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

Autoimmune diseases

Lupus Nephritis, (NCT06681337)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Neuromyelitis Optica Spectrum Disease, NMOSD, (NCT06633042)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Refractory Autoimmune Diseases, (NCT06485232)
Sponsors:
Xuanwu Hospital, Beijing

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Autoimmune Diseases, (NCT05859997)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Systemic Lupus Erythematosus, SLE, (NCT05988216)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Systemic Lupus Erythematosus, SLE, (NCT06255028)
Sponsors:
Century Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Systemic Lupus Erythematosus, SLE, (NCT06308978)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Systemic Lupus Erythematosus, SLE, (NCT06373991)
Sponsors:
EdiGene Inc.

IND Enabling

Phase I

Phase II

Phase III

Systemic Lupus Erythematosus, SLE, (NCT06752876)
Sponsors:
Caribou Biosciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Bacterial diseases

E. coli infections, (NCT05277350)
Sponsors:
SNIPR Biome

IND Enabling

Phase I

Phase II

Phase III

Urinary Tract Infections, UTI, (NCT04191148)

Open access

Sponsors:
Locus Biosciences

IND Enabling

Phase I

Phase II

Phase III

Urinary Tract Infections, UTI, (NCT05488340)
Sponsors:
Locus Biosciences

IND Enabling

Phase I

Phase II

Phase III

Cardiovascular diseases

Cardiovascular Disease or Calcific Aortic Valve Stenosis, (ACTRN12623001095651p)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Familial Hypercholesterolemia, FH, (NCT05398029)
Sponsors:
Verve Therapeutics, Inc.,

IND Enabling

Phase I

Phase II

Phase III

Familial Hypercholesterolemia, FH, (NCT06461702)
Sponsors:
YolTech Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Familial Hypercholesterolemia, HeFH, or Premature Coronary Artery Disease, CAD, (NCT06164730)
Sponsors:
Verve Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Refractory Hypercholesterolemia, NCT06451770
Sponsors:
Verve Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Haematological malignancies (blood cancers)

Acute Myeloid Leukaemia, AML, (2018-001018-14)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukaemia, AML, (ISRCTN14430213)
Sponsors:
Great Ormond Street Hospital for Children NHS Foundation Trust

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, (NCT04849910)
Sponsors:
Vor Biopharma

IND Enabling

Phase I

Phase II

Phase III

Acute Myeloid Leukaemia, AML, (NCT05066165)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT03232619)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT03666000)
Sponsors:
Imugene Limited

IND Enabling

Phase I

Phase II

Phase III

B-Cell Lymphoma, BCL, (NCT05934097)
Sponsors:
Fate Therapeutics

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.

IND Enabling

Phase I

Phase II

Phase III

B-Cell Non-Hodgkin Lymphoma, NHL, (NCT05169489)
Sponsors:
2seventy bio

IND Enabling

Phase I

Phase II

Phase III

B-Cell Non-Hodgkin Lymphoma, NHL, (NCT05631912)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

B-Cell Non-Hodgkin Lymphoma, NHL, (NCT06014073)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

B-cell Non-Hodgkin Lymphoma, NHL, (NCT06321289)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Blastic Plasmacytoid Dendritic Cell Neoplasm, BPDCN, (NCT03203369)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Diffuse Large B-cell Lymphoma, DLBCL, (NCT04026100)
Sponsors:
The First Affiliated Hospital of Nanjing Medical University

IND Enabling

Phase I

Phase II

Phase III

Hematologic Malignancies, (NCT04023071)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Large B-cell lymphoma, LBCL, (NCT06500273)
Sponsors:
Allogene Therapeutics

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, (NCT05069935)
Sponsors:
Fate Therapeutics

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

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Leukaemia and Lymphoma, (NCT05037669)
Sponsors:
University of Pennsylvania

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Acute Lymphocytic Leukemia, ALL, (NCT06481735)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Acute Myeloid Leukaemia, AML, (NCT03190278)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Acute Myeloid Leukaemia, AML, (NCT05662904)
Sponsors:
German Cancer Research Center

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Acute Myeloid Leukaemia, AML, (NCT05949125)
Sponsors:
AvenCell Europe GmbH

