Co-director at Children's Healthcare of Atlanta Inc
- 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
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