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renizgamglogene autogedtemcel
- Development stage
- Unknown
- Lead developer
- Editas Medicine
- 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, Gene Therapies, Tumor-Infiltrating Lymphocytes (TILs) → Native Immune Cells → Adoptive Cell Transfer → Cell Therapies
- Administration
- Intravenous
01
Overview
Renizgamglogene autogedtemcel (EDIT-301) is an investigational, autologous cell therapy based on CRISPR/Cas12a genome editing technology. The therapy uses a patient’s own CD34+ hematopoietic stem and progenitor cells, which are edited at the gamma globin gene (HBG1 and HBG2) promoters to increase fetal hemoglobin (HbF) production. Elevated HbF can compensate for defective or deficient adult hemoglobin in disorders such as sickle cell disease and beta-thalassemia. The product is administered intravenously after ex vivo modification of the patient’s cells. Renizgamglogene autogedtemcel is being developed by Editas Medicine for the treatment of sickle cell disease and beta-thalassemia[7].
- Other names
- renizgamglogene autogedtemcelreni-celEDIT-301EDIT301EDIT 301
02
Targets
HBG2 (Hemoglobin subunit gamma-2)Hemoglobin subunit gamma-1 promoter
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