demonstrate that a higher percentage of subjects who received the agent showed improvement in function and pain compared with results from the placebo injected group.

Targeted plans to report additional phase I/II results during 2008 at the American Society of Gene Therapy, the European League Against Rheumatism (EULAR) and ACR in May, June, and November, respectively.

Additional clinical trial protocols are being developed to evaluate efficacy and duration of response, as well as to further assess safety in patients with inflammatory arthritis who have ≤1 inflamed joints and who are not candidates for systemic therapy or who do not fully respond to systemic anti-TNF protein therapy.

tgAAC94 uses Targeted's recombinant Adeno-Associated Virus (rAAV) vector technology to deliver a DNA sequence that encodes a soluble form of the TNF-α receptor (TNFR: Fc). Soluble TNFR:Fc inhibits the immune stimulating activity of TNF-α. Direct injection of tgAAC94 into affected joints leads to the localized production of secreted TNFR:Fc within joint cells, reducing the activity of TNF-α within the joint and, potentially, leading to a decrease in the signs and symptoms of inflammatory disease and to inhibition of joint destruction.