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Intravitreal gene therapy shows promise in early-stage trial

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GAITHERSBURG, Md. — Gene therapy for the treatment of advanced neovascular age-related macular degeneration is showing promise in an early phase 1 trial, according to a press release.

GenVec’s AdPEDF gene therapy showed evidence of “a halt in disease progression lasting 6 to 12 months after patients received a single intravitreous injection,” the company said. A total of 28 patients were enrolled in the phase 1 study.

Patients in the group who received higher doses of AdPEDF showed no increase in the size of retinal lesions at 6 and 12 months after injection, compared with patients in the lower dose group whose lesions increased over time, the release said.

AdPEDF is an adenoviral-based vector containing the gene for human pigment epithelium-derived factor (PEDF). In this group of patients, there were no serious adverse events, severe ocular inflammation or dose-limiting toxicities reported.

The study is published in the February issue of Human Gene Therapy.