January 7, 2019
Dutch ProQR Therapeutics OKd by FDA to take sepofarsen (QR-110) into the next phase of development
A Dutch maker of of transformative RNA medicines for the treatment of severe genetic rare diseases, ProQR Therapeutics, has got the U.S. Food and Drug Administration (FDA) tips for a Phase 2/3 pivotal trial Illuminate for sepofarsen, in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation in the CEP290 gene, the leading genetic cause of childhood blindness.