First-in-human interventional study of rAAV8.hRKp.AIPL1 to treat children with AIPL1-associated severe retinal dystrophy

Status: Recruiting

Phase of Trial: Phase I

Latest Information Update: 21 Mar 2025

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At a glance

21 Mar 2025 New trial record
21 Feb 2025 According to a MeiraGTx media release, Following recent meetings with the MHRA, the Company intends to submit a Marketing Authorization Application (MAA) under exceptional circumstances for rAAV8.hRKp.AIPL1 based on the results from the 11 treated children with no further clinical data required, and the Company has aligned on an expedited CMC package to support approval.
21 Feb 2025 According to a MeiraGTx media release, data from this study were published in the Lancet.

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