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Acute Myeloid Leukaemia, AML, and Multiple Myeloma, MM, (NCT04614636)
Sponsors:
Fate Therapeutics, Inc

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Acute Myeloid Leukemia, AML, (NCT05984199)
Sponsors:
Vor Biopharma

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Acute Myeloid Leukemia, AML, (NCT06128044)
Sponsors:
Caribou Biosciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B cell Malignancies, (NCT04227015)
Sponsors:
Zhejiang University

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

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

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT04150497)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia, B-ALL, (NCT04154709)
Sponsors:
Xuzhou Medical University

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

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Lymphoma and Chronic Lymphocytic Leukemia, (NCT04245722)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Malignancies, (NCT06014762)
Sponsors:
Poseida Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Malignancies, (NCT03166878)
Sponsors:
Chinese PLA General Hospital

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

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Malignancies, (NCT03398967)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Malignancies, (NCT04035434)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Malignancies, (NCT04037566)
Sponsors:
Xijing Hospital, The Fourth Military Medical University

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Malignancies, (NCT04629729)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-cell Malignancies, (NCT04933825)
Sponsors:
EdiGene Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Malignancies, (NCT05336409)
Sponsors:
Century Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Malignancies, (NCT05643742)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma, B-NHL, (NCT04637763)
Sponsors:
Caribou Biosciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma, NHL, (NCT05741359)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Haematopoietic Malignancies, (NCT04767308)
Sponsors:
Huazhong University of Science and Technology

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Large B Cell Lymphoma, LBCL, (NCT05714345)
Sponsors:
Allogene Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Large B-Cell Lymphoma, LBCL, (NCT04416984)
Sponsors:
Allogene Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Lymphoma and Leukaemia, (NCT04030195)
Sponsors:
Precision BioSciences, Inc.

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

Relapsed or Refractory Multiple Myeloma, MM, (NCT04093596)
Sponsors:
Allogene Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Multiple Myeloma, MM, (NCT04142619)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Multiple Myeloma, MM, (NCT04171843)
Sponsors:
Precision BioSciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Multiple Myeloma, MM, (NCT04244656)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Multiple Myeloma, MM, (NCT04960579)
Sponsors:
Poseida Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Multiple Myeloma, MM, (NCT05000450)
Sponsors:
Allogene Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Multiple Myeloma, MM, (NCT05182073)
Sponsors:
Fate Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Multiple Myeloma, MM, (NCT05308875)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Multiple Myeloma, MM, (NCT05722418)
Sponsors:
Caribou Biosciences

IND Enabling

Phase I

Phase II

Phase III

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

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory T-cell Acute Lymphoblastic Leukaemia, T-ALL (NCT04984356)
Sponsors:
Wugen inc.

IND Enabling

Phase I

Phase II

Phase III

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

IND Enabling

Phase I

Phase II

Phase III

Relapsed/Refractory Hematologic Malignancies (NCT06492304)
Sponsors:
CRISPR Therapeutics

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

T-Cell Acute Lymphoblastic Leukaemia, T-ALL, (ISRCTN15323014)
Sponsors:
Great Ormond Street Hospital

IND Enabling

Phase I

Phase II

Phase III

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

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

IND Enabling

Phase I

Phase II

Phase III

T-Cell Acute Lymphoblastic Leukaemia, T-ALL, and Lymphoma, T-LL, (NCT05885464)
Sponsors:
Beam Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

T-Cell Lymphoblastic Leukaemia and Lymphoma, (NCT04264078)
Sponsors:
Xinqiao Hospital of Chongqing

IND Enabling

Phase I

Phase II

Phase III

Haemoglobinopathies

Beta-Thalassemia, BT, (NCT03655678)
Sponsors:
Vertex Pharmaceuticals Inc.

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, (NCT04205435)
Sponsors:
Bioray Laboratories - Bangyao Biotechnology 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

Beta-Thalassemia, BT, (NCT05442346)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT06024876)
Sponsors:
CorrectSequence Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Beta-Thalassemia, BT, (NCT06328764)
Sponsors:
CorrectSequence Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (2018-001320-19)
Sponsors:
Vertex Pharmaceuticals Incorporated

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

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT03745287)
Sponsors:
Vertex Pharmaceuticals

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:
Mark Walters, MD

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04819841)
Sponsors:
Kamau Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT04853576)
Sponsors:
Editas Medicine, Inc.

