Leber congenital amaurosis pipeline: Data Byte

The Leber congenital amaurosis pipeline includes at least 10 products in development targeting the most common genetic causes of the disease, including seven gene therapies, a gene editing candidate, an antisense oligonucleotide and a small molecule. 

There are at least 18 types of LCA characterized by different genetic mutations that disrupt retinal development and function, leading to early vision loss. Mutations in the GUCY2D, RPE65, CRB1 and CEP290 genes are the most common causes of the disorder, corresponding to LCA1, LCA2, LCA8 and LCA10...