Navigating the Regulatory Route and Challenges for Gene Therapies Targeting Common Diseases

Time: 1:30 pm
day: Conference Day One


  • Overview of regulatory challenges and strategies for gene therapies targeting common disease
  • Establishing long-term safety and efficacy of gene therapies to overcome safety concerns in larger patient cohort over extended periods
  • Identifying new approaches to upscale manufacturing to serve a larger market 
  • Confirming additional requirements on clinical data and post-marketing surveillance that would be required in a common disease setting compared to rare disease