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IC-100

Development stage: Preclinical
Lead developer: Astellas Pharma
Modality: Gene Therapies

Overview

IC-100 is a preclinical gene therapy candidate developed by IVERIC bio (now a wholly-owned subsidiary of Astellas Pharma) for the treatment of Leber congenital amaurosis type 10 (LCA10). LCA10 is an inherited retinal disease caused by mutations in the CEP290 gene. IC-100 is an adeno-associated virus (AAV) minigene therapy designed to address these genetic mutations. The program originated from an exclusive global license agreement with the University of Massachusetts.

Targets

CEP290 (Centrosomal protein 290)

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