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Posts tagged ProQR

Clinical Trial Authorized in the U.S. for Emerging LCA 10 Therapy

ProQR, a biotechnology company in the Netherlands, has received authorization from the U.S. Food and Drug Administration to start a Phase I/II clinical trial for its therapy known as QR-110, which is being developed for Leber congenital amaurosis type 10 (LCA 10). The genetic retinal condition causes severe vision loss in children. QR-110 targets the specific mutation p.Cys998X in the CEP290 gene, also known as c.2991+1655A>G mutation. It is estimated that it affects about 2,000 people in the Western world.

QR-110 was a featured topic at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore, May 7-11. About 12,000 eye researchers and industry professionals attended the event.
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