CRISPR CLINICAL TRIALS

Medicine is changing.

New CRISPR gene editing clinical studies are rapidly emerging and we think a comprehensive overview is valuable to help keep track of them all.

CRISPR is the hot new rapidly rising gene editing tool but we also include clinical trials of other gene editing modalities like the Zinc Finger Nucleases, TALENs, MegaTALS, CAS-CLOVER, MegaNucleases and any new variants that will be out there in the future. We will keep updating our overview.

Check already now the world's-first clinical trial for a CRISPR-Cas3 phage therapy for treatment of urinary tract infections, developed by Locus BioSciences and get all the details about their unique technology from this interview with VP, Business Development, Joseph Nixon.

The clinical trials are arranged in the following areas:

Antibacterials
Blood
Cancer
Eye
Metabolic disorders
Other genetic conditions

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Latest Trial News

Clinical Trial Roundup: Gene-Editing Therapies for...

Mar. 1, 2023

In this update, we summarise the two ongoing gene-editing clinical trials for renal cell carcinoma, which is the most common form of kidney cancer...

Clinical Update: TALEN-Edited CAR T-Cell Therapy...

Feb. 7, 2023

This week’s update looks at a Phase 1/2 study for ALLO-501A, a TALEN-edited CAR T-cell therapy for large B-cell lymphoma.

Therapeutics

Antibacterial

E. coli infections (NCT05277350)
Sponsors:
SNIPR Biome

IND Enabling

Phase I

Phase II

Phase III

Urinary Tract Infections, UTI, (NCT04191148)

Open access

Sponsors:
Locus Biosciences

IND Enabling

Phase I

Phase II

Phase III

Blood

Beta-Thalassemia, BT, (NCT03728322)
Sponsors:
Allife Medical Science and Technology Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Inc.

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT04211480)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT05442346)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Haemophilia B Factor IX, (2017-004805-42)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Haemophilia B, (NCT02695160)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV (NCT02388594)
Sponsors:
University of Pennsylvania

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV (NCT05143307)
Sponsors:
Excision BioTherapeutics

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT00842634)
Sponsors:
University of Pennsylvania

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT01044654)
Sponsors:
Sangamo Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT01252641)
Sponsors:
Sangamo Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT02225665)
Sponsors:
Sangamo BioSciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT02500849)
Sponsors:
City of Hope Medical Center

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT03164135)
Sponsors:
Affiliated Hospital to Academy of Military Medical Sciences

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT03617198)
Sponsors:
University of Pennsylvania

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT03666871)
Sponsors:
University of Cincinnati

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD (NCT05456880)
Sponsors:
Beam Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (2018-001320-19)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT03653247)
Sponsors:
Bioverativ Therapeutics Inc. (a Sanofi company)

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT03745287)
Sponsors:
Vertex Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04443907)
Sponsors:
Novartis Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04774536)
Sponsors:
Principal Investigator: Mark Walters, MD UCSF Benioff Children's Hospital Oakland

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04819841)

Discontinued

Sponsors:
Graphite Bio Inc

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04853576)
Sponsors:
Editas Medicine, Inc.

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT05329649)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion Dependent Beta-Thalassaemia, TDT, (NCT04208529)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Transfusion Dependent Beta-Thalassaemia, TDT, (ChiCTR2100052858)
Sponsors:
923rd Hospital of The People's Liberation Army

IND Enabling

Phase I

Phase II

Phase III

Transfusion Dependent Beta-Thalassaemia, TDT, (ChiCTR2100053406)
Sponsors:
The First Affiliated Hospital, Guangxi Medical University

IND Enabling

Phase I

Phase II

Phase III

Transfusion Dependent Beta-Thalassaemia, TDT, (NCT04925206)
Sponsors:
EdiGene (GuangZhou) Inc.

