HORA-CRB1 is in early-stage development for eye diseases caused by defects in the CRB1 gene

HORA-CRB1 is an adeno-associated viral (AAV) gene therapy vector containing a gene for the treatment of mutations in the CRB1 gene. These mutations are reported as a cause of Retinitis Pigmentosa, Lebers Congenital Amaurosis (LCA) and other eye disorders. Horama in-licensed this program in 2019 from its collaboration partners at the Department of Ophthalmology, LUMC, Leiden.