Biography
Chief Supervisor
Project Title
Gene therapy targeting C3b/C4b and VEGF using chimpanzee adenovirus vector for dry and neovascular age-related macular degeneration
Synopsis
Age-related macular degeneration (AMD) is the leading cause of vision loss among the elderly people over 55 years old. Clinically, it is classified as early-stage (dry AMD) to late-stage (neovascular AMD, nAMD). Dry AMD is characterized by drusen, and may progress to neovascular AMD, therefore leading to choroidal neovascularization (CNV) and severe vision loss.
AMD is a multifactorial disease, and vascular endothelial growth factor (VEGF) and complement system dysregulation have been indicated to play a vital role in the progression of AMD. Nowadays, gene therapy offers a sustained delivery of foreign genes and has been widely used in various diseases. In this study, chimpanzee adenovirus vectors (AdCs) will be used owing to their large capacity, simple production process, and a relative lower level of neutralizing antibodies in humans.
Efdamrofusp alfa (code: IBI302) is a novel bispecific decoy receptor fusion protein, which is designed to inhibit the activation of complement system by combining to C3b/C4b and meanwhile neutralize numerous isoforms of the VEGF family. In this study, we will use the chimpanzee adenovirus vector that carries efdamrofusp alfa, and test its therapeutic effect on both dry and neovascular AMD animal models.