IKI-001

IKI-001 (formerly known as IKAR-001) is a novel, bicistronic adeno-associated virus (AAV) gene therapy candidate being developed by Ikarovec for the treatment of geographic atrophy (GA), an advanced form of dry age-related macular degeneration (AMD). The therapy utilizes an AAV2 vector to deliver two therapeutic genes simultaneously: one encoding a complement regulatory protein (such as Complement decay-accelerating factor, CD55) to modulate the overactive complement cascade, and another encoding a neurotrophic factor (such as Pigment epithelium-derived factor, PEDF) to provide neuroprotection to retinal cells. By addressing both the inflammatory complement system and the survival of retinal pigment epithelium and photoreceptor cells, IKI-001 aims to slow the progression of retinal lesions and preserve vision in patients with GA. The program is currently in preclinical development.