Exploring Key Insights Gained from Recent Regulatory Submissions & Approvals Regulatory Differences Internationally

Time: 12:01 pm
day: Conference Day One - Exploring Key Insights 12pm


  • Gaining insights into breakthrough therapy designation, accelerated approval, and other fast-track mechanisms used to expedite the development and regulatory review of transformative gene therapies
  • Harnessing real world evidence to bolster clinical trial results offering additional safety and efficacy information and validating findings in broader patient populations
  • Demonstrating flexibility in adaptive trial designs to address emerging scientific insights and regulatory guidance to optimize patient recruitment, ensuring a timely and robust dataset