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HGI-004

Development stage: Preclinical
Lead developer: Shenzhen Hemogen Gene
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravenous

Overview

HGI-004 is an investigational gene therapy being developed by Shenzhen Hemogen for the treatment of Wiskott-Aldrich Syndrome (WAS). WAS is a rare X-linked primary immunodeficiency caused by mutations in the *WAS* gene, which encodes the Wiskott-Aldrich syndrome protein (WASp), a key regulator of the actin cytoskeleton in hematopoietic cells. HGI-004 aims to restore functional WASp expression in the patient's cells, thereby correcting the immune and platelet defects associated with the disease. The program is currently in the preclinical stage of development.

Targets

WAS (Wiskott-Aldrich syndrome protein)

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