Adeno-Associated Virus (AAV) gene therapy represents a breakthrough in the treatment of genetic diseases. Yet, recent clinical trials have highlighted safety concerns, including severe adverse events. These incidents underscore the critical importance of embedding safety-focused practices into every stage of trial design and execution.
This guide presents insights drawn from recent AAV trial experiences and offers actionable strategies for strengthening safety protocols. Readers will find lessons learned from recent trials and considerations on how those experiences can inform safer clinical practices. The guide outlines eight core practices for managing patient safety throughout a trial, from pre-enrollment through long-term follow-up. It also includes recommendations for fostering a culture of vigilance, transparency, and ethical responsibility across gene therapy programs. Finally, a comprehensive safety checklist is provided to help ensure that safeguards are in place before, during, and after patient dosing.
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