… 2,500 patients in the United States, leading to blindness in mid-adulthood. There are no approved therapies … peripheral vision during childhood and frequently leads to blindness in mid adulthood. In the United States, the most …
Top-line data now expected to be announced in Q1 2022Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
… retinitis pigmentosa (adRP), a rare inherited form of blindness with no approved therapy A first in human Phase 1/2 … peripheral vision during childhood and frequently leads to blindness in mid adulthood. In the United States, the most …
QR-110 demonstrated rapid and sustained improvement in vision in the majority of subjects, as measured by visual acuity and mobility course
QR-110 was well-tolerated with no serious adverse events
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by tra