Specialist
Co-director at Children's Healthcare of Atlanta Inc
Agenda
- Adeno-associated virus (AAV) gene therapy risks, discussing FDA evaluation of cancer risks and concerns of toxicity to the liver and brain
- Implications associated with FDA discussions regarding dosing of AAV vectors and path forward
- Deep dive into the clinical trials for haemophilia, spinal muscular atrophy (SMA) and X-linked myotubular myopathy (XLMTM) that were assessed by the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC)
- Outlook for 2021 and beyond
Questions
1.
The FDA has been posing questions for experts to help answer some of the many scientific questions that remain unanswered, and to evaluate an array of safety risks to AAV [adeno-associated virus] gene therapy. Can you outline the safety concerns that surround AAV gene therapy, specifically highlighting cancer risks and concerns of toxicity to liver and the brain?
2.
One of the main questions that is being asked to gene therapy experts includes whether the FDA should be setting an upper limit on the size of the gene therapy doses, due to the risk of serious brain and liver toxicity which you just outlined. Given your experience in the field, what is your take, and do you believe that an upper limit should be set for the total vector genome dose per subject?
3.
What is your assessment of the patient factors that should be evaluated when determining a dose? Obviously, weight is a factor here, but are there any that you believe are being overlooked?
