Biomedical Frontiers: Stories with Innovators in Healthcare
Dr. Stanley Crooke pioneered a new class of medicine—antisense oligonucleotides—and built a $5B biotech company, Ionis Pharmaceuticals, to prove it could work. Then he walked away to found a nonprofit, n-Lorem, to treat patients with one-in-a-billion genetic mutations—people no one else would help. In this episode, we explore: * What nano-rare diseases are, and why they represent medicine’s next great frontier. * The promise of antisense therapy for individualized, mutation-specific treatment. * The 30-year scientific journey behind genetic medicine—and what it takes to lead through failure. * Why n-Lorem offers a new nonprofit model for treating diseases too rare to commercialize. * The privilege, heartbreak, and hope of doing science not just for success, but for purpose. Mentioned Resources: * Antisense Technology: A Review (PubMed) [https://pubmed.ncbi.nlm.nih.gov/33600796/] * n-Lorem Foundation [https://www.nlorem.org/] * Hope Lies in Dreams – Nature’s 10-part series on Dr. Crooke [https://www.nature.com/immersive/d41587-021-00015-5/index.html] * The National Economic Burden of Rare Disease Study (EveryLife Foundation) [https://everylifefoundation.org/burden-study/] Follow Biomedical Frontiers for more: 📸 Instagram: @biomedicalfrontiers 🔗 LinkedIn: https://www.linkedin.com/company/uvacoulter [https://www.linkedin.com/company/uvacoulter]📧 Reach us: biomedicalfrontiers@virginia.edu [biomedicalfrontiers@virginia.edu] Love this podcast? Write to us with feedback or guest recommendations! Production Team: Director & Host: Dasha Tyshlek, StratCraft, Inc. www.strat-craft.com [http://www.strat-craft.com]Executive Producer: David Chen, Managing Director & Instructor of Engineering Design Senior Producer: Hannah Moore, Associate Director, UVA Coulter Design Director: Carolyn Wagner, Inc. & Link: carolynwagnerinc.com [http://carolynwagnerinc.com]Produced on behalf of: Wallace H. Coulter Center for Translational Research at University of Virginia
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