Releasing the Gene Genie
The gene therapy era in ophthalmology edges nearer and is making RP… treatable
Mark Hillen |
The last four months have seen a number of landmarks in retinal gene therapy for retinitis pigmentosa (RP). In April, Robert MacLaren performed the first ever human subretinal injection of AAV-XLRPGR gene therapy. But last month, the first phase III clinical trial data were published of Spark Therapeutics’ voretigene neparvovec (AAV2-hRPE65v2), as a potential one-time gene therapy candidate for the treatment of patients with vision loss (20/60 or worse) due to confirmed biallelic RPE65-mediated inherited retinal disease (1). In one week in November 2013, 31 individuals were enrolled with 21 being randomized to receive bilateral subretinal voretigene neparvovec injections, and 10 to receive control injections – although one patient from each group withdrew before the injections occurred.
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