IND Enabling

Phase I

Phase II

Phase III

Transfusion Dependent Beta-Thalassemia, TDT, (NCT03432364)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Transfusion Dependent Beta-Thalassemia, TDT, (NCT04390971)
Sponsors:
Institute of Hematology and Blood Diseases Hospital

IND Enabling

Phase I

Phase II

Phase III

Transfusion Dependent Beta-Thalassemia, TDT, (NCT05444894)
Sponsors:
Editas Medicine

IND Enabling

Phase I

Phase II

Phase III

Transfusion-Dependent β-Thalassemia, TDT, (NCT05356195)
Sponsors:
Vertex Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Cancer

Acute Lymphoblastic Leukemia, ALL, (NCT04037566)
Sponsors:
Xijing Hospital, The Fourth Military Medical University

IND Enabling

Phase I

Phase II

Phase III

Acute Lymphoblastic Leukemia, T-ALL (NCT04984356)
Sponsors:
Wugen inc.

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukaemia, AML, (2018-001018-14)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukaemia, AML, (NCT04106076)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukaemia, AML, (NCT04614636)
Sponsors:
Fate Therapeutics, Inc

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukemia, AML, (NCT03190278)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukemia, AML, (NCT04849910)
Sponsors:
Vor Biopharma

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukemia, AML, (NCT05066165)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukemia, AML, (NCT05309733)
Sponsors:
Vor Biopharma

IND Enabling

Phase I

Phase II

Phase III

Advanced Hepatocellular Carcinoma, HCC, (NCT04417764)
Sponsors:
Central South University

IND Enabling

Phase I

Phase II

Phase III

B Cell Malignancies, B Cell Leukemia, B Cell Lymphoma, NHL, (NCT03398967)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

B Cell Malignancy, NHL, (NCT03166878)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

B- cell Acute Lymphoblastic Leukemia, ALL, (NCT04557436)
Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust

IND Enabling

Phase I

Phase II

Phase III

B- cell Acute Lymphoblastic Leukaemia, ALL, (NCT03666000)
Sponsors:
Precision BioSciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukemia, ALL, (NCT02746952)
Sponsors:
Institut de Recherches Internationales Servier

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukemia, ALL, (NCT02808442)
Sponsors:
Institut de Recherches Internationales Servier

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukemia, ALL, (NCT03232619)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukemia, ALL, (NCT03298828)
Sponsors:
Third Military Medical University

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukemia, ALL, (NCT04150497)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukemia, ALL, (NCT04154709)
Sponsors:
Xuzhou Medical University

IND Enabling

Phase I

Phase II

Phase III

B-Cell Malignancies, NHL, (NCT04629729)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

B-cell Malignancy, NHL, (NCT04035434)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

B-cell Non-Hodgkin Lymphoma, NHL, (NCT04637763)
Sponsors:
Caribou Biosciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Billiary Tract Cancer, BTC, (NCT04976218)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Bladder Cancer, Invasive, IBC, (NCT02863913)
Sponsors:
Peking University

IND Enabling

Phase I

Phase II

Phase III

Castrate-Resistant Prostate Cancer, CRPC, (NCT04768608)
Sponsors:
Zhejiang University

IND Enabling

Phase I

Phase II

Phase III

Esophageal Cancer, (NCT03081715)
Sponsors:
Hangzhou Cancer Hospital

IND Enabling

Phase I

Phase II

Phase III

Gastro-Intestinal Cancer, GI, (NCT04426669)
Sponsors:
Intima Bioscience, Inc.

IND Enabling

Phase I

Phase II

Phase III

Haematologic malignancy, Non-Hodgkin Lymphoma, NHL, (NCT04649112)
Sponsors:
Precision BioSciences Inc.

IND Enabling

Phase I

Phase II

Phase III

Human papillomavirus (HPV) related cervical cancer, (NCT02800369)
Sponsors:
Huazhong University of Science and Technology

IND Enabling

Phase I

Phase II

Phase III

Human papillomavirus (HPV) related cervical cancer, (NCT03057912)
Sponsors:
First Affiliated Hospital, Sun Yat-Sen University

IND Enabling

Phase I

Phase II

Phase III

Human papillomavirus (HPV) related cervical cancer, (NCT03226470)
Sponsors:
Huazhong University of Science and Technology

IND Enabling

Phase I

Phase II

Phase III

Large B-Cell Lymphoma, LBCL, (NCT04416984)
Sponsors:
Allogene Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Metastatic epithelial-derived solid tumors (NCT05239143)
Sponsors:
Poseida Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Metastatic Non-small Cell Lung Cancer, NSCLC, (NCT02793856)
Sponsors:
Sichuan University

