Watershed Year for Cell and Gene Therapy: Review of 2024 CGT Approvals

Strategic Insights -

A recent study by researchers from the Cancer Hospital in Beijing, China, highlights the pivotal year 2024 as a significant turning point for therapeutic breakthroughs in human diseases, particularly in cell and gene therapies (CGTs). According to the study, 2024 saw the approval of 13 novel CGTs by the US FDA, China's NMPA, and the EU. The authors analyzed the 2024-approved CGTs in China and the US, employing a quadruple axle comparative framework to integrate mechanistic innovation with regulatory advancements.

Key Takeaways:

  • The study identified 2024 as a watershed year for therapeutic breakthroughs in human diseases, particularly in CGTs, with 13 novel CGTs approved by regulatory agencies worldwide.
  • The analysis employed a quadruple axle comparative framework to integrate mechanistic innovation with regulatory advancements, highlighting the convergence of diversity and agility in therapeutic development.
  • The study concluded that the mechanistic diversity of CGTs converges with regulatory agility to redefine therapeutic development.
  • The researchers noted that 2024 saw several firsts, including the first CRISPR therapy, the first MRI-guided intracranial AAV delivery gene therapy, and the first tumor infiltration lymphocyte therapy.
  • The study emphasized the curative potential of CGTs for previously refractory diseases, offering a new chapter in clinical translation of innovations in gene-editing and cell technologies.

Statistics:

  • 13 novel CGTs were approved by regulatory agencies worldwide in 2024.
  • The study analyzed 2024-approved CGTs in China and the US.
  • The quadruple axle comparative framework was employed to integrate mechanistic innovation with regulatory advancements.
  • 2024 saw several firsts in CGT development, including the first CRISPR therapy, MRI-guided intracranial AAV delivery gene therapy, and tumor infiltration lymphocyte therapy.

Sources:

  • Watershed Year of Cell and Gene Therapy (CGT): A Review of 2024 CGT Approvals. Cancer Letters, 2025:217980.
  • Cancer Hospital, Beijing, People's Republic of China.
  • Aiwen Jian, Yuning Wang, Shuhang Wang, and Ning Li.
  • Elsevier Ireland Ltd, Brookvale Plaza, East Park Shannon, Co, Clare, Ireland.