Eye Gene Therapy Linked to Vision Gains in Stargardt, Heads to Geographic Atrophy Trial

This article is based on Ophthalmology Times's report of data shown at the American Academy of Ophthalmology meeting in New Orleans. The underlying paper was not reviewed.

The advanced form of dry macular degeneration is called geographic atrophy. According to Ophthalmology Times, it is estimated to affect around 1 million people in the United States. Two drugs were approved for it in 2023, pegcetacoplan (Syfovre) and avacincaptad pegol (Izervay). Both slow the growth of damaged areas in the retina. According to the report, neither has been shown to improve visual acuity, and no approved therapy restores function where light-sensing cells are already gone.

The new approach is called sonpiretigene isteparvovec, or son-vec, formerly MCO010. It is an optogenetic therapy. According to Ophthalmology Times, a harmless virus delivers a gene for a light-sensitive protein into ON bipolar cells. These cells often survive after the photoreceptors degenerate. The cells then respond to light directly. The report says this approach does not target a specific mutation, and that the injection is given in the office.

The human data come from Stargardt disease, an inherited condition that damages the macula. According to the report, patients with vision of 20/200 or worse received a single injection. Those whose atrophy was limited to the macula gained an average of 12 letters on an eye chart. The report says the therapy was well tolerated, with no serious adverse events.

The report also describes an earlier published trial, called STARLIGHT, of six adults with Stargardt disease. At 48 weeks, average best-corrected acuity had improved by 5.5 letters. Acuity measured with a wearable magnifier improved by 13.3 letters. The reported side effects included conjunctival hemorrhage, ocular hypertension and vitreous cells. The report lists no deaths, hospitalizations, retinal detachments or endophthalmitis.

For geographic atrophy itself, the evidence is from animals. According to the report, monkeys with laser-induced lesions that model the disease showed stronger electrical responses in treated eyes than in untreated eyes. Those data have not been published in a peer-reviewed journal.

Several cautions apply. The report says the 12-letter figure comes from conference data. It does not give the subgroup size or the follow-up time, and the figure has not been published. Both Stargardt studies were open-label, with no masking or sham control. The people studied had an inherited disease, not age-related macular degeneration. Whether the effect carries over to older adults with geographic atrophy is unknown.

The investigators plan a randomized, controlled phase 2 trial in geographic atrophy, skipping a separate phase 1 safety study in that group. According to the report, positive results would lead to larger phase 2b/3a trials aimed at regulatory approval. The report says no registry record for the geographic atrophy trial could be identified. Those steps remain ahead, which is why any benefit for patients with this condition is still distant.

What to watch next

The registration and launch of the geographic atrophy trial, and publication of the Stargardt subgroup data.

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