Cell & Gene Day 2024

In this session you will hear about: 

• Scientific advances in cell & gene therapy 
• Recent and anticipated approvals and clinical milestones 
• The CGTx investment landscape 
• Patient access and affordability 

Sarepta’s gene therapy for Duchenne was the first in vivo gene therapy to win accelerated approval — a big milestone, and a contentious one. Join us as reporter Lei Lei Wu talks with Sarepta CEO Doug Ingram about the therapy’s rollout and the FDA’s evolving language around complex rare disease drugs.

In this session you will hear about:  

• Important considerations for clinical development for advanced therapies
• Integrated approach to clinical trial design and execution
• Optimized patient engagement strategies  
• Regulatory alignment and compliance