Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease

•The authors described an antigen-specific approach to induce immune tolerance to ERT for Pompe disease.•A short-term treatment with anti-CD4 mAb reduced anti-hGAA antibody in Pompe disease mice.•his study suggests an effective adjuvant therapy to ERT for patients with Pompe disease.

Approximately 35–40% of patients with classic infantile Pompe disease treated with enzyme replacement therapy (ERT) develop high, sustained antibody titers against the therapeutic enzyme alglucosidase alfa, which abrogates the treatment efficacy. Induction of antigen-specific immune tolerance would greatly enhance ERT for these patients. Here we show that a short-course treatment with non-depleting anti-CD4 monoclonal antibody successfully induced long-term ERT-specific immune tolerance in Pompe disease mice. Our data suggest an effective adjuvant therapy to ERT.