Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
peripheral blood stem cells (Cincinnati Children's Hospital Medical Center)
- Development stage
- Unknown
- Lead developer
- Cincinnati Children's Hospital Medical Center
- Modality
- Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
This biological cellular therapy consists of peripheral blood stem cells (PBSC) collected from a donor (mismatched related or matched unrelated) following mobilization with Granulocyte Colony-Stimulating Factor (G-CSF). The product undergoes T-cell depletion via positive CD34 selection using the Miltenyi CliniMACS system to minimize the risk of Graft-versus-Host Disease (GvHD). It is utilized in the RAFA (Risk-Adjusted HSCT for Fanconi Anemia) trial, a Phase II study conducted by Cincinnati Children's Hospital Medical Center. The trial investigates a risk-adjusted, chemotherapy-only conditioning regimen comprising Busulfan, Cyclophosphamide, Fludarabine, and rabbit anti-thymocyte globulin (ATG) without Cyclosporine. This approach aims to reduce transplant-related toxicities and improve engraftment outcomes for patients with Fanconi Anemia who also present with bone marrow failure, myelodysplastic syndrome (MDS), or acute myeloid leukemia (AML).
- Other names
- PBSCCD34+ selected peripheral blood stem cellsG-CSF mobilized peripheral blood stem cells
Beyond the preview
Go deeper on peripheral blood stem cells (Cincinnati Children's Hospital Medical Center).
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 peripheral blood stem cells (Cincinnati Children's Hospital Medical Center).