Biallelic RPE65 mutation-associated retinal dystrophy

Clinical trials for Biallelic RPE65 mutation-associated retinal dystrophy explained in plain language.

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Gene therapy brings light to rare eye disease in japanese patients

Disease control Completed

This study tested a gene therapy called voretigene neparvovec in 4 Japanese patients with a rare inherited eye disease that causes vision loss. The treatment was given as an injection under the retina to replace the faulty gene. The goal was to see if it could safely improve ligh…

Matched conditions: BIALLELIC RPE65 MUTATION-ASSOCIATED RETINAL DYSTROPHY

Phase 3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control

Last updated Jun 27, 2026 13:03 UTC

Biallelic RPE65 mutation-associated retinal dystrophy

Get alerted when new Biallelic RPE65 mutation-associated retinal dystrophy trials appear

Gene therapy brings light to rare eye disease in japanese patients