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IVB121

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

Overview

IVB121 is an adeno-associated virus (AAV)-based gene therapy candidate developed by InnoVec Pharmaceutical Technology for the treatment of Usher Syndrome type 2A (USH2A). USH2A is a rare genetic disorder caused by mutations in the USH2A gene, leading to combined hearing loss and progressive vision loss (retinitis pigmentosa). IVB121 aims to deliver a functional version of the USH2A gene or a therapeutic transgene to the target cells in the retina to restore protein function and prevent further sensory degradation. The program is currently in the preclinical stage of development.

Targets

USH2A (Usherin)

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