MeiraGTx Holdings plc announced the publication of results from its first-in-human interventional study of rAAV8.hRKp.AIPL1 in The Lancet. The study evaluated the gene therapy for AIPL1-associated severe retinal dystrophy, also known as Leber congenital amaurosis 4 (LCA4).
The published data demonstrated substantial benefits in visual acuity, functional vision, and protection against retinal degeneration in all 11 treated children. All 11 children, who were blind at birth, gained visual acuity in their treated eyes, with visual acuity improving to a mean of 0.9 logarithm of the minimal angle of resolution (LogMAR) from an equivalent of 2.7 LogMAR before intervention.
Based on these results, MeiraGTx is preparing to submit a Marketing Authorization Application (MAA) under exceptional circumstances with the UK's Medicines and Healthcare products Regulatory Authority (MHRA), with no further clinical data required. The company is also engaging with the Food and Drug Administration (FDA) to discuss a potentially similar expedited pathway for approval in the United States. The program has previously received Orphan Drug and Rare Pediatric Disease Designations from the FDA.
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