AGTC-402

AGTC-402 is a recombinant adeno-associated virus (AAV) vector gene therapy designed to deliver the human CNGA3 gene, which encodes the cyclic nucleotide gated channel alpha subunit, to retinal cells. It was developed as a potential treatment for achromatopsia caused by mutations in the CNGA3 gene, an autosomal recessive disorder leading to cone dysfunction and severe visual impairment. The therapy is administered via subretinal injection and aims to restore function by providing a functional copy of the defective gene[1][2][5]. Preclinical studies demonstrated improved vision in animal models, but clinical trials did not show significant clinical improvements in patients with CNGA3 mutations[5][6]. As of early 2022, further development has been discontinued due to lack of efficacy signals[6][7].

Other names

recombinant Adeno-associated virus vector expressing CNGA3