MeiraGTx(MGTX) Newsfilter·2025-02-21 11:30Core Insights - MeiraGTx Holdings plc announced positive results from a first-in-human interventional study using rAAV8.hRKp.AIPL1 to treat children with AIPL1-associated severe retinal dystrophy, showing significant improvements in visual acuity and functional vision [1][2][5] Study Details - The study involved 11 children, with 4 treated unilaterally and 7 treated bilaterally, all of whom were legally blind at birth and showed improved visual acuity after treatment [1][5][6] - The treatment was administered via subretinal injection, and the outcomes were measured using various methods including visual acuity tests and retinal imaging [3][4] Results - Prior to treatment, children's visual acuities were limited to light perception, but post-treatment, the mean visual acuity improved to 0.9 LogMAR from an equivalent of 2.7 LogMAR [4] - All treated children demonstrated enhanced visual function and structural preservation of the retina, with no serious adverse effects reported [2][4][6] Regulatory Pathway - Following the positive results, MeiraGTx is working with global regulators for expedited approval of rAAV8.hRKp.AIPL1, intending to submit a Marketing Authorization Application (MAA) based on the current data without the need for further clinical trials [7][8] Company Overview - MeiraGTx is a clinical-stage genetic medicines company with a focus on developing treatments for severe genetic conditions, including AIPL1-associated retinal dystrophy, and has capabilities in viral vector production and gene therapy technologies [10]