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Showing 1–24 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Intervention: Gene-modified CD34+ cells Clear all
Active, not recruiting Phase 1 Interventional

Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients

NCT02500849
View directory record Official record
Conditions
HIV
Interventions
SB-728mR-HSPC Infusion 3 days following busulfan conditioning
Genetic
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2026
U.S. locations
5
States / cities
Duarte, California • Los Angeles, California • San Francisco, California + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 1Phase 2 Interventional

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

NCT03812263
View directory record Official record
Conditions
Leukocyte Adhesion Defect - Type I
Interventions
RP-L201
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
3 Months and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 14, 2023 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Stem Cell Gene Therapy for Cystinosis

NCT03897361
View directory record Official record
Conditions
Lysosomal Storage Diseases, Cystinosis
Interventions
CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST)
Genetic
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Sep 23, 2026, 2:57 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Donor Lymphocyte Infusion (DLI) of T-cells Genetically Modified With iCasp9 Suicide Gene

NCT01875237
View directory record Official record
Conditions
Leukemia, Myeloma, Myeloproliferative Diseases
Interventions
Fludarabine, Melphalan, Alemtuzumab, Stem Cell infusion, Tacrolimus, Mini Methotrexate, G-CSF, Donor Lymphocyte Infusion (DLI), AP1903, Methylprednisolone, Questionnaire
Drug · Procedure · Behavioral
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years to 65 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 15, 2019 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 2Phase 3 Interventional

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

NCT03655678
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
6
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Sep 23, 2026, 2:57 PM EDT
Recruiting Phase 3 Interventional

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
3
States / cities
New York, New York • Charlotte, North Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 22, 2026 · Synced Sep 23, 2026, 2:57 PM EDT
Recruiting Phase 1 Interventional

Gene Editing For Sickle Cell Disease

NCT06506461
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Plerixafor, Busulfan, Gene-modified CD34+ cells, Motixafortide
Drug · Biological
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
18 Years to 24 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 1 Interventional

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

NCT04105166
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
RP-L301
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years to 50 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Sep 23, 2026, 2:57 PM EDT
Active, not recruiting Phase 3 Interventional

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

NCT05329649
View directory record Official record
Conditions
Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years to 11 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
4
States / cities
Charlotte, North Carolina • Philadelphia, Pennsylvania • Memphis, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Sep 23, 2026, 2:57 PM EDT
Withdrawn Phase 1 Interventional

Gene-Modified HIV-Protected Stem Cell Transplant in Treating Patients With HIV-Associated Lymphoma

NCT02378922
View directory record Official record
Conditions
AIDS-Related Hodgkin Lymphoma, AIDS-Related Non-Hodgkin Lymphoma, HIV Infection, Recurrent Hodgkin Lymphoma, Recurrent Non-Hodgkin Lymphoma
Interventions
Gene-Modified HIV-Protected Hematopoietic Stem Cells, Laboratory Biomarker Analysis, Transplant Conditioning
Biological · Other
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years to 66 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Dec 26, 2018 · Synced Sep 23, 2026, 2:57 PM EDT
Terminated Phase 1 Interventional

Treatment of Newly Diagnosed Brain Tumors With Chemotherapy and Radiation Using Cells Modified for Chemoprotection and an Experimental Drug to Decrease the Tumor Cell Resistance to Chemotherapy

NCT00272870
View directory record Official record
Conditions
Glioblastoma Multiforme (WHO Grade IV), Anaplastic Astrocytoma (WHO Grade III)
Interventions
06Benzylguanine, MGMT P140K, Meltenyi CliniMacs
Drug · Genetic · Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
5 Years to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 28, 2012 · Synced Sep 23, 2026, 2:57 PM EDT
Suspended Phase 2 Interventional

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

NCT06422351
View directory record Official record
Conditions
Pyruvate Kinase Deficiency
Interventions
RP-L301
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced Sep 23, 2026, 2:57 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

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

NCT04145037
View directory record Official record
Conditions
Gaucher Disease
Interventions
AVR-RD-02
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
18 Years to 50 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
San Diego, California • Iowa City, Iowa • Hackensack, New Jersey + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 17, 2024 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

