AAO 2025: Early optogenetic therapy shows promise for patients with inherited retinal diseases

Stephen Tsang, MD, PhD, professor at Columbia University and attending physician at NewYork-Presbyterian Hospital, shared encouraging early findings on the use of optogenetic therapy for patients with retinitis pigmentosa and Stargardt disease.

According to Tsang, a single optogenetic treatment has led to measurable improvements in vision for patients affected by these inherited retinal diseases. In patients with retinitis pigmentosa, vision gains averaged about five letters on an eye chart. For those with Stargardt disease, improvements reached nine letters without an amplifier and up to 13 letters when used in combination with amplification glasses.

“These improvements translate into better mobility and object recognition for patients,” Tsang noted.

While early-phase trials primarily focus on safety, results so far have been reassuring, with no serious adverse events that were not controllable by standard steroid treatment—typical for intravitreal gene therapies.

Tsang emphasized the broad potential of optogenetics, noting that the approach could benefit patients regardless of their specific genetic mutation. “There are more than 80 genes that can cause retinitis pigmentosa and over 500 mutations in Stargardt disease,” he explained. “Optogenetics has the advantage of addressing all these variations, whereas precision gene-editing techniques like CRISPR may not be practical for every patient.”