Dr Szilárd Kiss

Phase 1 clinical trial data shows promising results for patients with wet age-related macular degeneration (AMD), maintaining vision for at least six months after a single intravitreal injection of gene therapy.

“This is potentially paradigm shifting,” said study lead Dr Szilárd Kiss of Weill Cornell Medical College, New York. “It’s the next revolutionary leap in treating AMD. When you think about what is science fiction and what is science reality; gene therapy for AMD is becoming a clinical reality.”

The researchers, presenting at the American Academy of Ophthalmology (AAO) general meeting, said they hope gene therapy will free patients with AMD from nearly monthly eye injections by offering a potential of “one-and-done” treatment. “It’s not just about convenience; a more consistent treatment may also help people keep more of their vision.”

Dr Kiss said he expects a gene therapy for wet AMD could be available within the next three to five years, depending upon the results of further clinical studies and regulatory approvals.

The trial included 12 patients who received a single injection of gene therapy for wet AMD. Prior to the study, the patients had received an average of 35 anti-VEGF injections; one patient had 109. Since entering the gene therapy trial, the patients required no rescue treatments throughout the first six months.