Advancements in gene editing tools: Balancing innovation with regulatory expectations

New gene editing platforms are expanding therapeutic possibilities, yet clinical translation depends on balancing technical progress with rigorous regulatory standards. This spotlight session explores how developers can align innovation with safety, reproducibility, and oversight to ensure responsible advancement of gene editing in advanced therapies. 

Key takeaways: 

• Review recent advancements in gene editing tools and their therapeutic applications. 

• Assess safety margins, off-target effects, and strategies for risk mitigation. 

• Explore frameworks to enhance reproducibility and translational fidelity in gene editing. 

• Discuss regulatory expectations for clinical trial design and product characterization. 

• Identify approaches to maintain innovation while ensuring compliance and patient safety.