MeiraGTx and Janssen Pharmaceuticals have announced nine-month data from the ongoing phase I/II trial of their investigational gene therapy for X-linked retinitis pigmentosa (XLRP).
XLRP is a progressive condition that causes deterioration of the visual field and is the most severe form of retinitis pigmentosa – a group of inherited retinal diseases. The most frequent cause of XLRP is disease-causing variants in the RPGR gene, accounting for more than 70% of XLRP cases.
In nine-month data from the phase I/II trial, MeiraGTx and Janssen’s investigational gene therapy AAV-RPGR continued to demonstrate significant improvement in retinal sensitivity in treated eyes, across both the low and intermediate dose cohorts.
In addition, data from the assessment of vision-guided mobility carried out at the nine-month timepoint demonstrated a significant improvement in walk time, compared to baseline in treated eyes compared to untreated eyes in the low and intermediate dose cohorts.
Read more: http://www.pharmatimes.com/news/janssenmeiragtx_showcase_nine-month_data_for_sight_loss_gene_therapy_1352478