Daniel de Boer, Founder and CEO of ProQR, provides an overview of his company. ProQR’s focus is on diseases that are very severe or life threatening, and have limited treatment options. The company is developing new therapies for several forms of inherited blindness such as Leber’s congenital amaurosis and Usher syndrome, cystic fibrosis, and a debilitating skin disease called dystrophic epidermolysis bullosa.
Mr. de Boer also explains Leber’s congenital amaurosis Type 10 (LCA 10), a genetic eye disorder and the leading genetic cause of childhood blindness. ProQR is developing a therapy for patients that suffer from LCA 10 due to the p.Cys998X mutation in the CEP290 gene.