AAV Gene Therapy – Toxicity Risks – 9 September 2021

  • Public Equity
  • Healthcare
  • North America


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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