A Pharmacological and Toxicological Evaluation of the Gene Transfer Vectors sc-rAAV2.5IL-1Ra (Rat) and scr-AAV2.5IL-1Ra (Human) in Rats

National Center For Advancing Translational Sciences

Investigators

Abstract

Osteoarthritis is a disease of the joints in which the cartilage that normally covers the ends of the bones, reducing friction between them, wears thin. The result is pain and loss of motion or flexibility, which can become debilitating. Osteoarthritis is the most common form of arthritis, affecting more than 25 million Americans. Women are at greater risk for the disease than men. No cure is known for osteoarthritis, but treatments exist for the pain. These scientists are developing a gene therapy for osteoarthritis, which will be injected into the affected joint. The resulting gene product works by blocking an inflammatory agent that contributes to the deterioration of the cartilage. This can allow the cartilage to heal, restoring function in the joint. Pharmacokinetic/absorption, distribution, metabolism and excretion (PK/ADME) studies and Investigational New Drug (IND)-directed toxicology studies have been completed on the proposed gene vector by BrIDGs.