Gene-editing Clinical Trials

In 2023, therapeutic gene editing moved from promise to reality with the first approved CRISPR-based therapy, CASGEVY, which has since received regulatory clearance in multiple regions for the treatment of sickle cell disease and beta thalassemia.

Our manually curated CRISPR Medicine News Clinical Trials register tracks the advancement of gene-editing technologies in clinical development. The field now encompasses a diverse array of editing tools, including CRISPR-Cas, base editors, prime editors, zinc fingers, TALENs, meganucleases, CAS-CLOVER, RNA editors, and epigenetic-editing technology.

As of February 2025, CRISPR Medicine News monitors approximately 250 clinical trials involving gene-editing therapeutic candidates, with more than 150 trials currently active.

The clinical landscape spans multiple therapeutic areas, including:

  • Blood cancers
  • Haemoglobinopathies
  • Solid cancers
  • Viral diseases
  • Metabolic disorders
  • Autoimmune diseases
  • Hereditary amyloidosis
  • Inherited eye diseases
  • Cardiovascular disease
  • Bacterial diseases
  • Immunodeficiencies
  • Haemophilia
  • Neurological conditions
  • Muscular dystrophy
  • Other rare inherited diseases

As of February 2025, gene editing for blood disorders continues to lead the field, with the majority of Phase 3 trials targeting sickle cell disease and/or beta thalassemia. Phase 3 trials are are also underway in hereditary amyloidosis and immunodeficiencies.

Our clinical trial register is routinely updated to reflect new developments in the field.

Stay informed with our CMN Weekly Newsletter highlighting the latest trial updates and breakthroughs in CRISPR medicine.

This page was last updated on: 28th February 2025

 

Clinical Trials

Autoimmune diseases

Lupus Nephritis, (NCT06681337)
Sponsors:
Bioray Laboratories
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Refractory Autoimmune Diseases, (NCT06485232)
Sponsors:
Xuanwu Hospital, Beijing
Indicator
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
Systemic Lupus Erythematosus, SLE, (NCT06255028)
Sponsors:
Century 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
Systemic Lupus Erythematosus, SLE, (NCT06752876)
Sponsors:
Caribou Biosciences, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III

Bacterial diseases

Indicator
IND Enabling
Phase I
Phase II
Phase III
Urinary Tract Infections, UTI, (NCT04191148)
Open access


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

Cardiovascular diseases

Indicator
IND Enabling
Phase I
Phase II
Phase III
Familial Hypercholesterolemia, FH, (NCT05398029)
Sponsors:
Verve 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
Refractory Hypercholesterolemia, NCT06451770
Sponsors:
Verve Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III

Haematological malignancies (blood cancers)

Indicator
IND Enabling
Phase I
Phase II
Phase III
Acute Myeloid Leukaemia, AML, (ISRCTN14430213)
Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust
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
Acute Myeloid Leukaemia, AML, (NCT05066165)
Sponsors:
Intellia Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT03232619)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
B-Cell Lymphoma, BCL, (NCT05934097)
Sponsors:
Fate Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
B-Cell Non-Hodgkin lymphoma, NHL, (NCT04213469)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
IND Enabling
Phase I
Phase II
Phase III
B-Cell Non-Hodgkin Lymphoma, NHL, (NCT04649112)
Sponsors:
Precision BioSciences Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
B-Cell Non-Hodgkin Lymphoma, NHL, (NCT05631912)
Sponsors:
Chinese PLA General Hospital
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
B-Cell Non-Hodgkin Lymphoma, NHL, (NCT06014073)
Sponsors:
Chinese PLA General Hospital
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
B-cell Non-Hodgkin Lymphoma, NHL, (NCT06321289)
Sponsors:
Chinese PLA General Hospital
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Diffuse Large B-cell Lymphoma, DLBCL, (NCT04026100)
Sponsors:
The First Affiliated Hospital of Nanjing Medical University
Indicator
IND Enabling
Phase I
Phase II
Phase III
Hematologic Malignancies, (NCT04023071)
Sponsors:
Fate Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Large B-cell lymphoma, LBCL, (NCT06500273)
Sponsors:
Allogene Therapeutics
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, (NCT05069935)
Sponsors:
Fate Therapeutics 
Indicator
IND Enabling
Phase I
Phase II
Phase III
Relapsed of Refractory B-cell Acute Lymphoblastic Leukaemia, B-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
IndicatorIndicator
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
Indicator
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia, B-ALL, (2019-003462-40)
Sponsors:
Great Ormond Street Hospital for Children NHS Trust
Indicator
IND Enabling
Phase I
Phase II
Phase III
Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT02746952)
Sponsors:
Institut de Recherches Internationales Servier
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
Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT04557436)
Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust
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
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, (NCT03298828)
Sponsors:
Third Military Medical University
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
Relapsed or Refractory B-cell Malignancies, (NCT04037566)
Sponsors:
Xijing Hospital, The Fourth Military Medical University
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Relapsed or Refractory Haematopoietic Malignancies, (NCT04767308)
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Relapsed or Refractory Multiple Myeloma, MM, (NCT03492268)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
IND Enabling
Phase I
Phase II
Phase III
Relapsed or Refractory Multiple Myeloma, MM, (NCT03752541)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
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
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
T-cell Acute Lymphoblastic Leukaemia, T-ALL, (NCT05397184)
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
T-Cell Lymphoblastic Leukaemia and Lymphoma, (NCT04264078)
Sponsors:
Xinqiao Hospital of Chongqing
Indicator
IND Enabling
Phase I
Phase II
Phase III

