China is potentially the world’s largest market for rare disease treatments, but many experts believe that China will need to develop its own gene therapies, given that such imported products will be simply too costly for what is a largely self-pay market. For one father in China, the near-impossible journey to find a new gene therapy to treat his son’s extremely rare condition spanned two continents. This fascinating account from In Vivo illustrates the frustrations and sometimes heartbreak in a country where a confusing regulatory pathway for emerging technologies has hospitals and bio pharma firms being regulated by two separate regulatory oversight systems.