Table 3.
Clinical advances in transposon-based gene therapy.
| Sr. No. | Study | Disease | Intervention/ Treatment |
Phase | Clinical Trial Gov. Identifier |
|---|---|---|---|---|---|
| 1. | MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT | Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome), Mucopolysaccharidosis Type IH MPS IH, Hurler Syndrome |
Autologous Plasmablasts |
1/2 | NCT04284254 |
| 2. | Analysis of Transposon Control Pathways in Germinal Cancers of the Testicle | Germinal Cancers of the Testicle | Genetic: Extraction of total RNA from healthy and tumor tissues | NCT02873793 | |
| 3. | Transposon-manipulated Allogeneic CARCIK-CD19 Cells in Paediatric and Adult Patients With r/r ALL Post HSCT (CARCIK) | Acute Lymphoblastic Leukemia in Relapse |
Biological: CARCIK-CD19 | 1/2 | NCT03389035 |
| 4. | Anti-CD19 CAR in PiggyBac Transposon-Engineered T Cells for Relapsed/Refractory B-cell Lymphoma or B-cell Acute Lymphoblastic Leukaemia | B Cell Lymphoma, B-cell Acute Lymphoblastic Leukemia |
Biological: Anti-CD19 CAR-T Cells Injection | 1 | NCT04289220 |
| 5. | Mechanisms and Factors Responsible for the Inhibition of Transposons During Fatal Gonad Development in Humans | Medical Termination of Pregnancy, Voluntary Termination of Pregnancy |
Other: surgical biopsies | NCT02171845 | |
| 6. | Measurable Residual Disease Driven Strategy for One or Two Infusions of Non- Viral, Transposon-manipulated CARCIK (CD19) Cells: A Phase II Study in Paediatric and Adult Patients with Relapsed/Refractory B Cell Precursor ALL (BCP-ALL) | Acute Lymphoblastic Leukemia | Genetic: PTG-CARCIK-CD19 | 2 | NCT05252403 |
| 7. | A Phase II Study Using the Administration of Autologous T-Cells Engineered Using the Sleeping Beauty Transposon/Transposase System to Express T-Cell Receptors Reactive Against Mutated Neoantigens in Patients with Metastatic Cancer | