EDIT-101 is designed to treat inherited retinal degeneration caused by a specific genetic mutation in the CEP290 gene, known as the IVS26 variant responsible for Leber Congenital Amaurosis type 10 (LCA10) (Maeder et al., 2019, p. 10; Leroy et al., 2021). Here, CEP290 is linked to Leber congenital amaurosis 10.