Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence

Scientific Advancements in Gene Therapies:
Opportunities for Global Regulatory Convergence

Hybrid Public Workshop: In-Person and Virtual
September 4, 2024 | 10am-4pm (eastern)

The Reagan-Udall Foundation for the FDA, in collaboration with the Bill & Melinda Gates Foundation, hosted a hybrid public workshop on "Scientific Advancements in Gene Therapies: Opportunities for Global Regulatory Convergence."

Gene therapies work by replacing, silencing, and/or modifying specific genes within a cell to treat, prevent, or cure a disease, and have been used to treat both inherited diseases such as sickle cell disease and acquired disorders such as leukemia.

This public workshop provided a baseline about the current state of gene therapy interventions, including approved products and considerations for currently licensed gene therapy products and regulator roles regarding these therapies, particularly in low and middle income countries; explored the "next generation" of gene therapies and the breadth of where those products might be deployed; and discussed the opportunities and needs for regulatory oversight of these products in emerging markets. Speakers included patient advocates, clinicians, academic researchers, biotech/pharma representatives, and regulators.