AAV9-APOA1

AAV9-APOA1 is an experimental gene therapy consisting of an adeno-associated virus serotype 9 (AAV9) vector that encodes the apolipoprotein A-I (ApoA-I) protein. Developed by researchers at the University of California, San Francisco (UCSF), it is being investigated for the treatment of glioblastoma. The therapy is administered via convection-enhanced delivery (CED) directly into the tumor to bypass the blood-brain barrier and achieve broad distribution within the central nervous system. Its mechanism of action involves reprogramming the immunosuppressive glioblastoma immune microenvironment (TIME) by promoting the recruitment and activation of anti-tumor immune cells, specifically CD8+ T cells and microglia, while simultaneously reducing the infiltration of immunosuppressive macrophages. Preclinical studies in syngeneic mouse models have demonstrated that AAV9-APOA1 suppresses pro-tumorigenic cytokines like IL-6 and LIF, resulting in inhibited tumor growth and improved survival outcomes.

Other names

AAV9-Apoa1AAV-9-Apoa1AAV 9-Apoa1AAV9-APO-A1AAV-9-APO-A1AAV 9-APO-A1