August 16, 2016
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Gene therapy shows gain in functional vision in continuation of phase 3 trial

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Voretigene neparvovec demonstrated a gain in functional vision in patients with inherited retinal disease caused by the RPE65 gene, Spark Therapeutics announced in a press release.

The phase 3 trial included 20 patients who received voretigene neparvovec, formerly SPK-RPE65, in both eyes (intervention group) and a control group of nine patients. The primary endpoint of the randomized, controlled portion of the trial was change in bilateral mobility testing from baseline to 1 year. Spark previously announced a statistically and clinically significant improvement in bilateral mobility testing in treated patients compared with control patients.

After 1 year, all nine patients in the control group chose to cross over and receive voretigene neparvovec in both eyes. Eight of these patients then showed improvement in mobility testing at 1 year after administration. Mean improvement was 2.1 lux levels after 1 year in the crossover group and 1.9 lux levels after 2 years in the original intervention group, which was maintained from the first year.

Eight of nine crossover patients improved an average of almost 200-fold in full-field light sensitivity threshold testing after 1 year, compared with more than 100-fold in the original intervention group after 2 years, which was maintained from the first year.

Visual acuity improved an average of 4.5 letters in the crossover group and eight letters in the original intervention group.

No product candidate-related serious adverse events occurred. One eye experienced a serious adverse event that was related to the surgical procedure, not voretigene neparvovec.