HORA-001 is in early-stage development for eye diseases caused by defects in the CRB1 gene

HORA-001 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 major cause of early onset and delayed onset Inherited Retina Dystrophy.

Horama in-licensed this program in 2019 from its collaboration partners at the Department of Ophthalmology, LUMC, Leiden.