After more than two decades of successes and failures, the promise that cell and gene therapy has held for so long is finally a reality. As scientists continue to gain a better understanding of the human genome and how to use the body’s systems to fight disease, we now have therapies that have made it through clinical trials and patients whose lives have been transformed.

​In this playbook, experts weigh in on the evolution of CRISPR gene-editing technology and address the most important issues surrounding its use to understand and treat diseases, including how to:

• Maintain production at a level that matches the pace of innovation
• Work within the existing regulatory playbook
• Avoid inconsistencies in clinical trials that can lead to costly delays 

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