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Gene Therapy Dedicated to Rare Opthalmology Pathologies.

At HORAMA, we believe in gene therapy to cure a broad range of inherited disorders. Our initial focus is on rare retinal dystrophies with our lead clinical program targeting patients with PDE6B gene mutations that lead to progressive loss of vision in children and adults ultimately leading to blindness. Our team is pushing the boundaries of gene therapy by advancing next-generation delivery platforms that will improve the effectiveness and coverage of gene transfer to address multiple diseases.

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