GNSC-001

GNSC-001 is an investigational gene therapy developed for the treatment of knee osteoarthritis. It is a recombinant adeno-associated viral (AAV) vector that encodes an optimized form of interleukin 1 receptor antagonist (IL-1Ra), a naturally occurring protein that blocks interleukin 1 (IL-1) signaling, which is a key mediator in the pathogenesis of osteoarthritis. By delivering IL-1Ra directly to the joint via intra-articular injection, GNSC-001 aims to provide long-term, sustained inhibition of IL-1 activity after a single administration. This approach targets inflammation and disease progression in osteoarthritis by preventing IL-1 from binding its receptor and activating downstream inflammatory pathways. Preclinical studies have shown durable expression and therapeutic effects lasting over one year following treatment. GNSC-001 has received Fast Track Designation from the FDA for knee osteoarthritis[1][3][5][6][8].

Other names

IL-1RAIL1RAIL 1RAhIL-1RahIL1RahIL 1Ra