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EN-374
- Development stage
- Preclinical
- Lead developer
- Ensoma
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
EN-374 is an investigational gene therapy developed by Ensoma for the treatment of X-linked chronic granulomatous disease (X-CGD). The therapy delivers a functional CYBB gene directly to hematopoietic stem cells in vivo, using a promoter for selective expression in neutrophils—the primary cell type affected by X-CGD. By restoring CYBB function, EN-374 aims to enable patients' immune systems to effectively fight infections. The program has received rare pediatric disease and orphan drug designations from the FDA and is expected to enter clinical trials following an investigational new drug application submission planned for 2025[1].
02
Targets
NOX2 (NADPH oxidase 2)
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