The field of inherited retinal disease research is exploding with more than 30 therapies under investigation for Leber congenital amaurosis, retinitis pigmentosa, Stargardt disease, cone and cone-rod dystrophies, achromatopsia, choroideremia, Usher syndrome, and others. Although many still rely on traditional AAV vectors, several novel mechanisms of action and delivery methods hold promise for improved efficacy and safety and, potentially, gene agnostic treatment. Here, we provide a snapshot of the pipeline to help you educate patients—and foster hope that therapies may soon be within reach for some conditions.
- The Staff of Retina Today
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