OPTIC, ATROPHY, HEREDITARY, LEBER
Clinical trials for OPTIC, ATROPHY, HEREDITARY, LEBER explained in plain language.
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Gene therapy injection shows promise for rare eye disease
Disease control CompletedThis phase 3 trial tested a single injection of GS010 gene therapy in 39 people with Leber Hereditary Optic Neuropathy (LHON) who had vision loss for 6 months or less. The treatment aims to improve vision by delivering a working copy of the ND4 gene to the eye. Results measured c…
Matched conditions: OPTIC, ATROPHY, HEREDITARY, LEBER
Phase: PHASE3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Gene therapy shows promise for rare eye disease causing blindness
Disease control CompletedThis study tested a one-time gene therapy injection, GS010, in 37 adults with Leber Hereditary Optic Neuropathy (LHON), a genetic condition that causes rapid vision loss. Participants had vision loss for 6 to 12 months before treatment. The goal was to see if the therapy could im…
Matched conditions: OPTIC, ATROPHY, HEREDITARY, LEBER
Phase: PHASE3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC