RPQure for Retinitis Pigmentosa and Leber congenital amaurosis due to CRB1 mutations

RPQure is a team of researchers at the Leiden University Medical Center (LUMC) funded by the Foundation Fighting Blindness USA; "Uitzicht" Blindness Foundations NL and Rotterdamse Stichting Blindenbelangen and Stichting Blindenhulp, The Netherlands Organization for Health Research and Development (ZonMw), and Curing Retinal Blindness Foundation (CRBF) USA. Previously funded by the European Union Framework 7 Program "CRUMBS IN SIGHT" and Royal Netherlands Academy of Arts and Sciences (KNAW).

RPQure initially focuses on developing preclinical and clinical testing along with registering an AAV.CRB1 retinal gene therapy for juvenile retinitis pigmentosa and Leber congenital amaurosis, and macular degeneration in young adults, in the US and Europe. RPQure is searching for investors and grants for the pharmaceutical and clinical phase experiments.

Retinitis pigmentosa and Leber congital amaurosis due to mutations in the CRB1 gene are rare inherited causes of blindness that affect up to 80.000 people worldwide. The number of patients affected from their mid-20s to early-30s by macular degeneration due to mutations in the CRB1 gene is to be determined.