Accelerating Clinical Trials in Rare Diseases by Moving Beyond One Disease at a Time

Time: 8:40 am
day: Day Two


  • Exploring platform approaches to gene therapies to accelerate therapeutic development for monogenic diseases
  • How much can we push platform capacity of AAV’s?
  • How can we run clinical trials more efficiently?
  • The need to streamline our current approaches and enable a more scalable, and inherently patient-centric approach
  • Evaluating broader collaborative approaches that the NIH are taking to accelerate gene therapy clinical trials