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.
