NSR-ABCA4

NSR-ABCA4 is an adeno-associated virus (AAV)-based gene therapy candidate designed to deliver a functional copy of the human ABCA4 gene to retinal cells for the treatment of Stargardt disease, an inherited macular dystrophy caused by biallelic loss-of-function mutations in ABCA4. Nightstar Therapeutics developed NSR-ABCA4 as part of its retinal gene therapy pipeline prior to its acquisition by Biogen, with the goal of restoring ABCA4 transporter function in photoreceptors and reducing the accumulation of toxic bisretinoids such as lipofuscin in the retinal pigment epithelium, thereby slowing or preventing progressive vision loss.[4][6][10]