Opinion & analysis

Made In China Gene Therapy A Father’s Quest To Save Son With UltraRare Condition

With a population of 1.3 billion and a large patient pool with rare genetic conditions, China has the potential to be the world’s largest market for rare disease treatments. It is believed, however, 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.  Read about one very determined Chinese father’s journey on two continents to find a research provider with the particular expertise to find a new gene therapy to treat his young son’s very rare condition.