Search clinical trials for: gene therapy; lentiviral vector; KL003; β-thalassemia

Total 267948 results

Institute of Hematology & Blood Diseases Hospital
R&D Kanglin Biotech

Recruiting

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.

Transfusion-dependent Beta-Thalassemia

China
Institute of Hematology & Blood Diseases Hospital...
Kanglin Biotech

Recruiting

Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

Transfusion-dependent Beta-Thalassemia

China
Kanglin Biotechnology (Hangzhou) Co., Ltd.

Not yet recruiting

A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants

Transfusion-dependent Beta-Thalassemia

China
Essen Biotech

Not yet recruiting

Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease (BAH243)

Sickle Cell Disease | Sickle-Cell Disease With Crisis

China
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
Lantu Biopharma

Not yet recruiting

An Open-label Study of a Gene Therapy Product in Beta-Thalassemia

Beta-Thalassemia
Orchard Therapeutics

Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT (HURCULES)

MPS-IH (Hurler Syndrome)

Netherlands, United States, Italy, United Kingdom
Miltenyi Biomedicine GmbH

Not yet recruiting

Lentiviral Gene Therapy (Zamtocabtagene Autoleucel) LTFU

Non Hodgkin Lymphoma

United States
Shenzhen Hemogen

Recruiting

the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia

β-thalassemia

China
Shanghai BDgene Co., Ltd.
Ruijin Hospital

Not yet recruiting

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

β-thalassemia

China
Shanghai BDgene Co., Ltd.
Shanghai Children's Medical Center

Not yet recruiting

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia

β-thalassemia
Shenzhen Hemogen

Recruiting

Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients

β-thalassemia

China
University of California, Los Angeles

Recruiting

Autologous Mobilized Peripheral Blood CD34+ Hematopoietic Stem and Progenitor Cells (HSPC) Transduced With the Elongation Factor Alpha Short Promoter (EFS) - Adenosine Deaminase (ADA) Gene (EFS-ADA) Lentiviral Vector for Adenosine Deaminase Severe Combined Immune Deficiency (ADA SCID)

Adenosine Deaminase Severe Combined Immune Deficiency

United States
Pell Bio-Med Technology Co., Ltd.

Recruiting

Long-term Follow-up Study of Lentiviral-based Gene-edited Immune Cell Therapy

Diffuse Large B Cell Lymphoma | Primary Mediastinal Large B Cell Lymphoma | Large B-cell Lymphoma | Follicular Lymphoma Grade 3B | Follicular Lymphoma Grade 3A

Taiwan
David Williams
National Heart, Lung, and Blood Institute (NHLBI); bluebird bio; California Institute... and other collaborators

Recruiting

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) (GRASP)

Sickle Cell Disease

United States
Christian Medical College, Vellore, India
Dr. H. Trent Spencer, Professor, Emory University of Medicine, Atlanta Ga...

Recruiting

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

Hemophilia A

India
Bacchetta, Rosa, MD
Food and Drug Administration (FDA); California Institute for Regenerative Medicine...

Recruiting

CD4^LVFOXP3 in Participants With IPEX

IPEX

United States
Great Ormond Street Hospital for Children NHS Foundation...

Recruiting

Lentiviral Gene Therapy for p47 AR-CGD

P47-Phox, Deficiency of

United Kingdom
Assistance Publique - Hôpitaux de Paris

Recruiting

Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) (ARTEGENE)

Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency

France
Shanghai BDgene Co., Ltd.
920th Hospital of Joint Logistics Support Force of People's Liberation Army...

Recruiting

A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants

Hematologic Diseases

China
University College, London
Medical Research Council

Not yet recruiting

Lentiviral Gene Therapy for Epilepsy

Drug Resistant Epilepsy
BGI-research
Shenzhen Children's Hospital

Not yet recruiting

Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients

β-Thalassemia Major

China
Shenzhen Geno-Immune Medical Institute
Shenzhen Second People's Hospital; Shenzhen Third People's Hospital

Recruiting

Safety and Immunity of Covid-19 aAPC Vaccine

Treat and Prevent Covid-19 Infection

China
bluebird bio

Recruiting

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

Sickle Cell Disease

United States
Children's Hospital of Chongqing Medical University

Recruiting

Gene Therapy for X Linked Severe Combined Immunodeficiency

Gene Therapy

China
Shenzhen Geno-Immune Medical Institute
Shenzhen Second People's Hospital; Shenzhen Third People's Hospital

Recruiting

Immunity and Safety of Covid-19 Synthetic Minigene Vaccine

Pathogen Infection Covid-19 Infection

China
Medical College of Wisconsin

Recruiting

Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A

Hemophilia A

United States
Great Ormond Street Hospital for Children NHS Foundation...

