RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.
The combination of high cost, long development times, and small patient populations for rare diseases is driving new models to accelerate drug development. The story of the newly formed rare disease company Vtesse is a compelling example. Driven by parents, advanced to the clinic by the National Institutes of Health, and licensed by a new company formed out of an orphan drug accelerator, Vtesse is advancing an experimental drug to treat Niemann Pick Disease Type C and other lysosomal storage disorders. We spoke to Ben Machielse, CEO of Vtesse, about his company, the unusual collaborations that led to its formation, and what it says about how patients, government, investors, and industry can work together to speed the development of new therapies for rare diseases.