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IKAR-003

Development stage: Preclinical
Lead developer: ikarovec
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravitreal

Overview

IKAR-003 is a gene therapy candidate developed by Ikarovec for the treatment of intermediate age-related macular degeneration (AMD). It leverages VectorBuilder's advanced AAV capsid technology for its delivery, which has demonstrated superior efficacy in non-human primate studies by achieving broad and robust transduction of retinal cells. The goal is to enable a minimally invasive, office-based intravitreal administration, improving patient access.

Targets

C3 convertase (Ficolin-3)PEDF (Pigment epithelium-derived factor)

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