X-Linked Retinoschisis (visit patient site)

X-linked retinoschisis (XLRS) is an inherited form of retinal degeneration affecting young males. Patients present with poor vision either in infancy or at school age. Visual acuity usually worsens during the teenage years and then stabilizes until complicated by vitreous hemorrhage or retinal detachment during adulthood. There is no treatment available for the retinal degeneration in XLRS.

In animal models of XLRS, treatment with a gene therapy product leads to progressive and long-term improvement in retinal function and prevention of retinal cell degeneration. Based on the strong preclinical proof-of-concept data, AGTC has initiated a formal preclinical testing program that will lead to clinical evaluation of this product.

Achromatopsia (visit patient site)

Complete achromatopsia is an inherited condition that is associated with color blindness, visual acuity loss and extreme light sensitivity resulting in daytime blindness. There is no specific treatment for achromatopsia, although deep red tinted spectacles or contact lenses can reduce symptoms of light sensitivity.

In animal models of achromatopsia, treatment with a gene therapy product restored daylight visual function in affected dogs with daytime blindness. Based on the strong preclinical proof-of-concept data, AGTC has initiated a formal preclinical testing program that will lead to clinical evaluation of this product.