AGTC-402 (Applied Genetic Technologies Corporation)

**AGTC-402** is an investigational **AAV8-based gene therapy** developed by **Applied Genetic Technologies Corporation** for **CNGB3-associated achromatopsia**. It uses a **human cone arrestin promoter** to drive retinal expression of a functional **CNGB3** transgene in cone photoreceptors after **subretinal administration**, with the goal of restoring cone cyclic nucleotide-gated channel function and improving cone-mediated vision. The program was studied in Phase 1/2 adult and pediatric trials, where reported findings indicated biologic activity such as improvements in visual sensitivity and reduced light discomfort with an acceptable safety profile.

Other names

ACHM B3 gene therapy