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CGD-Grans
- Development stage
- Unknown
- Lead developer
- National Institute of Allergy and Infectious Diseases
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Cell Therapies
- Administration
- Intravenous
01
Overview
CGD-Grans is an experimental autologous cell therapy developed by the National Institute of Allergy and Infectious Diseases (NIAID) for the treatment of X-linked chronic granulomatous disease (CGD). The therapy involves collecting a patient's own granulocytes via apheresis and transfecting them ex vivo with messenger RNA (mRNA) encoding functional gp91phox, a critical component of the NADPH oxidase enzyme complex. This process temporarily restores the cells' ability to produce microbicidal oxidants, enabling them to kill bacteria and fungi. Unlike permanent gene therapies that modify hematopoietic stem cells, CGD-Grans provides a transient correction of the oxidase defect, intended to treat severe, refractory infections in patients with CGD without the need for HLA-matched donor granulocyte transfusions.
- Other names
- mRNA-transfected CGD-GransmRNA-transfected granulocyte-enriched cells
02
Targets
NOX2 (NADPH oxidase 2)
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