AGTC-002

AGTC-002 is an investigational adeno-associated virus (AAV) gene therapy developed for the treatment of achromatopsia (ACHM), a rare inherited retinal disorder characterized by severe color blindness, reduced visual acuity, and extreme light sensitivity due to mutations in cone photoreceptor genes. It utilizes a recombinant AAV2tYF vector (rAAV2tYF-PR1.7-hCNGA3) to deliver the full-length human CNGA3 gene, aiming to restore cone function by enabling expression of the cyclic nucleotide-gated channel alpha-3 subunit essential for phototransduction in cones. Administered via subretinal injection, early Phase 1/2 data indicated a favorable safety profile up to certain dose levels, though dose-limiting toxicities like intraocular inflammation were observed at the highest pediatric dose (3.2e12 vg/mL).