IND Enabling

Phase I

Phase II

Phase III

Multiple myeloma, MM (NCT04960579)
Sponsors:
Poseida Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Multiple myeloma, MM (NCT05000450)
Sponsors:
Allogene Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Multiple myeloma, MM (NCT05069935)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Multiple myeloma, MM (NCT05308875)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, MM, (NCT03399448)
Sponsors:
University of Pennsylvania

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, MM, (NCT03492268)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, MM, (NCT03752541)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, MM, (NCT04093596)
Sponsors:
Allogene Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, MM, (NCT04142619)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, MM, (NCT04171843)
Sponsors:
Precision BioSciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, MM, (NCT04244656)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Multiple Solid Tumor Adult, (NCT03545815)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Nasopharyngeal Carcinoma, (NCT03044743)
Sponsors:
Yang Yang, MD, PhD, MSCR, The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School

IND Enabling

Phase I

Phase II

Phase III

Non-Hodgkin Lymphoma, NHL, (NCT03939026)
Sponsors:
Allogene Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Non-Hodgkin Lymphoma, NHL, (NCT04030195)
Sponsors:
Precision BioSciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Non-Hodgkin Lymphoma, NHL, (NCT05169489)
Sponsors:
2seventy bio (No investigator disclosed)

IND Enabling

Phase I

Phase II

Phase III

Non-Hodgkin Lymphoma, NHL, (NCT05332054)
Sponsors:
Caribou Biosciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Non-small Cell Lung Cancer,NSCLC, (NCT03525782)
Sponsors:
First Affiliated Hospital of Guangdong Pharmaceutical University

IND Enabling

Phase I

Phase II

Phase III

Prostate Cancer, (NCT03525652)
Sponsors:
The First Affiliated Hospital of Guangdong Pharmaceutical University

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B cell malignancies, (NCT04227015)
Sponsors:
Zhejiang University

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Lymphoma, DLBCL, (NCT04026100)
Sponsors:
The First Affiliated Hospital of Nanjing Medical University

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Malignancies, (NCT03229876)
Sponsors:
Bioray Laboratories - Shanghai Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Malignancies, (NCT04933825)
Sponsors:
EdiGene Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Malignancies, Non-Hodgkin lymphoma, NHL, (2018-003916-38)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Malignancies, Non-Hodgkin lymphoma, NHL, (NCT04213469)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Relapsed refractory B- cell Acute Lymphoblastic Leukaemia, ALL, (2019-003462-40)
Sponsors:
Great Ormond Street Hospital for Children NHS Trust

IND Enabling

Phase I

Phase II

Phase III

Relapsed/Refractory Haematopoietic Malignancies (NCT04767308)
Sponsors:
Huazhong University of Science and Technology

IND Enabling

Phase I

Phase II

Phase III

Renal Cell Carcinoma, RCC, (NCT04438083)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Renal Cell Carcinoma, RCC, (NCT04696731)
Sponsors:
Allogene Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Solid Tumor Adult, (NCT03747965)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Solid tumors - Tumor infiltration lymphocytes, TILs (NCT04842812)
Sponsors:
Second Affiliated Hospital of Guangzhou Medical University

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors (NCT03970382)
Sponsors:
PACT Pharma, Inc.

IND Enabling

Phase I

Phase II

Phase III

Solid tumors, (NCT05395052)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Solid Tumours, EDIT-201

Discontinued

Sponsors:
Editas Medicine

IND Enabling

Phase I

Phase II

Phase III

T-Cell Acute Lymphoblastic Leukaemia, T-ALL, (ChiCTR1900025311)
Sponsors:
Xinqiao Hospital of Chongqing, Second Affiliated Hospital of the Army Medical University, Hematology Department

IND Enabling

Phase I

Phase II

Phase III

T-cell Acute Lymphoid Leukaemia, ALL, (NCT05397184)
Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust

IND Enabling

Phase I

Phase II

Phase III

T-Cell Chronic Lymphoblastic Leukaemia, CLL, (NCT04264078)
Sponsors:
Xinqiao Hospital of Chongqing

IND Enabling

Phase I