Using Exosomes to Tackle the Delivery Challenge of Genetic Medicines for the Brain

Genetic medicine has already begun to transform the treatment of some inherited diseases, but the brain remains one of its most formidable frontiers. For disorders such as Huntington’s disease and ALS, scientists may understand key genetic contributors and have increasingly powerful editing tools, yet getting those tools safely, efficiently, and selectively into vulnerable neurons is another matter entirely. Evox Therapeutics is developing an approach built around naturally occurring nanoparticles that cells use to communicate, known as exosomes, as delivery vehicles for CRISPR-based medicines. We spoke with Per Lundin, the company’s co-founder and CEO, about the promise and limitations of exosome-enabled gene editing, how the company is prioritizing its pipeline, and what it will take to establish this emerging therapeutic strategy in the clinic.