Clinical Case Study: Executing a Gene Therapy Trial in New Geographies & the Experience with Onasemnogene Abeparvovec in Spinal Muscular Atrophy
Time: 2:00 pm
day: Day 2- Clinical 2
Details:
- Insights into adapting clinical trial protocols and overcoming logistical challenges in diverse regulatory environments for gene therapy trials
- Key takeaways from conducting the OAV101 IT trial for later-onset spinal muscular atrophy in varied international settings
- Strategies to maintain data integrity, patient safety, and regulatory alignment across multiple geographic regions
