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ClinicalTrials.gov public records Last synced May 21, 2026, 7:16 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 700 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Gene Transfer Clear all
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Immunoglobulin A (IgA) Nephropathy
Interventions
PS-002
Genetic
Lead sponsor
Purespring Therapeutics Limited
Industry
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
3
States / cities
Miami, Florida • Baltimore, Maryland • Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Mar 19, 2026 · Synced May 21, 2026, 7:16 PM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, Plasmapheresis
Genetic · Procedure
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced May 21, 2026, 7:16 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
SMA - Spinal Muscular Atrophy, Gene Therapy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 180 Days
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
16
States / cities
Los Angeles, California • Stanford, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced May 21, 2026, 7:16 PM EDT
Completed Phase 2Phase 3 Interventional Results available
View directory record Official record
Conditions
Cerebral Adrenoleukodystrophy (CALD)
Interventions
Lenti-D Drug Product (eli-cel)
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Up to 17 Years · Male only
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2021
U.S. locations
3
States / cities
Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 24, 2022 · Synced May 21, 2026, 7:16 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Wiskott-Aldrich Syndrome
Interventions
OTL-103
Genetic
Lead sponsor
Fondazione Telethon
Other
Eligibility
Up to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced May 21, 2026, 7:16 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Hemophilia B
Interventions
Qualitative Interview - Participants/ Patients, Qualitative Interview - Investigators and Study Coordinators
Other
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 75 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 22, 2021 · Synced May 21, 2026, 7:16 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Catecholaminergic Polymorphic Ventricular Tachycardia
Interventions
SGT-501
Drug
Lead sponsor
Solid Biosciences Inc.
Industry
Eligibility
7 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
4
States / cities
Boston, Massachusetts • Rochester, Minnesota • Cleveland, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced May 21, 2026, 7:16 PM EDT
Recruiting Phase 1 Interventional
View directory record Official record
Conditions
Gastric Cancer, Breast Cancer, Cervical Cancer, Lung Cancer
Interventions
KK-LC-1 TCR-T cells, Aldesleukin
Biological · Drug
Lead sponsor
Christian Hinrichs
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
2
States / cities
New Brunswick, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced May 21, 2026, 7:16 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Wilson Disease
Interventions
UX701, Standard of Care (SOC)
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2034
U.S. locations
11
States / cities
Los Angeles, California • Redwood City, California • Sacramento, California + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced May 21, 2026, 7:16 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Rett Syndrome
Interventions
TSHA-102
Genetic
Lead sponsor
Taysha Gene Therapies, Inc.
Industry
Eligibility
5 Years to 8 Years · Female only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
3
States / cities
Chicago, Illinois • Saint Paul, Minnesota • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced May 21, 2026, 7:16 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Chronic Lymphocytic Leukemia, B-Cell Chronic Lymphocytic Leukemia, Lymphoma, B-Cell
Interventions
Anti-CD19 and anti-CD20 bicistronic CAR T- cells, Cyclophosphamide, Fludarabine
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years to 75 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 3, 2026 · Synced May 21, 2026, 7:16 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Hemophilia A
Interventions
Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced May 21, 2026, 7:16 PM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Metastatic Cancer, Metastatic Renal Cancer, Metastatic Melanoma
Interventions
PG13-MAGE-A3 TCR9W11 (anti-MAGE-A3/12 TCR) Transduced Autologous Peripheral Blood Lymphocytes, Aldesleukin, Cyclophosphamide, Fludarabine
Biological · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 27, 2015 · Synced May 21, 2026, 7:16 PM EDT
Terminated Phase 1 Interventional Results available
View directory record Official record
Conditions
Colorectal Cancer
Interventions
GVAX
Biological
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 11, 2023 · Synced May 21, 2026, 7:16 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
HIV-1-infection
Interventions
EBT-101
Biological
Lead sponsor
Excision BioTherapeutics
Industry
Eligibility
18 Years to 70 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
3
States / cities
San Francisco, California • St Louis, Missouri • Camden, New Jersey
Source: ClinicalTrials.gov public record
Updated Dec 1, 2024 · Synced May 21, 2026, 7:16 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 7:16 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Solid Tumor, Adult, Colorectal Cancer, NSCLC, Non Small Cell Lung Cancer, NSCLC, Recurrent, Non-Small Cell Squamous Lung Cancer, Pancreas Cancer, Pancreatic Neoplasm, Colorectal Adenocarcinoma, CRC, Colon Cancer, Rectal Cancer, Cancer, Ovarian Cancer, Ovarian Neoplasms, Mesothelioma, Mesothelioma, Malignant, Ovary Cancer, Lung Cancer, MESOM
Interventions
A2B694, A2B543, xT CDx with HLA-LOH Assay
Biological · Diagnostic Test
Lead sponsor
A2 Biotherapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
474 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
12
States / cities
Gilbert, Arizona • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced May 21, 2026, 7:16 PM EDT
Withdrawn Phase 1 Interventional
View directory record Official record
Conditions
Neuroblastoma
Interventions
cyclophosphamide, Fludarabine, GINAKIT Cells
Drug · Genetic
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Year to 18 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 13, 2017 · Synced May 21, 2026, 7:16 PM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Healthy Adults
Interventions
Injectable Plasmid Klotho Gene Therapy
Genetic
Lead sponsor
Minicircle
Industry
Eligibility
23 Years to 90 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Feb 16, 2026 · Synced May 21, 2026, 7:16 PM EDT
Enrolling by invitation No phase listed Observational
View directory record Official record
Conditions
Mucopolysaccharidosis I
Interventions
No Intervention
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced May 21, 2026, 7:16 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
X-Linked Retinitis Pigmentosa
Interventions
rAAV2tYF-GRK1-RPGR
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
8 Years to 50 Years · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
6
States / cities
Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 11, 2024 · Synced May 21, 2026, 7:16 PM EDT
Terminated Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Cystic Fibrosis
Interventions
tgAAVCF
Genetic
Lead sponsor
Targeted Genetics Corporation
Industry
Eligibility
12 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
11
States / cities
Birmingham, Alabama • Palo Alto, California • San Diego, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jan 27, 2008 · Synced May 21, 2026, 7:16 PM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
BAG3 Mutation Associated Dilated Cardiomyopathy
Interventions
ALXN2350
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
5
States / cities
Birmingham, Alabama • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced May 21, 2026, 7:16 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Hemophilia A
Interventions
valoctocogene roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
3
States / cities
Sacramento, California • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced May 21, 2026, 7:16 PM EDT