HORAMA is one of the key players in the field of ophthalmic gene therapy.
HORAMA develops new gene therapies for rare inherited ocular diseases to improve or restore patient autonomy and reduce healthcare costs for society. The objective of replacement gene therapy is to provide a non-mutated copy of the causative mutated gene.
HORAMA approach is based on vector technology using non-pathogenic, recombinant adeno-associated viruses (rAAV). The rAAV carrying the therapeutic gene of interest penetrates the target retinal cells, inducing the expression of the functional protein. This restores normal cell function, prevents further retinal deterioration, and improves or restores visual function.