Traditional gene therapies are uber-niche. Ocugen hopes to change that.

When Luxturna was approved in 2017 to treat a rare form of inherited vision loss, it became the first one-and-done therapy targeting a disease caused by mutations in a specific gene. Despite the historical achievement, there was a problem. The patient population was tiny, topping out at around 2,000 in the US., and the drug had become the priciest therapy on the market costing $425,000 per eye.