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT05329649)
Sponsors:
Vertex Pharmaceuticals Incorporated

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT05456880)
Sponsors:
Beam Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Sickle cell disease, SCD, (NCT05951205)
Sponsors:
Vertex Pharmaceuticals Incorporated

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

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, (NCT06565026)
Sponsors:
CorrectSequence Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion-Dependent Beta-thalassemia, TDT, (NCT05145062)
Sponsors:
Sangamo Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion-Dependent Beta-Thalassemia, TDT, (NCT05477563)
Sponsors:
Vertex Pharmaceuticals Incorporated

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

IND Enabling

Phase I

Phase II

Phase III

Transfusion-Dependent Beta-Thalassaemia, TDT, (ChiCTR2100053406)
Sponsors:
The First Affiliated Hospital, Guangxi Medical University

IND Enabling

Phase I

Phase II

Phase III

Transfusion-Dependent Beta-Thalassaemia, TDT, (NCT04925206)
Sponsors:
EdiGene (GuangZhou) 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

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, (NCT05015920)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Transfusion-Dependent Beta-Thalassemia, TDT, (NCT05356195)
Sponsors:
Vertex Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Transfusion-Dependent Beta-Thalassemia, TDT, (NCT05444894)
Sponsors:
Editas Medicine

IND Enabling

Phase I

Phase II

Phase III

Transfusion-Dependent Beta-Thalassemia, TDT, (NCT05577312)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Transfusion-Dependent Beta-Thalassemia, TDT, (NCT05752123)
Sponsors:
EdiGene (GuangZhou) Inc.

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

IND Enabling

Phase I

Phase II

Phase III

Transfusion-dependent Beta-Thalassemia, TDT, (NCT06291961)
Sponsors:
CorrectSequence Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Transfusion-dependent Beta-thalassemia, TDT, (NCT06298630)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Haemophilia

Hereditary amyloidosis

Transthyretin Amyloidosis Cardiomyopathy, TTR, (NCT06082050)
Sponsors:
YolTech Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Transthyretin Amyloidosis with Polyneuropathy, ATTRv-PN, (NCT06672237)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Transthyretin Amyloidosis, ATTR, (ChiCTR2400081216)
Sponsors:
AccurEdit Therapeutics

IND Enabling

Phase I

Phase II

Phase III

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

IND Enabling

Phase I

Phase II

Phase III

Transthyretin Amyloidosis, ATTR, (NCT06128629)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Transthyretin Amyloidosis, ATTR, (NCT06539208)
Sponsors:
YolTech Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Immunodeficiencies

Chronic Granulomatous Disease, CGD, (NCT06325709)
Sponsors:
National Institute of Allergy and Infectious Diseases (NIAID)

IND Enabling

Phase I

Phase II

Phase III

Hereditary Angioedema, HAE, (NCT05120830)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Hereditary Angioedema, HAE, (NCT06634420)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Inherited eye diseases

ABCA4 Retinopathy, (NCT06467344)
Sponsors:
Ascidian Therapeutics, Inc

IND Enabling

Phase I

Phase II

Phase III

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

IND Enabling

Phase I

Phase II

Phase III

Neovascular Age-related Macular Degeneration, nAMD, (NCT06031727)
Sponsors:
HuidaGene Therapeutics Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Neovascular Age-Related Macular Degeneration, nAMD, (NCT06623279)
Sponsors:
HuidaGene Therapeutics Co., Ltd.

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

IND Enabling

Phase I

Phase II

Phase III

Metabolic disorders

Glycogen Storage Disease Type Ia, GSDIa, (NCT06735755)
Sponsors:
Beam Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis 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

Ornithine Transcarbamylase Deficiency, OTC, (NCT06255782)
Sponsors:
iECURE, Inc.

