Insights Gained From the Successful Launch of a Gene Therapy Product into the EU

Time: 1:30 pm
day: Day Two


  • From preclinical research to FDA then EMA approval – an overview of the milestones in the approval process for Zolgensma
  • Explore how patient safety was demonstrated to the regulators and the key challenges faced in doing so
  • The specific challenges in developing a gene therapy product for a small biotech company and how these were overcome