Description
tgAAC94 utilizes our adeno-associated viral (AAV) vector platform to deliver a DNA sequence encoding an inhibitor of TNF-α, a potent proinflammatory cytokine that plays a major role in inflammatory arthritis. The product candidate is designed for local administration into inflamed joints for use in patients who, despite being treated with systemic TNF-α antagonists, have one or more joints that continue to be inflamed. The product candidate is also being developed for patients who may benefit from localized rather than systemic treatment of their disease.
In animal models of arthritis, local administration of tgAAC94 resulted in a profound suppression of arthritis, as evidenced by a reduction in bone and cartilage destruction, inflammatory cells and proinflammatory cytokines. This includes a reduction in circulating levels of TNF- α. These data were published in Molecular Therapy in 2002.
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