Ophthalmology – Gene therap – Ophthalmology – Gene therapy – AAV – PDE6B- RLBP1 – Clinical trials – Eye – Retinal distrophy

HORAMA, a clinical-stage biotech company, 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 (AAV) vectors for the treatment of currently incurable blinding diseases.
To date, the most advanced products in Horama’s portfolio are developed for the treatment of Retinis Pigmentosa :
HORA-PDE6B which is currently in Phase I/II
HORA-RLBP1 which will start a phase I/II in 2019