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GC801 is an investigational IgG-degrading enzyme (protease) developed by The Seventh Medical Center of Chinese PLA General Hospital. It is specifically designed to eliminate serum anti-adeno-associated virus serotype 9 (AAV9) antibodies in pediatric patients with Pompe disease. Pre-existing anti-AAV antibodies often preclude patients from receiving AAV-based gene therapies; GC801 functions by enzymatically cleaving these antibodies, thereby creating a window for the successful administration of AAV9-delivered therapeutic genes. It is currently being evaluated in Phase 1 clinical trials in China.
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