NCT03432364
View directory record Official record
Conditions
Transfusion Dependent Beta-thalassemia
Interventions
ST-400 Investigational product
Genetic
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 1 Interventional

Combination Chemotherapy Plus Gene Therapy in Treating Patients With CNS Tumors

NCT00005796
View directory record Official record
Conditions
Bone Marrow Suppression, Brain and Central Nervous System Tumors, Drug/Agent Toxicity by Tissue/Organ
Interventions
filgrastim, gene therapy, lomustine, procarbazine hydrochloride, vincristine sulfate, in vitro-treated peripheral blood stem cell transplantation
Procedure · Biological · Drug
Lead sponsor
Indiana University
Other
Eligibility
5 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2011
U.S. locations
2
States / cities
Indianapolis, Indiana • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced Sep 23, 2026, 2:57 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety Study of Gene Modified Donor T-cells Following Partially Mismatched Stem Cell Transplant

NCT01744223
View directory record Official record
Conditions
Acute Lymphoblastic Leukemia, Acute Myelogenous Leukemia, Lymphoma, Myelodysplastic Syndromes
Interventions
BPX-501 dose 1, Rimiducid, BPX-501 dose 2, BPX-501 dose 3, BPX-501 dose 4, SCT
Biological · Drug · Procedure
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2032
U.S. locations
8
States / cities
Atlanta, Georgia • Buffalo, New York • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2022 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 1Phase 2 Interventional

Stem Cell Gene Therapy for Sickle Cell Disease

NCT02247843
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
βAS3-FB vector transduced peripheral blood CD34+ cells
Biological
Lead sponsor
Donald B. Kohn, M.D.
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 16, 2025 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 1Phase 2 Interventional

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

NCT02234934
View directory record Official record
Conditions
Granulomatous Disease, Chronic, X-linked
Interventions
Lentiviral G1XCGD Gene Therapy
Biological
Lead sponsor
University of California, Los Angeles
Other
Eligibility
23 Months and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
3
States / cities
Los Angeles, California • Bethesda, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 1, 2025 · Synced Sep 23, 2026, 2:57 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease (CGD )

NCT06559176
View directory record Official record
Conditions
Chronic Granulomatous Disease, Granulomatous Disease, Chronic
Interventions
PM359
Biological
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
6 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
3
States / cities
Los Angeles, California • Bethesda, Maryland • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Sep 23, 2026, 2:57 PM EDT
Terminated Early Phase 1 Interventional

Gene Therapy for Chronic Granulomatous Disease

NCT00394316
View directory record Official record
Conditions
Chronic Granulomatous Disease
Interventions
Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
3 Years to 55 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2014
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 4, 2018 · Synced Sep 23, 2026, 2:57 PM EDT
Terminated Phase 1 Interventional

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

NCT04525352
View directory record Official record
Conditions
Infantile Malignant Osteopetrosis
Interventions
RP-L401
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
1 Month and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 12, 2022 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 2Phase 3 Interventional Results available

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

NCT03745287
View directory record Official record
Conditions
Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
Interventions
Exa-cel
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
9
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Sep 23, 2026, 2:57 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection

NCT01734850
View directory record Official record
Conditions
Human Immunodeficiency Virus
Interventions
Busulfan, Cal-1 modified HSPC, Cal-1 modified CD4+ T lymphocytes
Drug · Biological
Lead sponsor
Calimmune, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
2
States / cities
Los Angeles, California • San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 5, 2020 · Synced Sep 23, 2026, 2:57 PM EDT
Terminated Phase 1 Interventional

Gene Therapy for Fanconi Anemia

NCT01331018
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Bone Marrow Aspiration, Filgrastim, Genetically Engineered Hematopoietic Stem Progenitor Cells, Laboratory Biomarker Analysis, Leukapheresis, Methylprednisolone, Plerixafor, Prednisone
Procedure · Biological · Other + 1 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
4 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2024
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 16, 2024 · Synced Sep 23, 2026, 2:57 PM EDT