Achromatopsia

Achromatopsia is an inherited retinal condition causing extreme light sensitivity (i.e., day blindness), as well as reduced visual acuity and color discrimination. Achromatopsia is caused by mutations in any of several genes. The most common genes associated with the condition are the CNGB3 and CNGA3 genes — mutations in these cause about 75 percent of cases.

Applied Genetic Technologies Corporation (AGTC) is currently conducting a clinical study at five clinical centers in the U.S. to evaluate the safety and efficacy of an investigational gene therapy in patients with achromatopsia caused by mutations in the CNGB3 gene.

AGTC is also conducting a clinical study at five clinical centers in the U.S. and one in Israel to evaluate the safety and efficacy of an investigational gene therapy in patients with achromatopsia caused by mutations in the CNGA3 gene.