Patient with Preexisting Immunity to AAV Vectors Granted Gene Therapy After Antibody-Depleting Agent Allowed Eligibility
Historical barrier:
Many patients were ineligible for AAV-based gene therapy due to preexisting neutralizing antibodies, which block viral vector delivery.
Recent development:
Some clinical trials now use antibody-chopping drugs (e.g., immunoglobulin-cleaving enzymes, FcRn inhibitors, or similar agents) to temporarily reduce or eliminate neutralizing antibodies against AAV, allowing previously excluded patients to receive treatment.
Current status:
Patient stories of this kind are emerging, with reports in specialist forums and industry news, but as of October 2025, no large-scale clinical trial or FDA-approved protocol has been broadly announced for this use—these are early, investigational approaches.