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Illuminate is a Phase 2/3 clinical trial of investigational RNA therapy sepofarsen that aims to restore vision loss in Leber congenital amaurosis 10.

The clinical trial named Brighten evaluates the safety and tolerability of investigational RNA therapy sepofarsen in children with Leber congenital amaurosis.

During a ProQR Expert Perspective call Dr. Girach (CMO at ProQR) discusses findings from an open label extension study of sepofarsen for LCA10 with Retinal Disease expert Dr. MacDonald (Prof Emer University of Alberta).

Top-line results showed the Illuminate study did not meet its primary endpoint or secondary endpoints. We will continue to analyze the data.

There are currently around 160 people working at ProQR, but that wasn’t always the case. When Hee Lam Chan joined in 2013, she was only employee number six. As a result, she has seen the company grow and develop.