HORAMA aims to be a key player in the field of
ophthalmic gene therapy
HORAMA develops new gene therapies as treatments for rare inherited ocular diseases to improve or restore patient visual capabilities, autonomy, and reduce healthcare costs for society.
HORAMA’s approach is based on vector technology using non-pathogenic, recombinant adeno-associated viruses (rAAV) to provide a functional gene to replace the function of mutated, malfunctioning genes. The rAAV carrying the therapeutic gene of interest penetrates target retinal cells, inducing the expression of a functional protein. This restores normal cell function, prevents further retinal deterioration, and has the potential to improve visual function.
HORAMA has developed and optimized proprietary manufacturing processes in collaboration with its academic partners
The HORAMA platform can support a pipeline of innovative gene therapy products for several ocular diseases.
HORAMA has signed exclusive licensing agreements on several patent applications and on non-clinical and clinical (Phase I/II) know-how. In addition, HORAMA has been granted Orphan Drug Designations for HORA-PDE6B, providing incentives from health authorities and market exclusivity of 7 to 10 years.