Haemoglobinopathies

Beta-Thalassemia, BT, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Inc.
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, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, BT, (NCT04211480)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, BT, (NCT05442346)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, BT, (NCT06024876)
Sponsors:
CorrectSequence Therapeutics Co., Ltd
Indicator
IND Enabling
Phase I
Phase II
Phase III
Beta-Thalassemia, BT, (NCT06328764)
Sponsors:
CorrectSequence Therapeutics Co., Ltd
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (2018-001320-19)
Sponsors:
Vertex Pharmaceuticals Incorporated
IndicatorIndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03167450)
Sponsors:
National Human Genome Research Institute (NHGRI)
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03653247)
Sponsors:
Sangamo Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT03745287)
Sponsors:
Vertex Pharmaceuticals
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04443907)
Sponsors:
Novartis Pharmaceuticals
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04774536)
Sponsors:
Mark Walters, MD 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04819841)
Sponsors:
Kamau Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT04853576)
Sponsors:
Editas Medicine, Inc. 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT05329649)
Sponsors:
Vertex Pharmaceuticals Incorporated
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT05456880)
Sponsors:
Beam Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Sickle cell disease, SCD, (NCT05951205)
Sponsors:
Vertex Pharmaceuticals Incorporated
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06287086)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06287099)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06300723)
Sponsors:
Bioray Laboratories
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06506461)
Sponsors:
St. Jude Children's Research Hospital  
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Disease, SCD, (NCT06565026)
Sponsors:
CorrectSequence Therapeutics Co., Ltd
Indicator
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Sickle Cell Trait, (NCT06071377)
Sponsors:
National Alliance for Sickle Cell Centers
IND Enabling
Phase I
Phase II
Phase III
Transfusion-Dependent Beta-Thalassaemia, TDT, (ChiCTR2100052858)
Sponsors:
923rd Hospital of The People's Liberation Army 
Indicator
IND Enabling
Phase I
Phase II
Phase III
Transfusion-Dependent Beta-Thalassaemia, TDT, (ChiCTR2100053406)
Sponsors:
The First Affiliated Hospital, Guangxi Medical University  
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
Transfusion-Dependent Beta-Thalassemia, TDT, (NCT04390971)
Sponsors:
Institute of Hematology and Blood Diseases Hospital
IND Enabling
Phase I
Phase II
Phase III
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
Transfusion-Dependent Beta-Thalassemia, TDT, (NCT06041620)
Sponsors:
Institute of Hematology & Blood Diseases Hospital, China
IND Enabling
Phase I
Phase II
Phase III
Transfusion-dependent Beta-Thalassemia, TDT, (NCT06065189)
Sponsors:
Children's Hospital of Fudan University
Indicator
IND Enabling
Phase I
Phase II
Phase III
Transfusion-dependent Beta-Thalassemia, TDT, (NCT06291961)
Sponsors:
CorrectSequence Therapeutics Co., Ltd
Indicator
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III

Haemophilia

Hereditary amyloidosis

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
Transthyretin Amyloidosis, ATTR, (NCT04601051)
Sponsors:
Intellia Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Transthyretin Amyloidosis, ATTR, (NCT06128629)
Sponsors:
Intellia Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Transthyretin Amyloidosis, ATTR, (NCT06539208)
Sponsors:
YolTech Therapeutics Co., Ltd
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III

Immunodeficiencies

Chronic Granulomatous Disease, CGD, (NCT06325709)
Sponsors:
National Institute of Allergy and Infectious Diseases (NIAID)
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Hereditary Angioedema, HAE, (NCT05120830)
Sponsors:
Intellia Therapeutics
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Hereditary Angioedema, HAE, (NCT06634420)
Sponsors:
Intellia Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III

Inherited eye diseases

ABCA4 Retinopathy, (NCT06467344)
Sponsors:
Ascidian Therapeutics, Inc
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Leber Congenital Amaurosis, LCA, (NCT03872479)
Sponsors:
Editas Medicine, Inc.
IndicatorIndicator
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
Primary Open Angle Glaucoma, POAG, (NCT06465537)
Sponsors:
Shanghai BDgene Co., Ltd.
IND Enabling
Phase I
Phase II
Phase III
Retinal Degeneration, (NCT03396042)
Sponsors:
Editas Medicine, Inc.
IND Enabling
Phase I
Phase II
Phase III
Retinitis Pigmentosa, RP, (NCT05805007)
Sponsors:
Peking University Third Hospital
Indicator
IND Enabling
Phase I
Phase II
Phase III

