[HTML][HTML] Gene therapy for age-related macular degeneration

IJ Constable, MS Blumenkranz, SD Schwartz… - Asia-Pacific Journal of …, 2016 - Elsevier
The purpose of this article was to evaluate safety and signals of efficacy of gene therapy with
subretinal rAAV. sFlt-1 for wet age-related macular degeneration (wet AMD). A phase 1 dose-
escalating single-center controlled unmasked human clinical trial was followed up by
extension of the protocol to a phase 2A single-center trial. rAAV. sFlt-1 vector was used to
deliver a naturally occurring anti–vascular endothelial growth factor agent, sFlt-1, into the
subretinal space. In phase 1, step 1 randomized 3 subjects to low-dose rAAV. sFlt-1 (1ื 10 …