14 completed, 5 recruiting, 4 not yet recruiting, 3 active not recruiting. 17 Phase 1, 17 Phase 2, 2 Early Phase 1, 2 Phase 3.
Follow Genetic Therapy: new trials by email Literature Review of Genetic Therapy trials Research Report on Genetic Therapy
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus gene therapy - Parkinson's Disease (PD) | Early Phase 1 | Not yet recruiting | Second Affiliated Hospital, Zhejiang University, School of Medicine | 6 | 2026-05-01 | 2026-04-24 |
| A Phase I Study of CREPT-618 in Locally Advanced HCC gene therapy - Hepatocellular Carcinoma (HCC) | Phase 1 | Recruiting | Cancer Institute and Hospital, Chinese Academy of Medical Sciences | 13 | 2025-09-15 | 2026-01-23 |
| A Clinical Trial of CAP-003 Gene Therapy in Adult Patients With GBA1 Associated Parkinson's Disease gene therapy - GBA1 Parkinson Disease | Phase 1 / Phase 2 | Withdrawn | Capsida Biotherapeutics, Inc. | 2025-08-18 | 2026-06-22 | |
| A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease gene therapy, clozapine - Parkinson Disease (PD), Gene Therapy | Early Phase 1 | Not yet recruiting | Ruijin Hospital | 6 | 2025-08-01 | 2025-07-25 |
| A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy gene therapy - Developmental and Epileptic Encephalopathy | Phase 1 / Phase 2 | Terminated | Capsida Biotherapeutics, Inc. | 1 | 2025-07-03 | 2026-06-10 |
| Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A Gene therapy, Biological - Hemophilia A | Phase 1 | Not yet recruiting | Expression Therapeutics, LLC | 7 | 2024-09-01 | 2024-02-20 |
| Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure AB-1002 - Congestive Heart Failure | Phase 2 | Active not recruiting | AskBio Inc | 150 | 2023-10-20 | 2025-12-03 |
| An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3 Gene therapy, Enzyme Replacement Agent - Gaucher Disease, Type 3 | Phase 2 / Phase 3 | Withdrawn | AVROBIO | 2023-10-01 | 2023-08-09 | |
| Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy Gene therapy - Transfusion-dependent Beta-Thalassemia, Gene Therapy | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 100 | 2023-06-05 | 2023-08-14 | |
| The World Federation of Hemophilia Gene Therapy Registry Gene therapy - Hemophilia | Not yet recruiting | World Federation of Hemophilia | 5000 | 2022-01-01 | 2021-05-12 | |
| Gene Therapy for IGHMBP2-Related Diseases Gene Therapy - SMARD1, CMT2S | Phase 1 / Phase 2 | Enrolling by invitation | Megan Waldrop | 10 | 2021-11-04 | 2026-10-30 |
| Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID Gene therapy - Severe Combined Immunodeficiency Due to RAG1 Deficiency | Phase 1 / Phase 2 | Suspended | Videja B.V. | 10 | 2021-07-23 | 2026-07-16 |
| Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy - Hemophilia A | Phase 3 | Active not recruiting | Pfizer | 77 | 2020-08-18 | 2026-08-19 |
| A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy - Homozygous Familial Hypercholesterolemia (HoFH) | Unknown | REGENXBIO Inc. | 8 | 2019-09-30 | 2023-03-09 | |
| PDE6A Gene Therapy for Retinitis Pigmentosa subretinal injection of rAAV.hPDE6A - Retinitis Pigmentosa | Phase 1 / Phase 2 | Active not recruiting | STZ eyetrial | 9 | 2019-09-24 | 2024-04-18 |
| Phase II High Risk Prostate Cancer Trial Using Gene & Androgen Deprivation Therapies, Radiotherapy, & Surgery HSV-Tk, Valacyclovir, Bicalutamide - High-risk Prostate Cancer, Prostate Cancer | Phase 2 | Recruiting | The Methodist Hospital Research Institute | 60 | 2018-08-02 | 2021-04-06 |
| HSV-tk and XRT and Chemotherapy for Newly Diagnosed GBM ADV/HSV-tk (gene therapy) - Glioblastoma, Anaplastic Astrocytoma | Phase 1 / Phase 2 | Recruiting | The Methodist Hospital Research Institute | 62 | 2018-02-28 | 2026-03-30 |
| HSV-tk + Valacyclovir + SBRT + Chemotherapy for Recurrent GBM ADV/HSV-tk (gene therapy) - Glioblastoma Multiforme, Astrocytoma, Grade III | Phase 1 / Phase 2 | Recruiting | David Baskin MD | 62 | 2017-12-11 | 2026-03-30 |
| A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111 BIIB111 - Choroideremia | Phase 2 | Completed | Biogen | 66 | 2017-11-29 | 2024-02-23 |
| Molecular Characterization of Primary Mucosal Melanoma Somatic Genetic - Mucosal Melanoma | Completed | CHU de Reims | 65 | 2017-08-28 | 2024-08-22 |
Showing 20 of 32. Sign in to see all of them.
Astrocytoma (6) · Glioblastoma (6) · Brain Neoplasms (4) · Glioma (3) · Gliosarcoma (3) · Hemophilia A (3) · Oligodendroglioma (3) · Parkinson Disease (3) · Carcinoma, Hepatocellular (2) · Central Nervous System Neoplasms (2) · Hyperlipoproteinemia Type II (2) · Rare Diseases (2) · Aromatic amino acid decarboxylase deficiency (1) · Atherosclerosis (1) · Cardiovascular Diseases (1)
Capsida Biotherapeutics, Inc. (2) · The Methodist Hospital Research Institute (2) · AVROBIO (1) · Abramson Cancer Center at Penn Medicine (1) · AskBio Inc (1) · Beijing Chao Yang Hospital (1) · Biogen (1) · CHU de Reims (1) · Cancer Institute and Hospital, Chinese Academy of Medical Sciences (1) · City of Hope Medical Center (1) · David Baskin MD (1) · Expression Therapeutics, LLC (1) · Fondazione Telethon (1) · H. Lee Moffitt Cancer Center and Research Institute (1) · Helixmith Co., Ltd. (1)
United States (17) · China (5) · Germany (4) · Canada (3) · France (3) · Netherlands (3) · Spain (3) · Italy (2) · Poland (2) · South Korea (2) · Taiwan (2) · Turkey (Türkiye) (2) · United Kingdom (2) · Australia (1) · Austria (1)
Melanie Brandabur, MD (2) · Arjan C Lankester, Prof.dr. (1) · Asha Das, MD (1) · Bordignon Claudio, MD (1) · David S Baskin, MD (1) · David S. Baskin, MD (1) · Ding Ma, M.D. (1) · Dominik Fischer, Prof. (1) · E. Brian Butler, MD (1) · Fondazione Telethon (1) · Jack Kessler, MD (1) · James Croop, MD, PhD (1) · James M. Wilson (1) · Jana Portnow (1) · Jane B. Alavi, MD (1)
PMID 18218852, PMID 19179314, PMID 19633200, PMID 19652199, PMID 22184407, PMID 26000320, PMID 27129325, PMID 28319446, PMID 30169182, PMID 30194931, PMID 32322605, PMID 34763085, PMID 38355973, PMID 38970425, PMID 39878701
2017 (1), 2019 (1), 2023 (1)