The debut episode hosted by Elena Buglo, PhD features Josh Yoder, PhD, a medical affairs leader with expertise in virology and gene therapy, focusing on hemophilia.

Our discussion delves into:
🩸 Overview of Hemophilia: We explore where hemophilia stands in the realm of rare diseases and why it's a prime target for gene therapy.

🎯 Targeting Hemophilia with Gene Therapy: The rationale behind targeting hemophilia using gene therapy, we uncover intricacies and breakthroughs in treatment; we explore the unique challenges of liver-targeted gene therapy.

🔬 Tools in Hemophilia Gene Therapy: From the evolution of AAVs to the latest clinical and pre-clinical approaches, we discuss the advancements and challenges in targeting hemophilia.

💉 Immune Responses and Viral Vectors: We discuss the complexities of immune responses to AAV capsids and the latest developments in managing these responses, ensuring the safety of gene therapy.

🔍 Nuances Impacting Efficacy: We highlight factors like the Padua variant of factor IX, vector dosage tuning, and potential hepatotoxicity risks from overexpression.

💼 Insights from Industry Experience: Drawing from Dr. Yoder's experience at uniQure, we explore economic model changes, patient selection criteria, and access challenges.

💡Thoughts on the Future: Dr. Yoder shares his thoughts on the future impact of gene therapy, prospects of AI on clinical trial and therapy design, real-world evidence, and what lies ahead.

Join us in this educational journey as we foster knowledge, explore innovative ideas, and ignite conversations on the leading edge of gene therapy. Stay tuned for more thought-provoking discussions with academic thought leaders and industry experts.

Disclaimer: Opinions expressed on this podcast are those of the speaker and are separate from any professional affiliations. Nothing on this podcast should be deemed as professional or medical advice.

This episode is brought to you in media partnership with the CRISPR 2.0 Congress which will take place in Boston on November 28th - 30th, 2023. You can register here.