Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
autologous FANCA-transduced CD34+ hematopoietic stem cells
- Development stage
- Unknown
- Lead developer
- Rocket Pharmaceuticals
- Modality
- Gene Therapies, Stem Cell Therapies → Cell 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
- genetically engineered hematopoietic stem progenitor cells-Fred Hutchinson Cancer Center-Fanconi anemiaFANCA gene therapyGene Transfer for Fanconi Anemia Group A
Beyond the preview
Go deeper on autologous FANCA-transduced CD34+ hematopoietic stem cells.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on autologous FANCA-transduced CD34+ hematopoietic stem cells.