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.
To preserve or improve patient autonomy
& reduce the life-long financial burden on society.
HORAMA develops innovative gene therapy products for rare ophthalmic diseases to preserve/restore visual autonomy in affected patients. The objective of treatment is to delay or halt disease progression and to preserve or improve visual function by providing treatment in early disease stages.
HORAMA is focused on developing recombinant adeno-associated virus (rAAV) vectors for the treatment of currently incurable blinding diseases.
A lead program in clinical trials and a growing
pipeline of novel approaches
HORAMA is developing a novel gene therapy approach targeting Retinitis pigmentosa caused by PDE6B mutations. The program is currently being evaluated in a phase I/II clinical study with top-line results from the trial expected in 2021. Mutations in the CRB1 gene can cause early onset and delayed onset Inherited Retinal Dystrophy.