MeiraGTx Announces The Lancet Publication of Data Demonstrating the Efficacy of rAAV8.hRKp.AIPL1 for the Treatment of Leber Congenital Amaurosis 4 (LCA4) Retinal Dystrophy

LONDON and NEW YORK, Feb. 21, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the publication of results from the first-in-human interventional study to treat children with AIPL1-associated severe retinal dystrophy. The data were published in The Lancet in a paper titled, "Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study". This paper presents data from the first 4 children treated unilaterally with rAAV8.hRKp.AIPL1. A further 7 children with LCA4 were treated bilaterally with rAAV8.hRKp.AIPL1 demonstrating similarly remarkable effects on vision, with all 11 of the 11 children treated who were blind at birth now having visual acuity in the treated eyes.

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