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research-review:efficacy-and-safety-of-voretigene-neparvovec-phase3
This is an old revision of the document!
This article will be discussed on December 11th at Biotech without Borders as part of the Deep Dive series.
Efficacy and safety of voretigene neparvovec
Russell S, Bennett J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017;390(10097):849-860. doi:10.1016/S0140-6736(17)31868-8
Summary (tldr;)
This trial is the culmination of a long process of bringing a drug to market. It provides a report of a study of patients where 21 were assigned to the therapy and 10 to the control group. Endpoints investigated in this study are intended to support the approval of a gene therapy for a type of genetic blindness called Leber congenital amaurosis type 2 (RPE65-/-). Treatment involved the surgical injection of a recombinant adeno-associated virus (AAV) into th
Schultz BR, Chamberlain JS. Recombinant adeno-associated virus transduction and integration. Mol Ther. 2008;16(7):1189-1199. doi:10.1038/mt.2008.103
research-review/efficacy-and-safety-of-voretigene-neparvovec-phase3.1574806473.txt.gz · Last modified: 2019/11/26 22:14 by danny
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