Personalized Drug Design: New Therapies for Rare Genetic Diseases with Dr. Stanley Crooke

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)
• n-Lorem Foundation
• Hope Lies in Dreams – Nature’s 10-part series on Dr. Crooke
• The National Economic Burden of Rare Disease Study (EveryLife Foundation)

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Production Team: Director & Host: Dasha Tyshlek, StratCraft, Inc. 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 Produced on behalf of: Wallace H. Coulter Center for Translational Research at University of Virginia