AGTC-401

AGTC‑401 is a recombinant adeno-associated virus (AAV) vector-based gene therapy designed to deliver a functional copy of the cyclic nucleotide-gated cation channel beta 3 (CNGB3) gene to retinal cells in patients with achromatopsia caused by mutations in the CNGB3 gene[1][2][4]. The therapy aims to restore or improve visual function by enabling expression of the normal CNGB3 protein, which is essential for cone photoreceptor function in the retina[4]. It was developed initially by Applied Genetic Technologies Corporation and later Beacon Therapeutics, targeting rare inherited retinal disorders such as achromatopsia (ACHMB3)[2][4]. Clinical studies have shown improvements in visual sensitivity and a favorable safety profile, but development has been discontinued as of 2024[7].

Other names

CNGB3 gene therapyCNGB-3 gene therapyCNGB 3 gene therapyAAV8-CNGB3AAV-8-CNGB3AAV 8-CNGB3