Gene therapy has largely stayed out of the brain, and the blood-brain barrier has been the reason why — it protects the brain from harm, but it also blocks most advanced medicines from ever reaching it. That's now beginning to change. The recent FDA approval of a brain-penetrant enzyme therapy for Hunter syndrome showed that biologics can cross that barrier and work. The next question is whether gene editing can do the same.

My guest today is Dr. Per Lundin, Co-Founder and CEO of Evox Therapeutics, a UK biotech using engineered exosomes to deliver genetic medicines directly into the brain. Evox is now extending that platform from RNA and biologics into gene editing, targeting the genetic drivers of diseases like Huntington's and ALS, where treatment options remain extremely limited. Per also brings a background as a European Patent Attorney and repeat biotech founder. We'll talk delivery, pipeline, and what a commercially scalable genetic medicine for the brain might actually look like.

• 02:45 Per Lundin's background and Evox's platform
• 09:38 Denali's Hunter syndrome approval and its significance
• 14:58 Targeting MSH3 to treat Huntington's disease
• 20:03 What commercially scalable genetic medicine requires
• 22:13 Evox's dominant IP position in exosome delivery
• 27:34 What success could mean for patients in ten years

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To dive deeper into the topic: 

• Evox and Lilly Partner to Develop Neuro RNA Drugs in Deal Worth €1.1B
• Six exosome therapy companies driving development in the field
• Extracellular vesicles: a growing pipeline still searching for validation