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Article

Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial.

Nature communications - 10 Sept 2024

Kvanta Anders, Rangaswamy Nalini, Holopigian Karen, Watters Christine, Jennings Nicki, Liew Melissa S H, Bigelow Chad, Grosskreutz Cynthia, Burstedt Marie, Venkataraman Abinaya, Westman Sofie, Geirsdottir Asbjörg, Stasi Kalliopi, André Helder

Abstract excerpt

Gene therapy holds promise for treatment of inherited retinal dystrophies, a group of rare genetic disorders characterized by severe loss of vision. Here, we report up to 3-year pre-specified interim safety and efficacy results of an open-label first-in-human dose-escalation phase 1/2 gene therapy clinical trial in 12 patients with retinal dystrophy caused by biallelic mutations in the retinaldehyde-binding...

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Identifiers

PMID: 39256350
DOI: 10.1038/s41467-024-51575-4
PMCID: PMC11387776

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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