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Gene Therapy Moves to Committee

In a preliminary review, the US Food and Drug Administration says Spark Therapeutics' gene therapy for an inherited form of blindness appears to be effective, Reuters reports.

FDA posted the review to its website ahead of a meeting Thursday with outside advisors regarding the treatment.

If approved, Reuters notes that the therapy, called Luxturna, or voretigene neparvovec, would be the first gene-therapy approved in the US. The treatment is for Leber congenital amaurosis, which is caused by mutations in the RPE65 gene, though Spark has asked for it to be OK'd for any patient with retinal disease due to a biallelic RPE65 mutation. Spark reports that treatment with their approach in a clinical trial led to 93 percent of participants having some improved vision, according to Reuters.

While Luxturna is envisioned as a one-time treatment, Xconomy adds that the FDA is asking its advisors to consider whether such a treatment should be given more than once. As there is no data from more than a year after treatment, FDA points out that the long-term efficacy of the treatment is as yet unclear. The Philadelphia Inquirer notes, though, that multiple injections could trigger immune reactions.

The Inquirer adds that analysts expect a positive vote.