2 completed, 2 recruiting, 1 not yet recruiting, 1 terminated. 6 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 6 | Completed 2, Recruiting 2, Not yet recruiting 1, Terminated 1 |
| Phase 2 | 4 | Completed 1, Not yet recruiting 1, Recruiting 1, Terminated 1 |
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 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 |
| 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 |
| 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 |
Hematologic Diseases (3) · Blood Coagulation Disorders (2) · Blood Coagulation Disorders, Inherited (2) · Coagulation Protein Disorders (2) · Genetic Diseases, Inborn (2) · Hemophilia A (2) · Hemorrhagic Disorders (2) · Hemostatic Disorders (2) · Blood Protein Disorders (1) · Disease (1) · Eye Diseases (1) · Genetic Diseases, X-Linked (1) · Immune System Diseases (1) · Immunoproliferative Disorders (1) · Lymphoproliferative Disorders (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Hemic and Lymphatic Diseases (3) · Pathological Conditions, Signs and Symptoms (3) · Cardiovascular Diseases (2) · Genetic Diseases, Inborn (2) · Pathologic Processes (2) · Central Nervous System Diseases (1) · Congenital Abnormalities (1) · Eye Diseases, Hereditary (1) · Genetic Diseases, X-Linked (1) · Hemorrhagic Disorders (1) · Hereditary Sensory and Motor Neuropathy (1) · Heredodegenerative Disorders, Nervous System (1) · Immune System Diseases (1) · Immunologic Deficiency Syndromes (1)
Valoctocogene Roxaparvovec (1)
BioMarin Pharmaceutical (1) · BlackfinBio Ltd (1) · David Williams (1) · Genzyme, a Sanofi Company (1) · Kelonia Therapeutics, Inc. (1) · Spark Therapeutics, Inc. (1)
United States (5) · Australia (1) · South Africa (1) · South Korea (1) · Taiwan (1) · United Kingdom (1)
Sung-Yun Pai, MD (1) · Tiffany Chang, MD (1)
PMID 28526489, PMID 39207600, PMID 41491634
2017 (1)