6 recruiting, 4 completed, 2 not yet recruiting, 1 active not recruiting. 6 Phase 1, 5 Phase 2, 3 Not applicable, 2 Early Phase 1, 1 Phase 3.
Follow Gene Therapy: new trials by email Literature Review of Gene Therapy trials Research Report on Gene 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 |
|---|---|---|---|---|---|---|
| Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47) BFB-101 (AAV9-CBh-AP4B1) - HSP, Hereditary Spastic Paraplegia | Phase 1 / Phase 2 | Not yet recruiting | BlackfinBio Ltd | 5 | 2025-08-01 | 2025-04-28 |
| 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 Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma KLN-1010 - Multiple Myeloma in Relapse, Myeloma Multiple | Phase 1 | Recruiting | Kelonia Therapeutics, Inc. | 70 | 2025-07-16 | 2026-08-28 |
| Perceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss Rehabilitation training - Congenital Hearing Loss, Gene Therapy | Not applicable | Recruiting | Eye & ENT Hospital of Fudan University | 210 | 2025-07-04 | 2025-07-28 |
| A Prospective, Single-center, Open-label Study of Inhaled BMD003 (CFTR mRNA) in Chinese Adults With Cystic Fibrosis inhaled BMD003(CFTR mRNA) - Cystic Fibrosis (CF), Gene Therapy | Early Phase 1 | Active not recruiting | Peking Union Medical College Hospital | 12 | 2025-06-27 | 2026-10-01 |
| 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 | 2026-11-04 | |
| Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy Interview - Gene Therapy, Hemophilia B | Completed | St. Jude Children's Research Hospital | 150 | 2022-01-18 | 2025-11-14 | |
| Intravitreal AAVCAGsCD59 for Advanced Dry Age-related Macular Degeneration (AMD) With Geographic Atrophy (GA) Intravitreal AAVCAGsCD59, Intravitreal Sham Injection - Dry Age-related Macular Degeneration, Geographic Atrophy | Phase 2 | Withdrawn | Hemera Biosciences | 2021-07-31 | 2021-05-10 | |
| Gene Therapy for Chinese Hemophilia A Injection of GS001 - Hemophilia A, Gene Therapy | Not applicable | Recruiting | Institute of Hematology & Blood Diseases Hospital, China | 12 | 2021-03-04 | 2025-08-01 |
| Gene Therapy for X Linked Severe Combined Immunodeficiency Lentiviral Vector Gene Therapy - Gene Therapy | Not applicable | Unknown | Children's Hospital of Chongqing Medical University | 10 | 2020-05-01 | 2020-03-27 |
| Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors SPK-8016 - Adeno-Associated Virus (AAV), Blood Coagulation Disorder | Phase 1 / Phase 2 | Completed | Spark Therapeutics, Inc. | 4 | 2019-01-30 | 2024-02-23 |
| Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 Valoctocogene Roxaparvovec - Hemophilia A, Gene Therapy | Phase 1 / Phase 2 | Terminated | BioMarin Pharmaceutical | 3 | 2018-04-24 | 2025-08-22 |
| Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning autologous CD34+ cell transduced with G2SCID vector - Severe Combined Immunodeficiency, X Linked, Gene Therapy | Phase 1 / Phase 2 | Recruiting | David Williams | 12 | 2018-02-26 | 2025-12-11 |
| Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 Onasemnogene Abeparvovec-xioi - SMA - Spinal Muscular Atrophy, Gene Therapy | Phase 3 | Completed | Novartis Gene Therapies | 22 | 2017-10-24 | 2026-01-26 |
| Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD) AAV2-sFLT01 - Macular Degeneration, Age-Related Maculopathies | Phase 1 | Completed | Genzyme, a Sanofi Company | 19 | 2010-01-11 | 2018-08-22 |
| CIBMTR Research Database Autologous Stem Cell Transplantation, Allogeneic Stem Cell Transplantation | Recruiting | Center for International Blood and Marrow Transplant Research | 99999999 | 2002-07-01 | 2025-08-12 |
Hematologic Diseases (3) · Hemophilia A (3) · Blood Coagulation Disorders (2) · Blood Coagulation Disorders, Inherited (2) · Coagulation Protein Disorders (2) · Genetic Diseases, Inborn (2) · Hemorrhagic Disorders (2) · Hemostatic Disorders (2) · X-Linked Combined Immunodeficiency Diseases (2) · Blood Protein Disorders (1) · Cystic Fibrosis (1) · Disease (1) · Eye Diseases (1) · Genetic Diseases, X-Linked (1) · Geographic Atrophy (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (8) · Genetic Diseases, Inborn (6) · Hemic and Lymphatic Diseases (6) · Central Nervous System Diseases (3) · Genetic Diseases, X-Linked (3) · Hematologic Diseases (3) · Hemorrhagic Disorders (3) · Infant, Newborn, Diseases (3) · Nervous System Diseases (3) · Neurodegenerative Diseases (3) · Pathological Conditions, Signs and Symptoms (3) · Blood Coagulation Disorders (2) · Blood Coagulation Disorders, Inherited (2) · Cardiovascular Diseases (2) · Coagulation Protein Disorders (2)
Genetic Therapy (2) · Clozapine (1) · Interviews as Topic (1) · Valoctocogene Roxaparvovec (1) · Zolgensma (1) · salicylhydroxamic acid (1)
Institute of Hematology & Blood Diseases Hospital, China (2) · BioMarin Pharmaceutical (1) · BlackfinBio Ltd (1) · Center for International Blood and Marrow Transplant Research (1) · Children's Hospital of Chongqing Medical University (1) · David Williams (1) · Eye & ENT Hospital of Fudan University (1) · Genzyme, a Sanofi Company (1) · Hemera Biosciences (1) · Kelonia Therapeutics, Inc. (1) · Novartis Gene Therapies (1) · Peking Union Medical College Hospital (1) · Ruijin Hospital (1) · Spark Therapeutics, Inc. (1) · St. Jude Children's Research Hospital (1)
United States (8) · China (6) · Australia (1) · Nepal (1) · Peru (1) · South Africa (1) · South Korea (1) · Sri Lanka (1) · Taiwan (1) · Thailand (1) · United Kingdom (1) · Vietnam (1)
Lei Zhang, MD (1) · Nidhi Bhatt, MD (1) · Patricia Steinert, PhD, MBA (1) · Sung-Yun Pai, MD (1) · Tiffany Chang, MD (1) · Xiaodong Zhao, PHD (1)
PMID 28526489, PMID 31830234, PMID 33743238, PMID 34383289, PMID 39207600, PMID 41491634, PMID 41813646
2017 (1)