HG004

HG004 is a novel, one-time gene replacement therapy that utilizes a recombinant adeno-associated viral vector serotype 9 (AAV9) to deliver the human RPE65 gene directly to the retinal pigment epithelium (RPE). It is designed to restore, treat, and prevent blindness in both children and adults with severe visual impairment or blindness due to inherited retinal disease caused by mutations in the RPE65 gene. The therapy is being developed by HuidaGene Therapeutics, and it aims to offer superior transduction efficiency and clinical durability compared to existing AAV2-based gene therapies such as Luxturna, with lower vector doses and reduced risk of immunogenicity or ocular adverse events. HG004 has received FDA orphan drug designation and Rare Pediatric Disease Designation for the treatment of RPE65 mutation-associated inherited retinal diseases, including Leber's congenital amaurosis and retinitis pigmentosa[1][2][3][7][8].

Other names

Adeno-associated viral vector serotype 9 containing human RPE65 geneAAV9-hRPE65AAV-9-hRPE65AAV 9-hRPE65Cholgene