Spark Therapeutics Inc.’s treatment for a rare form of blindness improved patients’ sight in an advanced clinical test, suggesting it may become the first approved gene therapy in the U.S. The shares rose.
Spark’s medicine, called SPK-RPE65, improved vision among people with a form of blindness known as RPE65-mediated inherited retinal dystrophies, the Philadelphia-based company said in a statement Monday. Based on the results, Spark plans to submit the treatment to U.S. regulators for approval next year. The therapy is injected directly into patients’ eyes, where viruses insert a working gene into cells that are missing it.