Table 1.
Current LV-based clinical trials available from “clinicaltrials.gov” from September 2019 to September 2020 using the terms “Lentiviral vectors” as keywords.
| Disease | Intervention | Reference | Phase |
|---|---|---|---|
| X-linked severe combined immunodeficiency (SCID-X1) | Transfer IL2RG complementary DNA (ex vivo) | NCT04286815 | I |
| Mucopolysaccharidosis type IIIA | CD34+ cells expressing the human SGSH gene (ex vivo) | NCT04201405 | I, II |
| Metastatic breast cancer | CAR T cells specific for a cleaved form of MUC1 (ex vivo) | NCT04020575 | I |
| Fanconi anemia subtype A | CD34+ cells expressing the FANCA gene (ex vivo) | NCT04069533 | II |
| Sickle cell disease/anemia | CD34+ cells expressing the βAS3 globin gene (ex vivo) | NCT03964792 | I, II |
| CD34+ cells expressing human γ-globinG16D and short-hairpin RNA734 (ex vivo) | NCT04091737 | I | |
| CD34+ cells expressing LentiGlobin BB305 Drug Product (ex vivo) | NCT04293185 | III | |
| Non-Hodgkin’s lymphoma | Autologous CD19-directed chimeric antigen receptor (CAR) T cells (ex vivo) | NCT03938987 | I, II |
| Infusion of UCD19 CAR T cells (ex vivo) | NCT04240808 | I | |
| CD22 CAR T cells (ex vivo) | NCT04088890 | I | |
| Hemophilia A | CD34+ cells expressing the B domain deleted from of human coagulation factor VIII (ex vivo) | NCT03818763 | I |
| CD34+ cells expressing human factor VIII gene (ex vivo) | NCT04418414 | I | |
| CD34+ cells expressing a functional FVIII gene to overcome human clotting FVIII gene defect (ex vivo) | NCT03217032 | I | |
| COVID-19 | Use LV to modify artificial antigen-presenting cells to express SARS-CoV-2 proteins and immunomodulatory genes and activate T cells (in vivo) | NCT04299724 | I |
| Use LV to express SARS-CoV-2 proteins and immunomodulatory genes to modify dendritic cells and to activate T cells (in vivo) | NCT04276896 | I, II | |
| Metachromatic leukodystrophy | CD34+ cells expressing the human arylsulfatase A gene (ex vivo) | NCT04283227 | III |
| Pyruvate kinase deficiency | CD34+ cells expressing a correct copy of the deficient PKD gene (ex vivo) | NCT04105166 | I |
| Hemophilia B | LV to deliver a functional FIX gene to overcome human clotting FIX gene defect (ex vivo) | NCT03961243 | I |
| CD19 negative B-cell malignancies | Gene-modified T cells targeting CD19-negative B malignancies (ex vivo) | NCT04430530 | I, II |
| Hematological malignancy | Genetically engineered NK cells (ex vivo) | NCT04093622 | - |
| Hepatitis C | Immunotherapy (HCVax™) (in vivo) | NCT04318379 | I |
| Multiple myeloma | C-CAR088 Drug (ex vivo) | NCT04295018 | I |
| CD4+ chimeric antigen receptor immunotherapy (ex vivo) | NCT04162340 | II, III | |
| CD4+ chimeric antigen receptor immunotherapy (ex vivo) | NCT04162353 | I | |
| B-Cell lymphoma | CD22 CAR T cells (ex vivo) | NCT04088864 | I |