IND Enabling

Phase I

Phase II

Phase III

Phenylketonuria, PKU, (NCT05222178)
Sponsors:
Homology Medicines

IND Enabling

Phase I

Phase II

Phase III

Refractory Dyslipidemias, (ACTRN12623000809639)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Type 1 Diabetes, T1D, (NCT05210530)
Sponsors:
CRISPR Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Type 1 Diabetes, T1D, (NCT05565248)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Type 1 Primary Hyperoxaluria, PH1, (NCT06511349)
Sponsors:
RenJi Hospital

IND Enabling

Phase I

Phase II

Phase III

Muscular dystrophies

Duchenne Muscular Dystrophy, DMD, (NCT05514249)
Sponsors:
Cure Rare Disease, Inc

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

Limb Girdle Muscular Dystrophy, LGMD, (NCT05588401)
Sponsors:
Simone Spuler, MD

IND Enabling

Phase I

Phase II

Phase III

Neurological disease

Amyotrophic Lateral Sclerosis, ALS, and Frontotemporal Dementia, FTD, (NCT05683860)
Sponsors:
Wave Life Sciences Ltd.

IND Enabling

Phase I

Phase II

Phase III

Amyotrophic Lateral Sclerosis, ALS, or Frontotemporal Dementia FTD, (NCT04931862)
Sponsors:
Wave Life Sciences Ltd.

IND Enabling

Phase I

Phase II

Phase III

Huntington's Disease, (NCT05032196)
Sponsors:
Wave Life Sciences Ltd.

IND Enabling

Phase I

Phase II

Phase III

Solid cancers

Advanced Hepatocellular Carcinoma, HCC, (NCT04417764)
Sponsors:
Central South University

IND Enabling

Phase I

Phase II

Phase III

Advanced or metastatic melanoma, (2023-510417-25-00)
Sponsors:
Herlev Hospital

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

IND Enabling

Phase I

Phase II

Phase III

B-Cell Non-Hodgkin Lymphoma, NHL, (NCT05607420)
Sponsors:
Cellectis S.A.

IND Enabling

Phase I

Phase II

Phase III

Breast Cancer, BC, (NCT05812326)
Sponsors:
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

IND Enabling

Phase I

Phase II

Phase III

Castration-Resistant Prostate Cancer, CRPC, (NCT02867345)
Sponsors:
Peking University

IND Enabling

Phase I

Phase II

Phase III

Castration-Resistant Prostate Cancer, CRPC, (NCT04768608)
Sponsors:
Zhejiang University

IND Enabling

Phase I

Phase II

Phase III

Castration-Resistant Prostate Cancer, CRPC, (NCT06228404)
Sponsors:
Shanghai Changzheng Hospital

IND Enabling

Phase I

Phase II

Phase III

Castration-Resistant Prostate Cancer, CRPC, and Salivary Gland Cancer, SGC, (NCT04249947)
Sponsors:
Poseida Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Epithelial Ovarian Cancer, EOC, (NCT05617755)
Sponsors:
Arsenal Biosciences, Inc.

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 Melanoma, (NCT06783270)
Sponsors:
Inge Marie Svane

IND Enabling

Phase I

Phase II

Phase III

Muscle-Invasive Bladder Cancer, MIBC, (NCT02863913)
Sponsors:
Peking University

IND Enabling

Phase I

Phase II

Phase III

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

IND Enabling

Phase I

Phase II

Phase III

Non-small Cell Lung Cancer, NSCLC, (NCT03525782)
Sponsors:
First Affiliated Hospital of Guangdong Pharmaceutical University

IND Enabling

Phase I

Phase II

Phase III

Non-small Cell Lung Cancer, NSCLC, (NCT05566223)
Sponsors:
Intima Bioscience, Inc.

IND Enabling

Phase I

Phase II

Phase III

Prostate Cancer, (NCT03525652)
Sponsors:
The First Affiliated Hospital of Guangdong Pharmaceutical University

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory B-Cell Lymphoma, BCL, (NCT05950334)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Renal Cell Carcinoma, RCC, (NCT04438083)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Relapsed or Refractory Solid Tumors, (NCT05795595)
Sponsors:
CRISPR Therapeutics AG

IND Enabling

Phase I

Phase II

Phase III

Renal Cell Carcinoma, RCC, (NCT02867332)
Sponsors:
Peking University

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

Renal Cell Carcinoma, RCC, (NCT06245915)
Sponsors:
Arsenal Biosciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Solid Tumor Adult, (NCT03545815)
Sponsors:
Chinese PLA General Hospital

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

Solid Tumor, Adult, (NCT04976218)
Sponsors:
Chinese PLA General Hospital

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT03970382)
Sponsors:
PACT Pharma, Inc.

