ISCT Cell and Gene Therapy Global Regulatory Report H1 2024
Report
Clinical
Overview of Report
Welcome to the third biannual Cell and Gene Therapy Global Regulatory Report from ISCT and Citeline. This report provides a global overview of the cell and gene therapy regulatory landscape, including pipeline, late-stage (Phase III and pre-registration), and approved products. It covers Cell, Genetically-Modified Cell, and Gene Therapies.
Over the last few years, the regulatory agencies have been updating legislation and frameworks to keep up with the rapidly advancing cell and gene therapy space. As a result, ISCT and Citeline have collaborated to highlight some of these changes and offer commentary on the updates and their effects.
In this report, we see that year-over-year, non-genetically modified cell therapies continue to make up the majority of all approved products (68%). However, the pipeline for genetically modified cell therapies continues to be larger with 1,008 therapies in development, compared to 902 non-genetically modified cell therapies and 938 gene therapies.
Meanwhile, ISCT continues to track regulatory changes and events and provide comments on Key Global Legislative/Framework Changes including US FDA draft guidance in potency assurance for CGT products and EMA reflection paper on the use of Real-World Data in Non-Interventional Studies to Generate Real-World Evidence.
