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.
Base Editing for Spinal Muscular Atrophy with Dr. Mandana Arbab
33:08In today's episode, we dive into the base editing approaches in Spinal Muscular Atrophy (SMA), a degenerative motor neuron disease caused by the loss of motor neurons in the spinal cord. Our guest is Dr. Mandana Arbab is the Lodish Family Assistant Professor of Neurology at Harvard Medical School and faculty member of the Rosamund Stone Zender Translational Neuroscience Center at Boston Children's Hospital. She received her Ph. D. in Regenerative Medicine at the Hubercht Institute in the Netherlands and completed her postdoctoral fellowship at the Broad Institute with Professor David Liu. Dr. Arbab sheds light on the genetics of SMA, emphasizing the critical role of SMN genes and the potential of base editing to bring about transformative treatments. SMA stands out as the most common genetic cause of infant death worldwide, affecting patients within the first few months of life. The podcast delves into the existing therapeutics for SMA, such as antisense oligonucleotides and AAV gene therapy, examining their limitations and the quest for more precise and long-term solutions. The concept of base editing takes center stage, offering a promising approach to correct the genetic mutations associated with SMA. Join us as we discuss the challenges and opportunities of bringing base editing into clinical trials. Learn about the intricacies of delivery mechanisms, biodistribution, and the potential for combining base editing with existing therapies. The conversation extends to considerations for clinical trials, including addressing off-target effects and the complexities of patient eligibility. The podcast concludes with a glimpse into the future of CRISPR genome editing, and the exciting potential for transformative treatments in the realm of rare diseases. Tune in to “Gene Therapy Insights” and embark on a journey through the cutting-edge world of gene therapy, where science meets hope, and the future of medicine unfolds.
Energy Replacement in the Light of Aging with Dr. Shahaf Peleg
52:46Tune in to hear from Dr. Shahaf Peleg, PhD, a co-founder of Luminova Biotech and a researcher at the Research Institute for Farm Animal Biology as we explore the exciting work by the pioneers in the field of tackling age-related disorders with a light-activated energy replacement tool. 🔬 Join us as we explore the exciting work by the pioneers in the field, who are pushing the boundaries of science by converting light energy into mitochondrial chemical energy - MtON technology. It's not just a paradigm shift; it's a revolutionary way of thinking about human evolution as we face the disorders of aging. 🧬 Key topics explored: 1️⃣ Discovery of a light-activated proton pump in fungi and exploration of its applications in humans 2️⃣ Energy replacement and its place in mitochondrial dynamics 3️⃣ Connection between epigenetics and metabolism in the pursuit of combating diseases of aging 4️⃣ Comparisons to photobiomodulation as a mitochondrial biogenesis tool 5️⃣ Considerations in the experimental design of a human gene therapy targeting local mitochondria for potential restoration of eyesight and beyond 👉 Your support matters! Like, share, subscribe, and review to help us bring you more thought-provoking episodes in the future. 👥 We value your engagement! Comment below or on your preferred platform with questions, guest suggestions, or topics you'd like us to explore. 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. #BiotechInnovation #GeneTherapy #Optogenetics #MitochondrialScience #Agingscience #HealthcareRevolution #LuminovaBiotech #Longevity #ScienceExploration 🚀🔬
Spotlight on Hemophilia with Dr. Josh Yoder
55:51The 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.