Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.
L to R: Florence Allouche Ghrenassia, PharmD, President, SparingVision; Frédérique Vidal, French Minister of Higher Education, Research and Innovation; José-Alain Sahel, MD, Co-Founder, SparingVision and Fondation Voir & Entendre; David Brint, Chairman, Foundation Fighting Blindness; and Laure Reinhardt, Deputy CEO, Bpifrance
However, researchers identify a potentially powerful endpoint for evaluating emerging therapies in future studies.
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.