Search clinical trials for: thérapie génique

Total 25794 results

Ocugen

Recruiting

A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa (liMeliGhT)

Retinitis Pigmentosa

United States
Sensorion

Not yet recruiting

A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations (AUDIOGENE)

Otorhinolaryngologic Diseases | Ear Diseases | Hearing Disorders | Deafness | Hearing Loss, Sensorineural | Congenital Deafness | DFNB9 | OTOF Gene Mutation

France
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
National Cancer Institute (NCI)

Not yet recruiting

Anti-CD19 Chimeric Antigen Receptor T-Cell Immunotherapy for Chronic Lymphocytic Leukemia (CLL)

Leukemia, Lymphocytic, Chronic, B-Cell | B-Cell Chronic Lymphocytic Leukemia | B-Lymphocytic Leukemia, Chronic

United States
University Hospital Tuebingen

Recruiting

Natural History Study in Patients With PDE6A-, PDE6B- and RHO-linked Retinitis Pigmentosa

Retinitis Pigmentosa

Germany
Tenaya Therapeutics
Medical University of South Carolina; Johns Hopkins University; University of... and other collaborators

Recruiting

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) (RIDGE)

Arrhythmogenic Right Ventricular Cardiomyopathy

United States, United Kingdom, France, Italy
Spark Therapeutics, Inc.

Recruiting

Study of a Gene Therapy Treatment for Hemophilia A (KEYSTONE 1)

Hemophilia A

United States
Zhongmou Therapeutics

Not yet recruiting

Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients (ZM-02)

Retinitis Pigmentosa

China
Suzhou Municipal Hospital

Not yet recruiting

Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients

Familial Hypercholesterolemia | Refractory Hypercholesterolemia
ViGeneron GmbH

Recruiting

Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene

Retinitis Pigmentosa

Germany
Asklepios Biopharmaceutical, Inc.
Bayer

Not yet recruiting

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD) (REGENERATE-PD)

Parkinson Disease
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
Xinhua Hospital, Shanghai Jiao Tong University...
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Type II Gaucher Disease

China
UniQure Biopharma B.V.

Not yet recruiting

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Fabry Disease
National Cancer Institute (NCI)

Recruiting

Autologous T-cells Genetically Engineered to Express Receptors Reactive Against KRAS Mutations in Conjunction With a Vaccine Directed Against These Antigens in Participants With Metastatic Cancer

Breast Cancer | Colorectal Cancer | Ovarian Cancer | Non-Small Cell Lung Cancer | Gastrointestinal Cancer | Genitourinary Cancer | Metastatic Solid Cancers

United States
Eye & ENT Hospital of Fudan University
Shanghai Rehabilitation Institute for the Exceptional Children

Not yet recruiting

A Comparative Analysis of Speech Perception Between Cochlear Implant Patients and DFNB9 Patients Receiving Gene Therapy

Hearing Loss | Genetic Disease | Speech Perception

China
Tenaya Therapeutics
Johns Hopkins University; Mayo Clinic

Recruiting

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC (RIDGE-1)

Arrhythmogenic Right Ventricular Cardiomyopathy

United States
BioMarin Pharmaceutical

Recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Hemophilia A

Japan
Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b

Glutaric Acidemia Type I | Glutaric Aciduria Type I

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
Grace Science, LLC

Recruiting

Safety and Efficacy of GS-100 Gene Therapy in Patients With NGLY1 Deficiency

NGLY1 Deficiency

United States
AstraZeneca

Not yet recruiting

LTFU for All Cell and Gene Therapy Studies (LOCUS)

Hepatocellular Carcinoma | Prostate Cancer

United States
Novartis Pharmaceuticals

Not yet recruiting

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

Sickle Cell Disease
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
Solid Biosciences Inc.

Recruiting

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Duchenne Muscular Dystrophy

United States
First Affiliated Hospital Xi'an Jiaotong University

Recruiting

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

China
West China Hospital

Recruiting

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

China
Shanghai Belief-Delivery BioMed Co., Ltd
Shanghai MYGT Biopharmaceutical LLC

Recruiting

Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

Hemophilia A

China
Lexeo Therapeutics

Recruiting

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Arrhythmogenic Cardiomyopathy | PKP2-ACM | PKP2-ARVC

United States
The 923rd Hospital of Joint Logistics Support Force...
Guangzhou Reforgene Medicine Co., Ltd.