Metabolic disorders

IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Mucopolysaccharidosis I, MPS I, (NCT02702115)
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Phenylketonuria, PKU, (NCT05222178)
Sponsors:
Homology Medicines
Indicator
IND Enabling
Phase I
Phase II
Phase III
Refractory Dyslipidemias, (ACTRN12623000809639)
Sponsors:
CRISPR Therapeutics AG
Indicator
IND Enabling
Phase I
Phase II
Phase III
Type 1 Diabetes, T1D, (NCT05210530)
Sponsors:
CRISPR Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Type 1 Diabetes, T1D, (NCT05565248)
Sponsors:
CRISPR Therapeutics AG
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III

Muscular dystrophies

Duchenne Muscular Dystrophy, DMD, (NCT05514249)
Sponsors:
Cure Rare Disease, Inc
Indicator
IND Enabling
Phase I
Phase II
Phase III
Duchenne Muscular Dystrophy, DMD, (NCT06594094)
Sponsors:
HuidaGene Therapeutics Co., Ltd.
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III

Neurological disease

IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Huntington's Disease, (NCT05032196)
Sponsors:
Wave Life Sciences Ltd.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III

Solid cancers

Indicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Advanced Stage Epstein-Barr Virus (EBV) Associated Malignancies, (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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Breast Cancer, BC, (NCT05812326)
Sponsors:
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
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
Epithelial Ovarian Cancer, EOC, (NCT05617755)
Sponsors:
Arsenal Biosciences, Inc.
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
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
Metastatic Melanoma, (NCT06783270)
Sponsors:
Inge Marie Svane
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
Non-small Cell Lung Cancer, NSCLC, (NCT03525782)
Sponsors:
First Affiliated Hospital of Guangdong Pharmaceutical University
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Non-small Cell Lung Cancer, NSCLC, (NCT05566223)
Sponsors:
Intima Bioscience, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Prostate Cancer, (NCT03525652)
Sponsors:
The First Affiliated Hospital of Guangdong Pharmaceutical University
IndicatorIndicator
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
Renal Cell Carcinoma, RCC, (NCT02867332)
Sponsors:
Peking University
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
Renal Cell Carcinoma, RCC, (NCT06245915)
Sponsors:
Arsenal Biosciences, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumor Adult, (NCT03545815)
Sponsors:
Chinese PLA General Hospital
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
Solid Tumor, Adult, (NCT04976218)
Sponsors:
Chinese PLA General Hospital
Indicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT03970382)
Sponsors:
PACT Pharma, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT04551885)
Sponsors:
Fate Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT04842812)
Sponsors:
Second Affiliated Hospital of Guangzhou Medical University
Indicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT05239143)
Sponsors:
Poseida Therapeutics 
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT05361174)
Sponsors:
Iovance Biotherapeutics, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT05395052)
Sponsors:
Fate Therapeutics 
Indicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT06237881)
Sponsors:
M.D. Anderson Cancer Center
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT06241456)
Sponsors:
Fate Therapeutics
Indicator
IND Enabling
Phase I
Phase II
Phase III
Solid Tumors, (NCT06598371)
Sponsors:
M.D. Anderson Cancer Center
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III

Viral diseases

Chronic Hepatitis B, HBV, (NCT06671093)
Sponsors:
Tune Therapeutics, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
Chronic Hepatitis B, HBV, (NCT06680232)
Sponsors:
Precision BioSciences, Inc.
Indicator
IND Enabling
Phase I
Phase II
Phase III
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
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
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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
Induced Long-term Immunity in COVID-19 Patients, (NCT04990557)
Sponsors:
Mahmoud Ramadan mohamed Elkazzaz
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III

Other rare inherited blood disorders

Severe Pyruvate Kinase Deficiency, PKD, (ChiCTR2300073795)
Sponsors:
Shanghai Children's Medical Center
Indicator
IND Enabling
Phase I
Phase II
Phase III

Other rare inherited disorders

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
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Congenital Hearing Loss, (NCT06025032)
Sponsors:
HuidaGene Therapeutics Co., Ltd.
Indicator
IND Enabling
Phase I
Phase II
Phase III
MECP2 Duplication Syndrome, MDS, (NCT06615206)
Sponsors:
HuidaGene Therapeutics Co., Ltd.
IND Enabling
Phase I
Phase II
Phase III

Long-term follow-up (LTFU) studies

IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
B-Cell Non-Hodgkin Lymphoma, NHL, (NCT05332054)
Sponsors:
Caribou Biosciences, Inc.
IND Enabling
Phase I
Phase II
Phase III
Familial Hypercholesterolemia, FH, (NCT06112327)
Sponsors:
Verve Therapeutics, Inc.
IND Enabling
Phase I
Phase II
Phase III
Hematologic and Solid Malignancies, (NCT06208878)
Sponsors:
CRISPR Therapeutics AG
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
Hereditary Angioedema, HAE, (NCT06262399)
Sponsors:
Intellia Therapeutics
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
Indicator
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
IND Enabling
Phase I
Phase II
Phase III
Transthyretin Amyloidosis, ATTR, (NCT05697861)
Sponsors:
Intellia Therapeutics
IND Enabling
Phase I
Phase II
Phase III
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