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AProArt-CD34
- Development stage
- Phase 2
- Lead developer
- University of California, San Francisco
- Modality
- Patient-derived iPSCs → iPSCs → Pluripotent Stem Cells → Stem Cell Therapies → Cell Therapies, Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Autologous CAR-T → CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Mesenchymal Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Tumor-Infiltrating Lymphocytes (TILs) → Native Immune Cells → Adoptive Cell Transfer → Cell Therapies
- Administration
- Intravenous
01
Overview
AProArt-CD34 is an autologous gene therapy product designed for the treatment of Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a rare and severe primary immunodeficiency caused by mutations in the DCLRE1C gene. The therapy involves isolating CD34+ hematopoietic stem cells from the patient, transducing them ex vivo with a self-inactivating lentiviral vector (AProArt) carrying a functional copy of the human Artemis (DCLRE1C) cDNA under control of its endogenous promoter, and then infusing these genetically modified cells back into the patient following sub-ablative busulfan conditioning. The goal is to restore immune function by enabling proper T and B cell development through correction of the underlying genetic defect. This approach is intended for ART-SCID patients who lack suitable donors for allogeneic transplantation or have failed previous transplants[1][6][8][10].
- Other names
- AProArt Gene TherapyArtemis-deficient SCID gene therapy
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