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NY-ESO-1 (New York Esophageal Squamous cell carcinOma 1) is a highly validated tumor target neoantigen with aberrant expression in numerous cancer cell types, including myxoid/round cell liposarcoma (89%), Neuroblastoma (82%), Synovial Sarcoma (80%) and Multiple Myeloma (60%). Its ability to elicit spontaneous humoral and cellular immune responses, together with its restricted expression pattern, have established it as a compelling target for cancer immunotherapy.

PRAME (Preferentially Expressed Antigen in Melanoma) is a cancer-testis antigen that is normally expressed in the testis but overexpressed in various cancer types, including melanoma, leukemia, and breast cancer. As a neoantigen, PRAME can elicit an immune response when presented on the surface of cancer cells in the context of major histocompatibility complex (MHC) molecules.

Stargardt disease is a genetic eye disease that causes retinal degeneration and vision loss. It is the most common form of inherited macular degeneration, affecting about 30,000 people in the U.S. The progressive vision loss is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.

Retinitis pigmentosa is a group of rare eye diseases that affect the retina. RP causes progressive disfunction of rod photoreceptors with subsequent degeneration of cone photoreceptors and the retinal pigment epithelium, resulting in vision loss. Symptoms start in childhood and most individuals eventually lose the majority of their sight.

Usher 1B is a genetic disorder characterized by hearing loss, deafness and progressive vision loss.