Looking to the Future of Viral Vectors in Ocular Gene Therapy: Clinical Review.

Biomedicines · 2025 · PMC11852528 · PMID 40002778

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a positive trendno p-value reported

Despite this, according to the results of a clinical trial of gene therapy for X-linked RP [ NCT03116113 ] cotoretigene toliparvovec (BIIB112, AAV8-NSR-RPGR), the study did not meet the primary endpoint, but a positive trend was observed for several clinically relevant secondary endpoints.

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