ProQR Therapeutics N.V., a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, announced the first patient dosed in the Phase 2/3 ILLUMINATE clinical trial for sepofarsen (formerly named QR-110) in patients with Leber’s Congenital Amaurosis 10 (LCA10). Data from the study are expected at year-end 2020.
“Inherited retinal degenerations caused by mutations in the CEP290 gene frequently lead to blindness in early childhood. No form of therapy is currently available,” said Dr. Katarina Stingl of the Center for Ophthalmology in Tübingen, Investigator in the ILLUMINATE trial. “ProQR’s RNA therapy for the p.Cys998X mutation showed the potential to improve vision in an earlier trial, which is encouraging and of great importance to the affected patients.”
“The start of the ILLUMINATE trial marks an important milestone towards our goal of bringing this novel and most advanced therapy for LCA10 to patients,” commented Dr. Aniz Girach, Ophthalmologist and Chief Medical Officer of ProQR. “With the ongoing trials in LCA10 and Ushers syndrome type 2, and the start of our clinical program for adRP later this year, we are breaking new ground and paving the way for new treatments in multiple severe inherited retinal diseases.”
LCA10 is the leading genetic cause of blindness in childhood and currently there is no approved treatment available to patients. Sepofarsen targets the most common p.Cys998X mutation in CEP290 that affects approximately 2,000 people in the Western world.