Navigating Regulatory Complexities: Lessons from Bluebird Bio’s Approach to Gene Therapy Approval
Time: 9:30 am
day: Conference Day Two pt 1
Details:
- How to define and select surrogate endpoints that accurately reflect clinical benefit in rare disease gene therapies
- Strategies for engaging with the FDA to gain agreement on surrogate endpoints
- Collaborating closely with regulatory agencies to address unique gene therapy challenges and achieve approvals, including lessons learned from early submissions and the company’s proactive responses to regulatory feedback
