Spark Therapeutics treatment for a rare form of blindness may become the first approved gene therapy in the U.S. The SPK-RPE65 treatment has showed to improve sight in patients affected with a form of gene-associated blindness in an advanced clinical test.
"SPK-RPE65 improved vision among people with a form of blindness known as RPE65-mediated inherited retinal dystrophies," said the Philadelphia-based company.
In a study of 31 people with the genetic form of blindness, the therapy allowed patients to better navigate a mobility course under a variety of lighting conditions. The therapy is injected directly into patients' eyes, where viruses insert a working gene into cells that are missing it.
The trial also showed that participants who received the treatment outperformed those in the control group on two secondary goals: improving light sensitivity of the eye and change in mobility test score for the first eye treated.
"We saw substantial restoration of vision in patients who were progressing toward complete blindness," said Albert Maguire, the clinical trial's lead researcher and a professor of ophthalmology at the University of Pennsylvania's Perelman School of Medicine.
For a third endpoint, ability to read a standard eye chart, the treatment didn't result in a statistically significant benefit. Based on the results, Spark plans to submit the treatment to U.S. regulators for approval next year.