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<p>The rapid advancement of cell and gene therapies (CGT) in the past ten years has inspired biopharmaceutical companies, biotechnologies, and nonprofits to tackle diseases that have traditionally been challenging to treat. Rare diseases, where roughly 80% have a genetic basis, have enjoyed this scrutiny, but the complexity of CGT trial design and implementation have proven challenging. This manuscript offers general guidance for CGT clinical development, current regulatory requirements and guidelines governed by FDA and EMA, considerations around preclinical development, safety monitoring and the need for long-term monitoring and follow up.</p>

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Insights on Clinical Development of Cell and Gene Therapy for Rare Diseases—by DahShu Innovative Design Scientific Working Group (IDSWG)

  • 期刊论文

  • 发表日期：

    2025年8月8日

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  • Chenkun Wang,
  • Junrui Di,
  • Mercedeh Ghadessi,
  • Rui Tang,
  • Caroline Mulatya,
  • Daoyuan Shi,
  • Tu Xu,
  • Wenquan Wang,
  • Chaoqun Mei,
  • Susan Wang,
  • Bryan McComb,
  • Robert A. Beckman,
  • Gianna McMillan

摘要

The rapid advancement of cell and gene therapies (CGT) in the past ten years has inspired biopharmaceutical companies, biotechnologies, and nonprofits to tackle diseases that have traditionally been challenging to treat. Rare diseases, where roughly 80% have a genetic basis, have enjoyed this scrutiny, but the complexity of CGT trial design and implementation have proven challenging. This manuscript offers general guidance for CGT clinical development, current regulatory requirements and guidelines governed by FDA and EMA, considerations around preclinical development, safety monitoring and the need for long-term monitoring and follow up.