Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
RAG1 LV CD34+ autologous hematopoietic stem cells
- Development stage
- Unknown
- Lead developer
- Leiden University Medical Center
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Intravenous
01
Overview
RAG1 LV CD34+ autologous hematopoietic stem cells is an investigational ex vivo gene therapy product developed by Leids Universitair Medisch Centrum (LUMC) for the treatment of RAG1-deficient severe combined immunodeficiency (RAG1-SCID). The therapy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with a self-inactivating (SIN) lentiviral vector (pCCL.MND.coRAG1.wpre) encoding a codon-optimized human RAG1 gene. RAG1 is essential for V(D)J recombination during early T- and B-cell development, and its deficiency leads to a complete block in lymphocyte receptor rearrangement, causing life-threatening immunodeficiency. Following a single infusion of these gene-corrected cells, the therapy aims to achieve long-term immune reconstitution. It is currently being evaluated in a Phase I/II clinical trial (NCT04797260) in infants who lack an HLA-matched donor for standard allogeneic hematopoietic stem cell transplantation.
- Other names
- RAG1-LV-CD34+ cellsRAG1 LV CD34+ cellsautologous CD34+ cells transduced with pCCL.MND.coRAG1.wpre lentiviral vectorautologous RAG1 lentiviral CD34+ stem cell gene therapy
02
Targets
RAG2 (Recombination activating 2)RAG1 (Recombination Activating Gene 1)
Beyond the preview
Go deeper on RAG1 LV CD34+ autologous 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 RAG1 LV CD34+ autologous hematopoietic stem cells.