AAV-CNGB3, a gene therapy treatment designed to restore cone function, is delivered via subretinal injection to the area of the eye where most of the cones in the retina are located. MeiraGTx is currently conducting a Phase 1/2 clinical trial of AAV-CNGB3 in both adult and pediatric patients with achromatopsia (ACHM) due to mutations in the CNGB3 gene.

AAV-CNGB3 was granted Orphan Drug, Rare Pediatric Disease and Fast Track designations by the FDA, and Orphan Drug and PRIME designations by the EMA, for the treatment of ACHM caused by mutations in the CNGB3 gene.