AAV-CIB2 gene therapy

AAV-CIB2 gene therapy is an adeno-associated virus (AAV)-based gene augmentation therapy designed for the treatment of dry age-related macular degeneration (dry-AMD) and geographic atrophy (GA). Developed by researchers at the University of Maryland, the therapy utilizes AAV vectors (such as Anc80L65 and AAV2.7m8) to deliver a functional human CIB2 (calcium and integrin binding protein 2) gene to the retinal pigment epithelium (RPE). CIB2 serves as a critical regulator of autophagy by negatively regulating the mTORC1 signaling pathway through its interaction with inactive GDP-loaded Rheb. By restoring CIB2 levels, the therapy aims to enhance autophagic clearance of sub-RPE deposits (drusen), reduce lipid and protein accumulation, and preserve visual function. Preclinical studies have demonstrated that subretinal or intravitreal administration of AAV-CIB2 can rescue electroretinogram (ERG) responses and improve visual acuity in animal models of dry-AMD.

Other names

hCIB2 gene therapyhCIB-2 gene therapyhCIB 2 gene therapyCIB2 gene deliveryCIB-2 gene deliveryCIB 2 gene delivery