The FDA’s AAV Approval Playbook Has Gaps That Could Define the Next Decade of Gene Therapy
FDA's reviews of Zolgensma, Luxturna, and Hemgenix reveal critical gaps in AAV trial design, CMC standards, and long-term safety...
AAV Gene Therapy
FDA's reviews of Zolgensma, Luxturna, and Hemgenix reveal critical gaps in AAV trial design, CMC standards, and long-term safety...