AAV-RPGR is designed to treat the most common form of X-linked retinitis pigmentosa (XLRP) caused by mutations in the eye-specific form of the RPGR gene called RPGR open reading frame 15 (RPGR ORF15). Both rods and cones photoreceptors require RPGR ORF15 to function.
We are currently conducting an ongoing Phase 1/2 clinical trial of AAV-RPGR in adult and pediatric patients, and expect to progress AAV-RPGR into the Phase 3 Lumeos clinical trial. In 2020, MeiraGTx announced six, nine, and 12-month results from the dose escalation phase of the Phase 1/2 trial, with data demonstrating statistically significant improvement in retinal sensitivity and vision-guided mobility.
AAV-RPGR has received Fast Track and Orphan Drug designations from the FDA, as well as PRIME, ATMP and Orphan Medicinal Product designations from the EMA.