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, 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 and Metabolic disorders.

CRISPR-Cas | TALEN | ZFN | MegaNuclease | MegaTAL

Latest Trial News

Clinical Roundup: Gene-Edited CAR T Therapies

Mar. 31, 2021

In recent years, researchers have turned to gene editing to address the limitations of CAR T therapies. This roundup summarises the clinical-stage...

Therapeutics

Antibacterial

Urinary Tract Infections, UTI, (NCT04191148)

Open access

Sponsors:
Locus Biosciences

IND Enabling

Phase I

Phase II

Phase III

Blood

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

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia,
Sponsors:
Editas Medicine

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

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

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, (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:
Case Western Reserve University

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (EDIT-301)
Sponsors:
Editas Medicine

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 Incorporated

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)
Sponsors:
Graphite Bio 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, (NCT03432364)
Sponsors:
Sangamo Therapeutics, Inc.

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 Myeloid Leukaemia, AML
Sponsors:
Intellia Therapeutics

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

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, (NCT04150497)
Sponsors:
Cellectis S.A.

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

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

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

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, MM, (ALLO-605)
Sponsors:
Allogene Therapeutics, Inc.

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, (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

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, 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

T-cell Acute Lymphoblastic Leukemia, ALL, (NCT03690011)
Sponsors:
Baylor College of Medicine

IND Enabling

Phase I

Phase II

Phase III

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

IND Enabling

Phase I

Phase II

Phase III

T-Cell Lymphoma, (NCT04502446)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Eye

Herpes Simplex Virus Refractory Keratitis, (NCT04560790)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Leber Congenital Amaurosis, (NCT03872479)
Sponsors:
Editas Medicine, Inc.

IND Enabling

Phase I

Phase II

Phase III

Metabolic Disorder

Mucopolysaccharidosis type II, MPS II, (2018-000192-33)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis I, MPS I, (2018-000206-28)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis II, MPS II, (NCT03041324)
Sponsors:
Sangamo Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis type I, MPS I, (NCT02702115)
Sponsors:
Sangamo Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Other Genetic Conditions

Rare Diseases

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

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

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

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

Herpes Simplex Virus Refractory Keratitis, (NCT04560790)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Leber Congenital Amaurosis, (NCT03872479)
Sponsors:
Editas Medicine, Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis type II, MPS II, (2018-000192-33)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis I, MPS I, (2018-000206-28)
Sponsors:
Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis II, MPS II, (NCT03041324)
Sponsors:
Sangamo Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis type I, MPS I, (NCT02702115)
Sponsors:
Sangamo Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (EDIT-301)
Sponsors:
Editas Medicine

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 Incorporated

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)
Sponsors:
Graphite Bio 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

Transthyretin amyloidosis, ATTR, (NCT04601051)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Autoimmune Diseases

Diagnostics

CRISPR SARS-CoV-2 Detection platform for COVID-19 Disease
Sponsors:
Mammoth Biosciences

CRISPR SARS-CoV-2 Diagnostic kit for COVID-19 Disease
Sponsors:
SHERLOCK Biosciences

CRISPR-based Mycobacterium tuberculosis Diagnostic Test for Pulmonary Tuberculosis
Sponsors:
Huashan Hospital

Human Enterovirus Infections, HEV, (NCT04535648)
Sponsors:
Children's Hospital of Fudan University

Pertussis -Whooping Cough, (NCT04535505)
Sponsors:
Children's Hospital of Fudan University

Pneumonia, (NCT04178382)
Sponsors:
Chinese Medical Association