Clinical Development of Gene Therapies

Interview with Oscar Segurado, MD PhD

Key points:

• Gene Therapy Uniqueness: Differences from traditional drug development
• Safety & Dosing: Single-dose approach, safety considerations
• Statisticians' Role: Essential contributions in planning and analysis
• Guest Background: Oscar’s expertise in academia, med tech, biopharma, Humira development
• Clinical Development: Phases from trials to commercialization
• Rare Disease Focus: Hemophilia A, spinal muscular atrophy
• Challenges & Opportunities: Unique clinical and statistical challenges
• Future of Medicine: Insights into advancements in gene therapy

Oscar’s expertise sheds light on the unique challenges and groundbreaking possibilities in this field, highlighting the critical role that statisticians and data scientists play in advancing these therapies.

If you found this episode insightful, I encourage you to listen in full and share it with colleagues, friends, or anyone interested in the future of healthcare. Let’s spread the word on how statistical expertise is driving the next frontier in medicine—tune in, learn, and pass it on!