Recruiting

Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency

Severe Combined Immunodeficiency, X-Linked

United Kingdom
University of California, San Francisco

Recruiting

Autologous Gene Therapy for Artemis-Deficient SCID

Severe Combined Immunodeficiency

United States
University of Pennsylvania
Children's Hospital of Philadelphia

Recruiting

CD22 Redirected Autologous T Cells for ALL

B Cell Lymphomas | B Cell Leukemias

United States
Donald B. Kohn, M.D.
California Institute for Regenerative Medicine (CIRM)

Recruiting

Stem Cell Gene Therapy for Sickle Cell Disease

Sickle Cell Disease

United States
University of California, Los Angeles
National Heart, Lung, and Blood Institute (NHLBI); Boston Children's Hospital; California Institute for Regenerative Medicine (CIRM) and other collaborators

Recruiting

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease

Granulomatous Disease, Chronic, X-linked

United States
National Institute of Allergy and Infectious Diseases...

Recruiting

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

X-linked Severe Combined Immunodeficiency (XSCID)

United States
National Institute of Allergy and Infectious Diseases...

Enrolling by invitation

pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

Chronic Granulomatous Disease | p47 | Autosomal Recessive

United States
David Williams

Active, not recruiting

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Gene Therapy | Severe Combined Immunodeficiency, X Linked

United States, United Kingdom
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...

Suspended

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants (LVXSCID-ND)

Severe Combined Immunodeficiency Disease, X-linked

United States
Hospital Infantil Universitario Niño Jesús, Madrid...
Universitat Autonoma de Barcelona; Instituto de Investigación Sanitaria de... and other collaborators

Completed

Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A (FANCOLEN-1)

Fanconi Anemia

Spain
University of Manchester
University College, London; Manchester University NHS Foundation Trust; Orchard... and other collaborators

Active, not recruiting

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA

Mucopolysaccharidosis Type IIIA

United Kingdom
bluebird bio

Completed

A Study Evaluating the Safety and Efficacy of bb1111 in Severe Sickle Cell Disease

Sickle Cell Disease

United States
Fondazione Telethon
Ospedale San Raffaele

Completed

Gene Therapy for Wiskott-Aldrich Syndrome (TIGET-WAS)

Wiskott-Aldrich Syndrome (WAS)

Italy
City of Hope Medical Center
National Cancer Institute (NCI); Mustang Bio, Inc.

Active, not recruiting

Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm

Acute Myeloid Leukemia | Recurrent Adult Acute Myeloid Leukemia | Secondary Acute Myeloid Leukemia | Untreated Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia in Remission | Refractory Acute Myeloid Leukemia | Blastic Plasmacytoid Dendritic Cell Neoplasm | Minimal Residual Disease | Acute... and other conditions

United States
City of Hope Medical Center
National Cancer Institute (NCI)

Active, not recruiting

Memory Enriched T Cells Following Stem Cell Transplant in Treating Patients With Recurrent B-Cell Non-Hodgkin Lymphoma

Recurrent Mantle Cell Lymphoma | Recurrent Diffuse Large B-Cell Lymphoma | Refractory Diffuse Large B-Cell Lymphoma | Refractory Mantle Cell Lymphoma | Transformed Recurrent Non-Hodgkin Lymphoma

United States
AVROBIO

Terminated

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Gaucher Disease

United States, Canada
Assistance Publique - Hôpitaux de Paris

Active, not recruiting

Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease (DREPAGLOBE) (DREPAGLOBE)

Sickle Cell Disease

France
City of Hope Medical Center
National Cancer Institute (NCI)

Active, not recruiting

Genetically Modified T-cell Infusion Following Peripheral Blood Stem Cell Transplant in Treating Patients With Recurrent or High-Risk Non-Hodgkin Lymphoma

Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma | Recurrent Adult Diffuse Large Cell Lymphoma | Recurrent Adult Diffuse Mixed Cell Lymphoma | Recurrent Adult Diffuse Small Cleaved Cell Lymphoma and other conditions

United States
AVROBIO

Terminated

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Fabry Disease

United States, Australia, Brazil
City of Hope Medical Center
National Cancer Institute (NCI)

Active, not recruiting

Cellular Immunotherapy in Treating Patients With High-Risk Acute Lymphoblastic Leukemia

B Acute Lymphoblastic Leukemia | Recurrent Acute Lymphoblastic Leukemia | Refractory Acute Lymphoblastic Leukemia

United States
M.D. Anderson Cancer Center

Completed

Modified T Cells, Chemotherapy, and Aldesleukin With or Without LV305 and CMB305 in Treating Participants With Advanced or Recurrent Sarcoma

Recurrent Synovial Sarcoma | HLA-A*0201 Positive Cells Present | NY-ESO-1 Positive Tumor Cells Present | Recurrent Myxoid Liposarcoma

United States
AVROBIO

Terminated

Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201

Fabry Disease

Australia, Brazil
David Williams

Active, not recruiting

Gene Transfer for Sickle Cell Disease

Sickle Cell Disease

United States

Search clinical trials for: gene therapy; lentiviral vector; KL003; β-thalassemia

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