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.

The clinical trials are arranged in four broad areas: Antibacterials, Blood, Cancer, Eye and Metabolic disorder.

CRISPR-Cas | TALENS | ZFN

Antibacterial

Urinary Tract Infections, UTI, (NCT04191148)
Sponsors:

Locus Biosciences

IND Enabling

Phase I

Phase II

Phase III

Blood

Transfusion Dependent Beta-Thalassemia, TDT, (NCT03432364)
Sponsors:

Sangamo Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, (NCT03655678)
Sponsors:

Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, (NCT03745287)
Sponsors:

Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, (NCT03653247)
Sponsors:

Bioverativ, a Sanofi company

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

Human Immunodeficiency Virus Infection, HIV, (NCT03617198)
Sponsors:

University of Pennsylvania

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, (NCT02500849)
Sponsors:

City of Hope Medical Center

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, (NCT01252641)
Sponsors:

Sangamo Therapeutics Inc.

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, (NCT00842634)
Sponsors:

University of Pennsylvania

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

Hemophilia B, (NCT02695160)
Sponsors:

Sangamo Therapeutics, Inc.

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

Cancer

T-cell Acute Lymphoblastic Leukemia, ALL, (NCT03690011)
Sponsors:

Baylor College of Medicine

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

Solid Tumor Adult, (NCT03747965)
Sponsors:

Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, (NCT04142619)
Sponsors:

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukemia, AML, (NCT03190278)
Sponsors:

IND Enabling

Phase I

Phase II

Phase III

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

Non-Hodgkin Lymphoma, NHL, (2018-003916-38)
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

Multiple Myeloma, (NCT03399448)
Sponsors:

University of Pennsylvania

IND Enabling

Phase I

Phase II

Phase III

Multiple Myeloma, (NCT04244656)
Sponsors:

CRISPR Therapeutics AG

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

Acute Lymphoblastic Leukemia, ALL, (NCT04037566)
Sponsors:

Xijing Hospital, The Fourth Military Medical University

IND Enabling

Phase I

Phase II

Phase III

Invasive Bladder Cancer, IBC, (NCT02863913)
Sponsors:

Peking 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

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, (NCT02800369)
Sponsors:

Huazhong University of Science and Technology

IND Enabling

Phase I

Phase II

Phase III

Esophageal Cancer, (NCT03081715)
Sponsors:

Hangzhou Cancer Hospital

IND Enabling

Phase I

Phase II

Phase III

B-cell Malignancy, (NCT04035434)
Sponsors:

CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukemia, ALL, (NCT04150497)
Sponsors:

IND Enabling

Phase I

Phase II

Phase III

B Cell Lymphoma, (NCT03166878)
Sponsors:

Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukaemia, AML, (NCT04106076)
Sponsors:

IND Enabling

Phase I

Phase II

Phase III

Eye

Leber Congenital Amaurosis, (NCT03872479)
Sponsors:

IND Enabling

Phase I

Phase II

Phase III

Metabolic disorder

Mucopolysaccharidosis type I, MPS I, (NCT02702115)
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 I, MPS I, (2018-000206-28)
Sponsors:

Sangamo Therapeutics, 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