Recruiting

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Hemoglobinopathies | Hereditary Diseases | Alpha Thalassemia Hemoglobin H Constant Spring

China
UniQure Biopharma B.V.

Not yet recruiting

Safety, Tolerability, and Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Amyotrophic Lateral Sclerosis (ALS) Patients With SOD1 Mutations

Amyotrophic Lateral Sclerosis
Zhongmou Therapeutics

Recruiting

Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients

X-linked Retinoschisis

China
HuidaGene Therapeutics Co., Ltd.
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Recruiting

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial (LIGHT)

Leber Congenital Amaurosis

China
AviadoBio Ltd

Recruiting

A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN) (ASPIRE-FTD)

Frontotemporal Dementia | FTD | FTD-GRN | Dementia, Frontotemporal

Netherlands, Spain, Poland
A2 Biotherapeutics Inc.
Tempus AI

Recruiting

A Study to Evaluate the Safety and Efficacy of A2B694, a Logic-gated CAR T, in Subjects With Solid Tumors That Express MSLN and Have Lost HLA-A*02 Expression (EVEREST-2)

Cancer | Colorectal Cancer | Ovarian Neoplasms | Ovarian Cancer | NSCLC | Lung Cancer | Rectal Cancer | Non Small Cell Lung Cancer | Mesothelioma | Mesothelioma, Malignant | Colon Cancer | Pancreas Cancer | Colorectal Adenocarcinoma | Solid Tumor, Adult | CRC | Pancreatic Neoplasm | Ovary Cancer | NSCLC, Recurrent | Non-Small Cell... and other conditions

United States
Shanghai General Hospital, Shanghai Jiao Tong University...

Not yet recruiting

An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA

To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)
Institute of Hematology & Blood Diseases Hospital

Recruiting

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Gene Therapy | Transfusion-dependent Beta-Thalassemia

China
Neuracle Genetics, Inc
ORA, Inc.

Recruiting

NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration

Age-Related Macular Degeneration

Canada
Beijing Northland Biotech. Co., Ltd.

Recruiting

Angiogenesis and Blood Perfusion Effect of HGF Gene Therapy in PAD Patients

Peripheral Artery Disease

China
St. Jude Children's Research Hospital

Not yet recruiting

Liver Biopsy Following Gene Therapy For Hemophilia

Hemophilia A | Hemophilia B

United States
4D Molecular Therapeutics

Recruiting

4D-150 in Patients With Diabetic Macular Edema

Diabetic Retinopathy | Diabetic Macular Edema

United States, Puerto Rico
Affiliated Hospital of Guangdong Medical University

Recruiting

Safety and Efficacy Study of KL-7SHRNA Injection Solution in the Treatment of AIDS Patients With Lymphoma

HIV Infections | Lymphoma

China
Rocket Pharmaceuticals Inc.

Recruiting

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

United States
Tenaya Therapeutics

Recruiting

Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM (MyPEAK-1)

Hypertrophic Cardiomyopathy

United States
Genascence Corporation
California Institute for Regenerative Medicine (CIRM)

Recruiting

Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis (DONATELLO)

Knee Osteoarthritis | Osteoarthritis, Knee | Osteo Arthritis Knee

United States
Xiangya Hospital of Central South University

Not yet recruiting

Safety and Efficacy Study of Parkinson's Disease Gene Therapy Drug (BBM-P002)

Parkinson's Disease

China
National Cancer Institute (NCI)

Recruiting

Trial of Anti-CD19 and Anti-CD20 Bicistronic Chimeric Antigen Receptor T Cells for Treating B-Cell Malignancies

Lymphoma, B-Cell | Chronic Lymphocytic Leukemia | B-Cell Chronic Lymphocytic Leukemia

United States
Regeneron Pharmaceuticals

Recruiting

A Study of DB-OTO, an Adeno-associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin Mutations (CHORD)

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Spain, United Kingdom, United States
Shanghai Jiao Tong University School of Medicine
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

An Early Clinical Trial to Evaluate VGN-R09b for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) Deficiency.

Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

China
First Affiliated Hospital of Guangxi Medical University
Genmedicn Biopharma Ltd.

Recruiting

A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]

Transfusion Dependent Beta-Thalassemia

China

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