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RAG1 LV CD34+ cells
- Development stage
- Unknown
- Lead developer
- Mustang Bio
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
RAG1 LV CD34+ cells is an autologous ex vivo hematopoietic stem cell gene therapy developed by Leiden University Medical Center (LUMC) and licensed to Mustang Bio as MB-110. The therapy involves the transduction of a patient's own CD34+ hematopoietic stem cells with a lentiviral vector (SIN-LV-RAG1 or pCCL.MND.coRAG1.wpre) encoding a codon-optimized version of the human RAG1 gene. It is specifically designed to treat RAG1-deficient severe combined immunodeficiency (SCID), a condition where mutations in the RAG1 gene impair V(D)J recombination, leading to a lack of functional T and B cells. By reintroducing a functional RAG1 gene into the patient's stem cells, the therapy aims to restore immune system development and function. The program is currently being evaluated in a Phase I/II clinical trial (NCT04797260) for infants and young children.
- Other names
- RAG1-deficient SCID gene therapyRAG-1-deficient SCID gene therapyRAG 1-deficient SCID gene therapyAutologous Hematopoietic Stem Cell Gene Therapy for RAG1-Deficient SCID
02
Targets
RAG2 (Recombination activating 2)RAG1 (Recombination Activating Gene 1)
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