rAAV9.pIR-sIL-1Ra

rAAV9.pIR-sIL-1Ra is an in vivo gene therapy vector based on recombinant adeno-associated virus serotype 9 (rAAV9) designed to deliver an inflammation-inducible, secreted human interleukin-1 receptor antagonist (sIL-1Ra). The vector utilizes an inflammation-responsive promoter (pIR) that drives the expression of sIL-1Ra specifically in response to pro-inflammatory cytokines and bone morphogenic proteins (BMPs) found in inflamed joints. This regulated expression mechanism aims to provide localized therapeutic effects while avoiding the potential side effects of constitutive transgene expression. Developed at the University of Massachusetts Chan Medical School, the therapy is being investigated for chronic inflammatory diseases, including rheumatoid arthritis and autoinflammatory conditions like Deficiency of IL-1 Receptor Antagonist (DIRA).