MGTX - MeiraGTx's gene therapy shows sustained benefit in inherited retinal disorder

MeiraGTx ([[MGTX]] +2.5%) has announced 12-month data from the ongoing Phase 1/2 trial of AAV-RPGR, an investigational gene therapy for the treatment of X-linked retinitis pigmentosa ((XLRP)). Data were presented at the American Academy of Ophthalmology.The primary endpoint of the trial is safety, with secondary endpoints assessing changes in visual function at pre-specified timepoints post-treatment.In the dose escalation phase of the trial, data at the 12-month time point demonstrated statistically significant improvement in retinal sensitivity in treated eyes in both the low (n=3) and intermediate (n=4) dose cohorts, with six of seven patients demonstrating improved or stable vision in the treated eye one year after treatment.Efficacy signals were observed at the first post-treatment assessments at three months, with improvements sustained or increased at 12 months.AAV-RPGR is well-tolerated till data, with no dose-limiting events occurred.MeiraGTx and development partner Janssen are preparing to initiate the pivotal Phase 3 Lumeos clinical trial of

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MeiraGTx's gene therapy shows sustained benefit in inherited retinal disorder