ProQR Doses First Patient in Phase I/II Trial for Leber’s Congenital Amaurosis 10

November 24, 2017: By Jon Swedien

ProQRProQR Therapeutics announced Nov. 13 that the first patient has been dosed in its Phase I/II open-label trial of QR-110, a proposed treatment for Leber’s congenital amaurosis 10 (LCA 10).

The trial will enroll about six adults and six children who have LCA 10 due to a p.Cys998X mutation in the CEP290 gene, ProQR said. Subjects will receive a dose of QR-110 every three months for a total of four doses in one eye.

The trial is being conducted at the University of Iowa in Iowa City, Iowa; the Scheie Eye Institute at the University of Pennsylvania in Philadelphia; and Ghent University Hospital in Ghent, Belgium.

LCA 10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation is the most common. It leads to early loss of vision, causing most people to lose their sight in the first few years of life. To date, there are no approved treatments or other products in clinical development that treat the underlying cause of the disease, ProQR said.

QR-110 is ProQR’s lead program in the ophthalmology pipeline. The pipeline also includes two programs for Usher syndrome, a program for Fuchs endothelial corneal dystrophy, and a program for Stargardt disease. QR-110 is ProQR’s second program to enter clinical development. It follows QR-010, which is being developed for the most common mutation causing cystic fibrosis.

Netherlands-based ProQR said at the end of Q3-2017 that it had $46.6 million on hand. The company said it raised about $20 million from an issuance of ordinary shares in November that will fund operations through potential clinical data readouts in three different programs.

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