The first gene therapy for an inherited disease in the U.S. is closer to reality than ever before.
Spark Therapeutics is only the second company to pursue an application to the U.S. Food and Drug Administration for such a treatment, but it’s likely to be the first to hit the market.
Speaking at EmTech MIT 2016 on Tuesday, Katherine High, Spark’s cofounder, confirmed that the company is on track to launch its first product next year. The gene therapy, known as SPK-RPE65, targets mutations in people’s eyes that often lead to blindness. Currently, there are no drugs available to treat these disorders, known as inherited retinal dystrophies.
Katherine High, cofounder of Spark Therapeutics, talks with MIT Technology Review’s Antonio Regalado.
Spark plans to complete its FDA application by early 2017. If approved next year, the therapy would become the first for an inherited disease to be given the green light in the U.S. Two such gene therapies, Strimvelis and Glybera, have already been approved in Europe…
Source: MIT Tech Review