Regenerative medicineOct 7, 2025

Current progress and challenges in gene therapy for inherited eye diseases

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Jan 2, 2026

Abstract

Voretigene neparvovec-rzyl (Luxturna) has demonstrated proof-of-concept for restoring visual function in a specific inherited retinal disease.

The approval of Luxturna represents a significant advancement in gene therapy for inherited retinal diseases (IRDs).
Current gene therapy options are limited to a narrow subset of IRDs, with no other therapies receiving regulatory approval to date.
Challenges in the field include narrow gene- or variant-specific indications and limitations of available delivery methods.
Emerging strategies such as dual AAV vectors, non-viral delivery systems, and precision gene editing techniques may enhance treatment possibilities.
There is a noted need for improved regulatory frameworks and ethical considerations in gene-based therapies for IRDs.

Simplified

Gene therapy has ushered in a new era for the treatment of inherited retinal diseases (IRDs). The approval of voretigene neparvovec-rzyl (Luxturna) for-associated retinal dystrophy marked a pivotal milestone, establishing proof-of-concept that gene addition can restore visual function in IRDs. However, the success of Luxturna is tempered by the reality that it applies to a narrow subset of IRDs, and that no other IRD gene therapy has thus far received regulatory approval. This review outlines the current landscape of IRD gene therapy, including trials for several forms of IRD including achromatopsia, choroideremia, Leber congenital amaurosis, X-linked retinitis pigmentosa, and X-linked retinoschisis. We highlight the central challenges facing the field: narrow gene- or variant-specific indications, vector limitations, and reliance on suboptimal clinical trial endpoints. The review also discusses emerging strategies - including dual AAV and split-intein vectors, non-viral delivery platforms, and precision gene editing technologies such as CRISPR, base editing, and prime editing. These innovations promise to expand therapeutic reach. Finally, we emphasize the need for improved regulatory frameworks and ethical considerations for gene-based therapies for IRD. The field now stands at a critical juncture, where the lessons of Luxturna can inform a more scalable, inclusive, and transformative future. RPE65

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Current progress and challenges in gene therapy for inherited eye diseases

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