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Gene and cell therapies in China: booming landscape under dual-track regulation

Chen Yin1, Jianchao Gao2, Guanqiao Li3

  • 1Tsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, China.

Journal of Hematology & Oncology
|October 5, 2022
PubMed

Insights

Gene and cell therapy (GCT) in China is rapidly advancing, particularly for cancer treatment. Dual regulatory pathways complement each other, accelerating GCT development, especially after 2017.

Area of Science:

  • Oncology
  • Biotechnology
  • Regulatory Science

Background:

  • Gene and cell therapy (GCT) has seen significant global growth, with a strong focus on cancer applications.
  • A comprehensive analysis of 953 clinical trials in China (up to March 2021) was conducted, examining Investigational New Drugs (IND) and investigator-initiated trials (IITs).
  • GCT products were categorized to analyze clinical development across phases, regulatory tracks, disease areas, indications, and targets.

Discussion:

  • CAR-T therapies (ex vivo) and stem/somatic cells (non-gene) are the most investigated GCT types, predominantly targeting cancers.
  • Investigator-initiated trials (IITs) significantly outnumbered IND-registered trials, except for the in vivo category.
  • A notable boom in all GCT categories, particularly ex vivo, occurred after the 2017 cell therapy guideline issuance.

Key Insights:

  • China's dual-track regulatory system effectively complements IITs and IND-registered trials, fostering GCT development.
  • The period after 2017 marked a significant acceleration in GCT product development across all categories.
  • CAR-T and stem/somatic cell therapies represent the leading areas of GCT research and clinical application in China.

Outlook:

  • Harmonizing technical standards and implementing risk-based regulations are crucial for future GCT advancements.
  • Continued collaboration between regulatory bodies and research institutions will expedite the delivery of novel GCT products to patients.
  • The findings underscore the synergistic effect of China's regulatory framework in promoting the gene and cell therapy landscape.
Keywords:
Clinical developmentDual-track regulationGCT

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