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ICM-403

Development stage: Preclinical
Lead developer: ICM
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies

Overview

ICM-403 is an adeno-associated virus (AAV)-based gene therapy developed by ICM Co., Ltd. for the treatment of congenital hearing loss. It specifically targets mutations in the *SLC26A4* gene, which encodes the protein Pendrin. Pendrin mutations are a leading cause of hereditary sensorineural hearing loss, particularly in East Asian populations, and are associated with Pendred syndrome and enlarged vestibular aqueduct (EVA). The therapy aims to deliver a functional copy of the *SLC26A4* gene to the inner ear to restore Pendrin function and improve hearing outcomes. It is currently in preclinical development and has completed small-scale production for research purposes.

Other names: ICM-403 AAV gene therapyICM403 AAV gene therapyICM 403 AAV gene therapy

Targets

Neuraminidase

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