Spark Therapeutics announced The Lancet, has published Phase III clinical trial data of voretigene neparvovec, for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease.

Spark Therapeutics announced The Lancet, has published Phase III clinical trial data of voretigene neparvovec, an investigational, potential one-time gene therapy candidate for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD). In the trial, investigational voretigene neparvovec improved functional vision, light sensitivity and visual field in study participants with RPE65-mediated IRD. A natural history study has shown that people with this IRD eventually progress to complete blindness.

The publication presents the results of the Phase III trial, including the intent-to-treat population of all randomized subjects, through the one-year timepoint. Results showed a statistically significant and clinically meaningful difference between intervention (n=21) and control participants (n=10) at one year, per the clinical trial's primary endpoint, mean bilateral multi-luminance mobility testing (MLMT) change score (difference of 1.6; 95% CI, 0.72, 2.41; p=0.0013). Participants maintained functional gains observed 30 days post-administration at the one-year primary endpoint. MLMT evaluates functional vision by documenting the participants' ability to navigate a mobility course under a variety of specified light levels ranging from one lux (equivalent to, for example, a moonless summer night) to 400 lux (equivalent to, for example, an office environment.

See-"Gene therapy for RPE65-mediated inherited retinal dystrophy completes phase 3."- Helena Lee, Andrew Lotery. Published: 13 July 2017 DOI: http://dx.doi.org/10.1016/S0140-6736(17)31622-7.

See- "Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial."- Dr Prof Stephen Russell, MD'Correspondence information about the author Dr Prof Stephen Russell Prof Jean Bennett, MD, Jennifer A Wellman, MS, Daniel C Chung, DO et al., Published: 13 July 2017. DOI: http://dx.doi.org/10.1016/S0140-6736(17)31868-8 .