tgAAG76

tgAAG76 is an adeno-associated virus (serotype 2) delivered gene therapy designed for the potential treatment of vision loss caused by RPE65 gene abnormalities, specifically targeting inherited retinal diseases such as Leber congenital amaurosis type 2. The therapy uses a recombinant adeno-associated virus vector containing the human RPE65 gene under the control of its native promoter and a bovine growth hormone polyadenylation signal. It is administered via subretinal injection to deliver functional copies of the RPE65 gene to retinal cells, aiming to restore visual function in patients with biallelic mutations in this gene. The drug was developed jointly by Targeted Genetics, Moorfields Eye Hospital, and University of London[1][2][3][5].

Other names

rAAV 2/2.hRPE65p.hRPE65