Gene therapy cotoretigene toliparvovec for treatment of X-linked retinitis pigmentosa fails to meet primary endpoint in phase II/III study

XIRIUS study (n=18 phase I to establish dose; additional 23 enrolled in phase II/III for efficacy/safety; age > 10 years) showed no significant improvement over the control group in changes in primary endpoint of retinal sensitivity at one year using a macular integrity test.

Source:

PharmaTimes