Table 1.
Clinical trials involving ex vivo or in vivo CRISPR-based genome editing.
| Status | NCT Number | Diseases | Target gene | Intervention/treatment | Clinical phase | Delivery method |
|---|---|---|---|---|---|---|
| Completed | NCT04191148 | Urinary tract infections | E. coli genome | LBP-EC01 | Phase I | crPhage cocktail - in vivo |
| Unknown | NCT03728322 | Thalassemia genetic diseases | Hemoglobin subunit beta (HBB) | No | Phase I | ex vivo |
| Active, not recruiting | NCT03655678 | Thalassemia genetic diseases | BAF chromatin remodeling complex subunit 11 A (BCL11A) | CTX001 | Phase II/III | Electroporation - ex vivo |
| Active not recruiting | NCT04205435 | Beta-thalassemia | Hemoglobin subunit beta (HBB) | β-globin restored autologous HSC | Phase I/II | Electroporation - ex vivo |
| Enrolling by invitation | NCT05143307 | HIV | Undisclosed | EBT-101 | Phase I | AAV9 - in vivo |
| Concerned | 2018-001320-19 | Sickle cell disease, hematological diseases, hemoglobinopathies | HBB | CTX001 | Phase I/II | Electroporation - ex vivo |
| Active | NCT03745287 | Sickle cell disease, hematological diseases, hemoglobinopathies | BAF chromatin remodeling complex subunit 11 A (BCL11A) | CTX001 | Phase II/III | Electroporation - ex vivo |
| Active | ChiCTR2100052858 | Transfusion dependent beta-thalassemia | Undisclosed | RM-001 | Phase I | Undisclosed - ex vivo |
| Active | NCT04037566 | Leukemia lymphocytic acute in relapse, acute lymphocytic leukemia (ALL) refractory lymphoma, B-cell, CD19 positive, ALL | CD19 molecule, HPK1 hematopoietic progenitor kinase 1 (also known as MAP4K1) | Cyclophosphamide/Fludarabine | Phase I | Lentivirus (LV) and electroporation - ex vivo |
| Active | NCT04560790 | Herpes simplex virus refractory keratitis | UL8/UL29 | BD111 adult single group dose | Phase I/II | mRNA transfection - in vivo |
| Active | NCT03872479 | Blindness, Leber congenital amaurosis | Centrosomal protein 290 (CEP290) | EDIT-101 | Phase I/II | Adeno-associated virus (AAV5) - in vivo |
| Active | NCT05210530 | Type 1 diabetes | – | VCTX210A unit | Phase I | Ex vivo |
| Active | NCT05120830 | Hereditary angioedema | Kallikrein B1 (KLKB1) | Biological NTLA-2002 | Phase I/II | Lipid nanoparticles - in vivo |
| Active | NCT04601051 | Hereditary transthyretin amyloidosis | Transthyretin (TTR) | NTLA-2001 | Phase I | Lipid nanoparticles - in vivo |