Gene Therapies for Inherited Retinal Diseases

Augmentation, RNA editing, and gene-agnostic approaches offer hope for patients with RP, Stargardt disease, LCA, and XLRS.

By: Christine Nichols Kay, MD

Retinal Physician October 1, 2025 Vol 22, Issue October 2025 Page(s): 10-13

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Gene therapies such as augmentation and RNA editing are advancing treatment options for inherited retinal diseases like RP and Stargardt disease.
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Voretigene neparvovec (Luxturna) was the first retinal gene therapy approved in 2017, significantly changing the IRD treatment landscape.
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The phase 3 LUMEOS trial of bota-vec for XLRP showed directionally supportive results in vision-guided mobility, though primary endpoints were not statistically significant.
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Laru-zova, a gene therapy for XLRP, is undergoing pivotal trials and has shown promising initial results in improving visual function.
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ACDN-01, an RNA exon editor for Stargardt disease, is the first of its kind in clinical development, demonstrating effective RNA editing in preclinical studies.

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