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Stargardt disease, the most prevalent inherited retinal disease (IRD), affects approximately 1 in 8,000 to 10,000 individuals worldwide. Caused by mutations in the ABCA4 gene, Stargardt disease, along with other ABCA4-linked retinal dystrophies, leads to a gradual decline in visual acuity and, in some cases, legal blindness. Currently, there are no approved treatments to halt disease progression, highlighting a critical unmet medical need for effective therapies.

Retinitis pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. There are currently no approved treatments for RP. RP initially presents as nighttime blindness during childhood or early adulthood, progressing to peripheral visual field loss and “tunnel vision,” central visual impairment, reduced visual acuity and, ultimately, complete blindness. Retinitis pigmentosa is the most common type of inherited retinal diseases (IRDs). It is estimated to affect 1 in 3,500 to 1 in 4,000 people in the United States and Europe, respectively.

Age-related macular degeneration is the major cause of irreversible vision loss among elderly and affects currently 30 million individuals worldwide. The disease can manifest in a dry (atrophic) and wet (neovascular) form. AMD affects a small area near the center of the retina, the macula, which is responsible for central vision and is needed for detailed tasks such as reading, driving, and recognizing faces. Peripheral vision and night vision are generally not affected in AMD, but slow adjustment of vision to darkness and reduced dim light vision often occur in the early stages of the disease.