status NCT Num diseases Target gene Intervention/treatment Gene editing method phase Delivery method Trial link
Completed NCT04191148 Urinary Tract Infections, UTI E.coli genome LBP-EC01 CRISPR-Cas3 Phase I crPhage cocktail - In-vivo https://clinicaltrials.gov/ct2/show/record/NCT04191148
Unknown NCT03728322 Thalassemia Genetic Diseases Hemoglobin Subunit Beta (HBB) No CRISPR-Cas9 Phase I Ex-vivo https://clinicaltrials.gov/ct2/show/record/NCT03728322
Active, not recruiting NCT03655678 Thalassemia Genetic Diseases BAF Chromatin Remodeling Complex Subunit 11A (BCL11A) CTX001 CRISPR-Cas9 Phase II/III Electroporation - Ex-vivo https://clinicaltrials.gov/ct2/show/record/NCT03655678
Active not recruiting NCT04205435 Beta-Thalassemia Hemoglobin Subunit Beta (HBB) β-globin restored autologous HSC CRISPR-Cas9 Phase I/II Electroporation - Ex-vivo https://clinicaltrials.gov/ct2/show/record/NCT04205435
Enrolling by invitation NCT05143307 HIV Undisclosed EBT-101 CRISPR-Cas9 Phase I AAV9 - In-vivo https://clinicaltrials.gov/ct2/show/NCT05143307
Concerned 2018-001320-19 Sickle Cell Disease, Haematological Diseases, Haemoglobinopathies HBB CTX001 CRISPR-Cas9 Phase I/II Electroporation - Ex-vivo https://www.clinicaltrialsregister.eu/ctr-search/trial/2018-001320-19/BE#A
Active NCT03745287 Sickle Cell Disease, Haematological Diseases, Haemoglobinopathies BAF Chromatin Remodeling Complex Subunit 11A (BCL11A) CTX001 CRISPR-Cas9 Phase II/ III Electroporation - Ex-vivo https://clinicaltrials.gov/ct2/show/record/NCT03745287
Active ChiCTR2100052858 Transfusion Dependent Beta-Thalassaemia, TDT Undisclosed RM-001 CRISPR-Cas9 Phase I Undisclosed- Ex-vivo https://www.chictr.org.cn/hvshowproject.aspx?id=106695
Active NCT04037566 Leukemia Lymphocytic Acute in Relapse, Leukemia Lymphocytic Acute (All) Refractory Lymphoma, B-Cell, CD19 Positive, ALL CD19 molecule , HPK1 hematopoietic progenitor kinase 1 (also known as MAP4K1) Cyclophosphamide/ Fludarabine CRISPR-Cas9 Phase I Lentivirus (LV) and electroporation - Ex-vivo https://clinicaltrials.gov/ct2/show/record/NCT04037566
Active NCT04560790 Herpes Simplex Virus Refractory Keratitis UL8/UL29 BD111 Adult single group Dose CRISPR-Cas9 Phase I/II mRNA transfection - In-vivo https://clinicaltrials.gov/ct2/show/record/NCT04560790
Active NCT03872479 Blindness, Leber Congenital Amaurosis Centrosomal Protein 290 (CEP290) EDIT-101 CRISPR-Cas9 Phase I/II Adeno-associated virus (AAV5) - In-vivo https://clinicaltrials.gov/ct2/show/record/NCT03872479
Active NCT05210530 Type 1 Diabetes, T1D - VCTX210A unit CRISPR-Cas9 Phase I Ex-vivo https://clinicaltrials.gov/ct2/show/NCT05210530
Active NCT05120830 Hereditary Angioedema, HAE Kallikrein B1 (KLKB1) Biological NTLA-2002 CRISPR-Cas9 Phase I/II Lipid Nano Particles (LNPs) - In-vivo https://clinicaltrials.gov/ct2/show/NCT05120830
Active NCT04601051 Hereditary Transthyretin Amyloidosis, ATTR Transthyretin (TTR) NTLA-2001 CRISPR-Cas9 Phase I Lipid Nanoparticles (LNP) - In-vivo https://clinicaltrials.gov/ct2/show/record/NCT04601051