In Vivo CRISPR Treatment for Hereditary Angioedema Receives BLA Approval After Phase III Success

An in vivo CRISPR gene editing treatment for hereditary angioedema (HAE) has cleared Phase III clinical trials and received Biologics License Application (BLA) approval, marking a significant clinical milestone for in-body gene editing. This approval demonstrates that CRISPR-based therapies can achieve regulatory success through pivotal trials, advancing the field beyond ex vivo editing approaches.