A panel of advisors to the US Food and Drug Administration has endorsed a gene therapy to treat a form of childhood blindness, the Associated Press reports. The panel voted unanimously in favor of the therapy, it adds
Spark Therapeutics' voretigene neparvovec, or Luxturna, has been developed to treat blindness stemming from mutations in the RPE65 gene. As Nature News notes, the treatment involves packing viruses with normal versions of RPE65 gene and injecting them into the eye to coerce it to produce healthy RPE65 proteins. In a trial conducted by Spark of some 30 patients, more than 90 percent of those treated had some degree of improvement in their vision, NPR adds.
"I've been able to see things that I've never seen before, like stars, fireworks, and even the moon," patient Christian Guardino told the committee, according to NPR.
NPR says that FDA doesn't have to follow the recommendations of its expert panels, but that it usually does. If approved, this would be the first approved gene therapy in the US. A final decision from the agency is expected in mid-January, Nature News says.