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Drug intelligence / Profile preview
autologous FANCA-transduced CD34+ hematopoietic stem cells
- Development stage
- Unknown
- Lead developer
- Rocket Pharmaceuticals
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
This autologous ex vivo gene therapy, developed by the Fred Hutchinson Cancer Center, is designed to treat Fanconi anemia complementation group A (FA-A). The therapeutic process involves the collection of a patient's own CD34+ hematopoietic stem and progenitor cells (HSPCs) from bone marrow or mobilized peripheral blood. These cells are then genetically modified ex vivo using the FancA-sW lentiviral vector, which carries a functional copy of the *FANCA* gene under the control of a phosphoglycerate kinase (PGK) promoter. The vector also includes a safety-modified woodchuck post-transcriptional regulatory element (WPRE). Once the gene-corrected cells are reinfused into the patient, they are expected to possess a selective growth advantage over the endogenous, DNA-repair-deficient cells, potentially restoring normal hematopoiesis and preventing the progression of bone marrow failure.
- Other names
- FANCA gene therapyGene Transfer for Fanconi Anemia Group A
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