Cell And Gene Therapy Global Regulatory Report: 2024

The ISCT H1 2024 Global Regulatory Report offers a comprehensive analysis of the evolving cell and gene therapy (CGT) landscape, covering pipeline development, regulatory updates, and approval trends. With contributions from Citeline and ISCT’s Global Regulatory Task Force, the report examines the global CGT pipeline, including non-genetically modified cell, genetically modified cell, and gene therapies.

In H1 2024, non-genetically modified cell therapies continue to dominate with 68% of approved products. However, genetically modified cell therapies show robust growth, with 1,008 therapies in development, surpassing the 902 non-genetically modified and 938 gene therapy projects. The majority of therapies remain in preclinical development, particularly gene therapies with 720 projects at this stage.

Significant regulatory events and updates are highlighted, such as the US FDA’s draft guidance on potency assurance for CGT products and the EMA’s reflection paper on using Real-World Data in non-interventional studies. Noteworthy global approvals include gene-editing therapies for sickle cell anemia and the first allogeneic T-cell immunotherapy in Europe.

This presentation also emphasizes the regulatory environment’s adaptation to rapid CGT advancements, with ISCT playing a key role in consulting on draft guidelines and providing expert insights. These efforts are designed to address challenges in the CGT sector, ensuring safe, effective therapies reach patients efficiently. ISCT’s report serves as an essential resource for stakeholders navigating the CGT regulatory landscape in 2024 and beyond.