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MB-110
- Development stage
- Phase 1
- Lead developer
- Mustang Bio
- Modality
- Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
MB-110 is a first-in-class ex vivo lentiviral gene therapy developed by Mustang Bio, exclusively licensed from Leiden University Medical Centre (LUMC). It is designed for the treatment of RAG1-SCID, a rare genetic disease, by correcting RAG1 gene mutations.
- Other names
- RAG1 gene therapyRAG-1 gene therapyRAG 1 gene therapyautologous hematopoietic stem and progenitor cell gene therapy for RAG1-SCIDlentiviral RAG1 gene therapy
02
Targets
RAG1 (Recombination Activating Gene 1)
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