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Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
- Development stage
- Phase 2
- Lead developer
- National Institute of Allergy and Infectious Diseases
- Modality
- Stem Cell Therapies → Cell Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47 is an investigational gene therapy product designed for the treatment of autosomal recessive chronic granulomatous disease (AR-CGD) caused by mutations in the NCF1 gene, which encodes the p47phox subunit of NADPH oxidase. The therapy involves collecting a patient's own hematopoietic stem/progenitor cells (CD34+), genetically modifying them ex vivo using a self-inactivating lentiviral vector (pCCLCHIM-p47) carrying a codon-optimized human NCF1 cDNA under control of a myeloid-specific promoter, and then reinfusing these corrected cells back into the patient. The modified stem cells engraft and differentiate to produce functional phagocytes capable of restoring NADPH oxidase activity, thereby improving pathogen killing and reducing infection risk in CGD patients. This approach offers an alternative to allogeneic hematopoietic stem cell transplantation for patients lacking suitable donors[2][4][5][7].
- Other names
- Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47
02
Targets
NOX2 (NADPH oxidase 2)
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