Advanced Cell and Gene Therapy

Regulatory Landscape for Cell and Gene Therapies

The regulatory landscape for cell and gene therapies is evolving rapidly to keep pace with the scientific advancements in these fields. Regulatory agencies worldwide are working to establish frameworks that ensure the safety, efficacy, and quality of these innovative therapies while facilitating their development and approval. The FDA’s Center for Biologics Evaluation and Research (CBER) oversees the regulation of cell and gene therapies. Key regulatory pathways include Investigational New Drug (IND) applications, Biologics License Applications (BLA), and Fast Track, Breakthrough Therapy, and Regenerative Medicine Advanced Therapy (RMAT) designations. The EMA’s Committee for Advanced Therapies (CAT) is responsible for the assessment of advanced therapy medicinal products (ATMPs), including cell and gene therapies. The centralized marketing authorization procedure is mandatory for ATMPs in the EU. In the U.S., a BLA is submitted to the FDA for the approval of biologic products, including cell and gene therapies. This application includes comprehensive data from clinical trials and manufacturing processes. The regulatory landscape for cell and gene therapies is complex and rapidly evolving, reflecting the innovative nature of these treatments.