Medical and Regulatory Affairs

Operationalizing Gene Therapy Trials

Guide to gene therapy’s biggest challenges

Gene therapy holds great promise for treating and curing previously incurable diseases, ranging from cancer and diabetes to rare genetic disorders. But despite revolutionary advances, a clear path from bench to bedside remains elusive.

This white paper examines gene therapy from its origins through the details of operationalizing modern clinical trials, recruiting, and keeping patients enrolled. You’ll learn:

  • How the field’s genesis in the 1960s laid the groundwork for more than 2,400 gene therapy trials performed to date
  • Considerations for effective trial planning at every stage, from vendor and site selection to creating backup plans for each study component
  • Strategies for overcoming challenges in recruiting from small patient populations and retaining subjects for lengthy follow-up periods

The outlook for gene therapy is brighter today than ever. Download the white paper for more tips on seizing this unprecedented opportunity.