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News|Articles|August 4, 2026

Pediatric eye care in H1 2026: Gene therapy momentum, one regulatory setback

Author(s)Kassi Filkins

The first half of 2026 turned on inherited pediatric eye disease, with 2 gene therapy programs advancing toward registration and a high-profile complete response letter in a childhood-onset optic neuropathy.

If the first half of 2026 had a throughline for children's eye health—it was inherited retinal and optic disease moving off the bench and toward the clinic. Two AAV-based programs cleared meaningful regulatory hurdles, while a closely watched optic neuropathy therapy stalled. Here is what pediatric and retinal specialists should have on their radar heading into the second half of the year.