ProQR, a biotech company in the Netherlands, ha s reported vision improvements for patients in a Phase 1/2 clinical trial for QR-110 , a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world. The company reported that 60 percent of su bjects in the trial demonstrated improvements in visual acuity and their ability to navigate a mobility course . The treatment was also safe for patients. As a result of the encouraging interim results, ProQR has concluded the Phase 1/2 trial and plans to move the treatment into a Phase 2/3 clinical trial. Ten people were treated in the Phase 1/2 study. Results from the interim analy sis were presented on September 5, 2018, at the Retinal Degeneration 2018 meeting in Killarney, Ireland , by principal investigator Artur Cideciyan , PhD , research professor of ophthalmology at the Scheie Eye Ins...
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