The news published today is a significant milestone in the clinical development focusing on LCA10.
LCA is the most common cause of blindness due to genetic disease in children. It consists of a group of diseases of which LCA10 is the most frequent and one of the most severe forms. LCA10 is caused by mutations in the CEP290 gene and leads to early loss of vision causing most people to lose their sight in the first few years of life.
There are currently no approved treatments for people and families living with LCA10, and our research has the potential to bring forward the first approved therapy to address this high unmet medical need.