HORAMA is a new leader in the field of gene therapy in ophthalmology.
HORAMA is developing new gene therapies in the field of rare ophthalmological diseases to restore patients’ autonomy & reduce costs for the community. The aim of gene therapy for the treatment of rare genetic diseases is to provide a healthy copy of the disease-causing mutated gene in order to restore the missing function and hence stop disease evolution.
We are developing our own gene therapy approach based on vector technology using non-pathogenic, recombinant adeno-associated viruses (rAAV). The rAAV bearing the therapeutic gene of interest is administered into retinal cells where it expresses its encoded healthy protein, thus restoring normal function and preventing further deterioration of the retina.
We have our proprietary manufacturing processes set up with our academic partners. Our platform develops a solid pipeline of innovative gene therapy medicinal products for several applications in the field of retinal diseases.
HORAMA benefits from exclusive licensing agreements on several patent applications relating to either rAAV technologies or to results obtained from preclinical or clinical (Phase 1/2) results. Our specific patented know-how, added to our Orphan Drug Designations, confirms our ability to bring new drugs to the market.