At 12 months, data from the phase 1/2 study of AAV5-RPGR gene therapy for RPGR-associated X-linked retinitis pigmentosa showed significant improvement in retinal sensitivity and functional mobility with the low and intermediate doses.
“Given the robust safety and efficacy signals observed, these doses are being further explored with analyses at additional data time points in the ongoing randomized controlled dose-expansion phase of the study,” Michel Michaelides, MD, said at the virtual American Academy of Ophthalmology annual meeting.
In the low- and intermediate-dose cohorts,
Uncategorized