Table 1.
Breakdown of US gene therapy clinical trials.
| Disease | Vector | Gene Target | Trial Number |
|---|---|---|---|
|
| |||
| Phase I | |||
|
| |||
| Leber's Hereditary Optic Neuropathy (LHON) | Adeno-Associated Virus | G11778G ND4 | NCT02161 380 |
| Late-Onset Pompe Disease | Adeno-Associated Virus | α-Glucosidase | NCT02240 407 |
| Late Infantile Neuronal Ceroid Lipofuscinosis | Adeno-Associated Virus | CLN2 | NCT01161 576 |
| Parkinson's Disease | Adeno-Associated Virus | Aromatic L-Amino Acid Decarboxylase |
NCT01973 543 |
| α1-Antitrypsin Deficiency | Adeno-Associated Virus | α1-Antitrypsin | NCT02168 686 |
| Leber Congenital Amaurosis | Adeno-Associated Virus | RPE65 | NCT00821 340 |
| MERTK-Associated Retinal Disease | Recombinant Adeno-Associated Virus |
MERTK | NCT01482 195 |
| Sickle Cell Disease | Lentivirus (ex novo) | Anti-Sickling (βAS3) | NCT02247 843 |
| Fanconi Anemia | Lentivirus | Fanconi Anemia Complementation Group A |
NCT01331 018 |
| Recurrent Malignant Glioma | Retroviral Replicating Vector | Cytosine Deaminase | NCT01156 584 |
| Solid Tumors | Liposome | RB94 | NCT01517 464 |
|
| |||
| Phase II | |||
|
| |||
| Heart Failure | Adeno-Associated Virus | Sarcoplasmic Reticulum Calcium ATPase |
NCT01966 887 |
| Blindness Caused by Choroideremia | Adeno-Associated Virus | Rab-Escort Protein 1 | NCT01461 213 |
| Hemophilia B | Adeno-Associated Virus | Human Factor IX | NCT02484 092 |
| Malignant Pleural Effusion | Recombinant Adenovirus | p53 | NCT02429 726 |
| Malignant Melanoma and Other Solid Tumors | Adenoviral serotype 5 | CD40L | NCT01455 259 |
| β-Thalassemia Major | Lentivirus (ex novo) | β-A(T87Q)-Globin | NCT01745 120 |
| HIV-1 | Lentivirus | shRNA For CCR5, HIV-1 Fusion Inhibitor, C46 |
NCT01734 850 |
| Transfusion Dependent β-Thalassemia | GLOBE Lentivirus | Human β-Globin | NCT02453 477 |
| Wiskott-Aldrich Syndrome | Retrovirus | Wiskott-Aldrich Syndrome Protein (WASP) |
NCT01410 825 |
| Severe Combined Immunodeficiency, X-linked (SCID-X1) |
Self-Inactivating (SIN) Gammaretrovirus |
IL-2RG | NCT01129 544 |
| X-linked Chronic Granulomatous Disease (X-CGD) | SIN Gammaretrovirus | gp91phox Subunit of NADPH Oxidase Complex |
NCT01906 541 |
| Leukaemia | GMP Grade Retrovirus | Wilms Tumor 1 | NCT01621 724 |
| Non-Hodgkin Lymphoma | Autologous T Cells | CD19 CAR | NCT02134 262 |
|
| |||
| Phase III | |||
|
| |||
| Myocardial Ischemia | Adenovirus Serotype-5 | Fibroblast Growth Factor-4 | NCT01550 614 |
| Duchenne Muscular Dystrophy | Lentivirus | AON for Exon 51 of Dystrophin |
NCT02255 552 |
| Painful Diabetic Neuropathy | Non-Viral | Hepatocyte Growth Factor | NCT02427 464 |