rAAV2-CB-hRPE65

rAAV2-CB-hRPE65 is a recombinant adeno-associated virus serotype 2 (AAV2) vector-based gene therapy designed to deliver a functional copy of the human RPE65 gene to retinal pigment epithelial cells. The drug consists of a nonreplicating AAV vector containing cDNA encoding the human retinal pigment epithelium-specific protein hRPE65. It is administered via subretinal injection, typically in patients with inherited retinal degenerations such as Leber congenital amaurosis (LCA) caused by mutations in the RPE65 gene. The mechanism of action involves transducing target cells with the functional RPE65 gene, enabling restoration of visual cycle function and improvement or stabilization of vision. Clinical studies have shown that treatment can lead to improvements in visual acuity and field, particularly when administered at younger ages[1][2][5][6].

Other names

Leber congenital amaurosis gene therapy