Clinical Trials

Our Mission is to Restore Vision for People with Retinal Degeneration

Retinitis Pigmentosa: Observational Study

RayTx established The Vision Research and Assessment Institute (VRAI) with the express purpose of serving as a testing facility for efficacy endpoints for patients with Low Vision.

The mission of the VRAI is to enable the highest quality, standardized efficacy testing of patients with visual impairment to be performed by expert clinicians in an optimized environment.

The proposed assessments or testing modalities to be evaluated in this non-interventional study are published in some capacity, and some of these publications already include patients with severe-to-profound vision loss from retinitis pigmentosa and other inherited retinal diseases.

RTx-015 for Retinitis Pigmentosa: Phase 1

This Phase 1, open-label, non-randomized, multicenter clinical trial is to evaluate the safety and preliminary efficacy of a single, uniocular intravitreal injection of an investigational optogenetic gene therapy, RTx-015, in patients with retinitis pigmentosa.

Up to 3 dose cohorts are planned, and each cohort will consist initially of 3 patients. Eligible patients will be assigned to a dose cohort by sequential enrollment.

Retinitis Pigmentosa

Retinitis pigmentosa (RP), is a genetic disease in which the photoreceptors gradually degenerate resulting in complete, or nearly complete blindness for most patients.

The symptoms of RP include night blindness, reduced visual fields, and eventual loss of visual acuity. Patients are typically diagnosed in the first decades of life. More than half a million people are probably affected by RP worldwide. At present, no effective treatment is available for RP.

For more information, we recommend these excellent resources:

Inherited Retinal Degenerations

These retinal diseases are like RP because the photoreceptors gradually degenerate but different genes are frequently involved, different patterns of progression occur and in some instances your ophthalmologist may recognize different appearances to the retina.

Examples of inherited retinal degenerations that could be treated by optogenetics include Stargardt macular dystrophy, choroideremia, cone-rod dystrophy and Leber congenital amaurosis.

For more information, we recommend these excellent resources: