Inherited retinal dystrophies

Recessive inherited retinal dystrophies are ideal targets for gene therapy

HORAMA is developing rAAV-based gene therapy products in the area of inherited ophthalmological diseases. Our targets are inherited, monogenic orphan diseases with no existing treatment that ultimately lead to total loss of vision.

These inherited retinal dystrophies are rare ophthalmic pathologies with two main kinds of dystrophies depending of the mutated gene: the Retinopathies (+ 3.5 million of patients in the world) and the Maculodystrophies ( + 1.6 million of patients in the world)

Taken together, they account for a large number of patients but each pathology is linked to a unique gene and is rare.



Difficulties in dim light, visual field constriction leading to difficulties in daily activities (e.g. retinitis pigmentosa, Leber congenital amaurosis…)


Retinitis pigmentosa
> 60% of cases

Leber congenital amaurosis
> 5% of cases



Decrease in visual acuity,
difficulties to read
(e.g. Stargardt disease)


Stargardt Disease

of cases