Using a “one-and-done” gene therapy to treat wet AMD

Six patients with wet age-related macular degeneration have gone for six months without the need for regular injections

DNA image
US researchers have highlighted the potential of using gene therapy to treat wet age-related macular degeneration (AMD) at the annual meeting of the American Academy of Ophthalmology (12–15 October, San Francisco).

Dr Szilard Kiss, from Weill Cornell Medical College, shared his belief that a gene therapy for wet AMD could be available within the next three to five years.

Dr Kiss and his team developed a vector that inserts genetic material producing a molecule similar to anti-VEGF medicine aflibercept within the cells of the eye.

Once inserted, the DNA sequence begins making aflibercept protein.

“Instead of taking a vile of aflibercept and injecting it into the eye, your eye makes the aflibercept,” Dr Kiss highlighted.

“The goal is a potentially one-and-done treatment. You may need a booster once in a while, but this gene therapy could theoretically last a lifetime,” he added.

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