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 Trial Update – CRISPR-Engineered Dual...

Jan. 20, 2021

This week’s gene-editing clinical trial update looks at a universal dual specificity CD19 and CD20/CD22 CAR-T cell immunotherapy that is being...

Therapeutics

Antibacterial

Urinary Tract Infections, UTI, (NCT04191148)

Open access

Sponsors:
Locus Biosciences

IND Enabling

Phase I

Phase II

Phase III

Blood

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, (NCT03728322)
Sponsors:
Allife Medical Science and Technology Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

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

IND Enabling

Phase I

Phase II

Phase III

Hemophilia 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

Severe Sickle Cell Disease, SCD, (GPH101)
Sponsors:
Graphite Bio

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

Transfusion Dependent Beta-Thalassemia ,TDT, (ET-01)
Sponsors:
EdiGENE Corporation

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

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

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

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

IND Enabling

Phase I

Phase II

Phase III

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

IND Enabling

Phase I