Spotlight on Hemophilia with Dr. Josh Yoder

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Gene Therapy Insights

Elena Buglo

5 episodes

2 days ago

Discover Gene Therapy Insights - your portal to the cutting-edge world of gene therapy and precision medicine. Our mission is to gather and ignite novel insights that propel the field of genomic medicines forward. Join your host neurogeneticist Elena Buglo, PhD as we explore the growing spectrum of therapeutic targets for gene therapy, the challenges in clinical development, delivery, commercialization and the highlight the monumental breakthroughs with industry experts and thought leaders. Subscribe now for a journey into the future of medicine.

Life Sciences

Science

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All content for Gene Therapy Insights is the property of Elena Buglo and is served directly from their servers with no modification, redirects, or rehosting. The podcast is not affiliated with or endorsed by Podjoint in any way.

Life Sciences

Science

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Spotlight on Hemophilia with Dr. Josh Yoder

Gene Therapy Insights

55 minutes 51 seconds

1 year ago

Spotlight on Hemophilia with Dr. Josh Yoder

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.