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT04551885)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT04842812)
Sponsors:
Second Affiliated Hospital of Guangzhou Medical University

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT05239143)
Sponsors:
Poseida Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT05361174)
Sponsors:
Iovance Biotherapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT05395052)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT06237881)
Sponsors:
M.D. Anderson Cancer Center

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT06241456)
Sponsors:
Fate Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Solid Tumors, (NCT06598371)
Sponsors:
M.D. Anderson Cancer Center

IND Enabling

Phase I

Phase II

Phase III

Viral diseases

Chronic Hepatitis B, HBV, (NCT06671093)
Sponsors:
Tune Therapeutics, Inc.

IND Enabling

Phase I

Phase II

Phase III

Chronic Hepatitis B, HBV, (NCT06680232)
Sponsors:
Precision BioSciences, 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

Herpes Simplex Virus Type I Stromal Keratitis, (NCT06474416)
Sponsors:
Shanghai BDgene Co., Ltd.

IND Enabling

Phase I

Phase II

Phase III

Herpes Simplex Virus Type I Stromal Keratitis, (NCT06474442)
Sponsors:
Shanghai BDgene Co., Ltd.

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, (NCT02388594)
Sponsors:
University of Pennsylvania

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:
University of Cincinnati

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT05144386)
Sponsors:
Excision BioTherapeutics

IND Enabling

Phase I

Phase II

Phase III

Induced Long-term Immunity in COVID-19 Patients, (NCT04990557)
Sponsors:
Mahmoud Ramadan mohamed Elkazzaz

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

IND Enabling

Phase I

Phase II

Phase III

Other rare inherited disorders

Alpha 1-Antitrypsin Deficiency, AATD, (NCT06389877)
Sponsors:
Beam Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Alpha 1-Antitrypsin Deficiency, AATD, (NCT06677307)
Sponsors:
Korro Bio

IND Enabling

Phase I

Phase II

Phase III

Alpha-1 Antitrypsin Deficiency, AATD, (NCT06186492)
Sponsors:
Wave Life Sciences Ltd.

IND Enabling

Phase I

Phase II

Phase III

Alpha-1 Antitrypsin Deficiency, AATD, (NCT06405633)
Sponsors:
Wave Life Sciences Ltd.

IND Enabling

Phase I

Phase II

Phase III

Alpha-1 Antitrypsin Deficiency, AATD, (NCT06622668)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Congenital Hearing Loss, (NCT06025032)
Sponsors:
HuidaGene Therapeutics Co., Ltd.

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

Acute Myeloid Leukaemia, AML, (NCT05309733)
Sponsors:
Vor Biopharma

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

Hemophilia B & Mucopolysaccharidosis, MPS, (NCT04628871)
Sponsors:
Sangamo Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Hereditary Angioedema, HAE, (NCT06262399)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT04201782)
Sponsors:
Sangamo Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Human Immunodeficiency Virus Infection, HIV, (NCT05143307)
Sponsors:
Excision BioTherapeutics

IND Enabling

Phase I

Phase II

Phase III

Participants Who Received an Allogeneic Chimeric Antigen Receptor T-Cell Product (PBCAR) in a Precision BioSciences, Inc., Clinical Study (NCT05822427)
Sponsors:
Precision BioSciences, Inc.

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion Dependent Beta-Thalassemia, TDT, (NCT06363760)
Sponsors:
Editas Medicine, Inc.

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion-Dependent Beta-Thalassemia, TDT, (NCT04208529)
Sponsors:
Vertex Pharmaceuticals

IND Enabling

Phase I

Phase II

Phase III

Sickle Cell Disease, SCD, and Transfusion-Dependent Beta-thalassemia, TDT, (NCT05145062)
Sponsors:
Sangamo Therapeutics

IND Enabling

Phase I

Phase II

Phase III

Transfusion-dependent Beta-thalassemia, TDT, (NCT06314529)
Sponsors:
Bioray Laboratories

IND Enabling

Phase I

Phase II

Phase III

Transthyretin Amyloidosis, ATTR, (NCT05697861)
Sponsors:
Intellia Therapeutics

IND Enabling

Phase I

Phase II

Phase III