scAAV.IL-1Ra

**scAAV.IL-1Ra** is a gene therapy agent utilizing a self-complementary adeno-associated virus (scAAV) vector, typically with AAV2.5 capsid, to deliver the cDNA encoding interleukin-1 receptor antagonist (IL-1Ra)—either equine (eqIL-1Ra) or human (hIL-1Ra)—directly into joint tissues via intra-articular injection. It enables sustained local production of IL-1Ra, a natural competitive inhibitor of IL-1 signaling, to suppress inflammation, cartilage degradation, pain, and progression in osteoarthritis (OA). Preclinical studies in equine models of post-traumatic OA (PTOA) demonstrated elevated synovial IL-1Ra levels (up to >400-fold over IL-1, sustained for months at doses like 5×10^12 vg), reduced lameness (30-40%), lowered PGE2, improved osteochondral repair, and contained vector biodistribution with no systemic transgene spread. A Mayo Clinic phase 1 trial confirmed safety and tolerability in human knees with moderate OA; phase 1b completion anticipated in 2025.[1][2][7]

Other names

AAV2.5-IL-1RaAAV-2.5-IL-1RaAAV 2.5-IL-1RaHumulin R U-100-Mayo Clinic-osteoarthritis (knee)