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

Therapeutics

Antibacterial

Urinary Tract Infections, UTI, (NCT04191148)
Open access


Sponsors:
Locus Biosciences
Indicator
IND Enabling
Phase I
Phase II
Phase III

Blood

Beta - thalassemia, BT, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
IndicatorIndicator
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, BT, (NCT03728322)
Sponsors:
Allife Medical Science and Technology Co., Ltd
Indicator
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.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Haemophilia B, (NCT02695160)
Sponsors:
Sangamo Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
Human Immunodeficiency Virus Infection, HIV, (NCT03666871)
Sponsors:
Case Western Reserve University
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (EDIT-301)
Sponsors:
Editas Medicine 
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03653247)
Sponsors:
Bioverativ Therapeutics Inc. (a Sanofi company)
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03745287)
Sponsors:
Vertex Pharmaceuticals Incorporated
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04443907)
Sponsors:
Novartis Pharmaceuticals
IndicatorIndicator
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04819841)
Sponsors:
Graphite Bio Inc
IndicatorIndicator
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III

Cancer

Acute Lymphoblastic Leukemia, ALL, (NCT04037566)
Sponsors:
Xijing Hospital, The Fourth Military Medical University
Indicator
IND Enabling
Phase I
Phase II
Phase III
Acute Myeloid Leukaemia, AML
Sponsors:
Intellia Therapeutics
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Acute Myeloid Leukaemia, AML, (NCT04614636)
Sponsors:
Fate Therapeutics, Inc
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
B Cell Malignancy, NHL, (NCT03166878)
Sponsors:
Chinese PLA General Hospital
IndicatorIndicator
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
B-cell Acute Lymphoblastic Leukemia, ALL, (NCT02746952)
Sponsors:
Institut de Recherches Internationales Servier
Indicator
IND Enabling
Phase I
Phase II
Phase III
B-cell Acute Lymphoblastic Leukemia, ALL, (NCT02808442)
Sponsors:
Institut de Recherches Internationales Servier
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
B-Cell Malignancies, NHL, (NCT04629729)
Sponsors:
Fate Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
B-cell Malignancy, NHL, (NCT04035434)
Sponsors:
CRISPR Therapeutics AG
Indicator
IND Enabling
Phase I
Phase II
Phase III
B-cell Non-Hodgkin Lymphoma, NHL, (NCT04637763)
Sponsors:
Caribou Biosciences, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Esophageal Cancer, (NCT03081715)
Sponsors:
Hangzhou Cancer Hospital
Indicator
IND Enabling
Phase I
Phase II
Phase III
Gastro-Intestinal Cancer, GI, (NCT04426669)
Sponsors:
Intima Bioscience, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Human papillomavirus (HPV) related cervical cancer, (NCT02800369)
Sponsors:
Huazhong University of Science and Technology
Indicator
IND Enabling
Phase I
Phase II
Phase III
Human papillomavirus (HPV) related cervical cancer, (NCT03057912)
Sponsors:
First Affiliated Hospital, Sun Yat-Sen University
Indicator
IND Enabling
Phase I
Phase II
Phase III
Human papillomavirus (HPV) related cervical cancer, (NCT03226470)
Sponsors:
Huazhong University of Science and Technology
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Multiple Myeloma, MM, (ALLO-605)
Sponsors:
Allogene Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Multiple Myeloma, MM, (NCT03399448)
Sponsors:
University of Pennsylvania
Indicator
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
Multiple Myeloma, MM, (NCT04142619)
Sponsors:
Cellectis S.A.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Multiple Myeloma, MM, (NCT04171843)
Sponsors:
Precision BioSciences, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Multiple Myeloma, MM, (NCT04244656)
Sponsors:
CRISPR Therapeutics AG
Indicator
IND Enabling
Phase I
Phase II
Phase III
Multiple Solid Tumor Adult, (NCT03545815)
Sponsors:
Chinese PLA General Hospital
Indicator
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Non-Hodgkin Lymphoma, NHL, (NCT03939026)
Sponsors:
Allogene Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Non-Hodgkin Lymphoma, NHL, (NCT04030195)
Sponsors:
Precision BioSciences, Inc.
IndicatorIndicator
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
IndicatorIndicator
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
Relapsed/Refractory Haematopoietic Malignancies (NCT04767308)
Sponsors:
Huazhong University of Science and Technology
Indicator
IND Enabling
Phase I
Phase II
Phase III
Renal Cell Carcinoma, RCC, (NCT04438083)
Sponsors:
CRISPR Therapeutics AG
Indicator
IND Enabling
Phase I
Phase II
Phase III
Renal Cell Carcinoma, RCC, (NCT04696731)
Sponsors:
Allogene Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumor Adult, (NCT03747965)
Sponsors:
Chinese PLA General Hospital
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
T-Cell Chronic Lymphoblastic Leukaemia, CLL, (NCT04264078)
Sponsors:
Xinqiao Hospital of Chongqing
Indicator
IND Enabling
Phase I
Phase II
Phase III
T-Cell Lymphoma, (NCT04502446)
Sponsors:
CRISPR Therapeutics AG
Indicator
IND Enabling
Phase I
Phase II
Phase III

Eye

IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Leber Congenital Amaurosis, (NCT03872479)
Sponsors:
Editas Medicine, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III

Metabolic Disorder

IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis I, MPS I, (2018-000206-28)
Sponsors:
Sangamo Therapeutics, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis II, MPS II, (NCT03041324)
Sponsors:
Sangamo Therapeutics Inc. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis type I, MPS I, (NCT02702115)
Sponsors:
Sangamo Therapeutics Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III

Other Genetic Conditions

Rare Diseases

Beta - thalassemia, BT, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Incorporated
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, BT, (NCT03728322)
Sponsors:
Allife Medical Science and Technology Co., Ltd
Indicator
IND Enabling
Phase I
Phase II
Phase III
Haemophilia B Factor IX, (2017-004805-42)
Sponsors:
Sangamo Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Haemophilia B, (NCT02695160)
Sponsors:
Sangamo Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Leber Congenital Amaurosis, (NCT03872479)
Sponsors:
Editas Medicine, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis I, MPS I, (2018-000206-28)
Sponsors:
Sangamo Therapeutics, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis II, MPS II, (NCT03041324)
Sponsors:
Sangamo Therapeutics Inc. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis type I, MPS I, (NCT02702115)
Sponsors:
Sangamo Therapeutics Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (EDIT-301)
Sponsors:
Editas Medicine 
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03653247)
Sponsors:
Bioverativ Therapeutics Inc. (a Sanofi company)
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03745287)
Sponsors:
Vertex Pharmaceuticals Incorporated
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04443907)
Sponsors:
Novartis Pharmaceuticals
IndicatorIndicator
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04819841)
Sponsors:
Graphite Bio Inc
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Transthyretin amyloidosis, ATTR, (NCT04601051)
Sponsors:
Intellia Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III

Autoimmune Diseases

Diagnostics

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
close
Search CRISPR Medicine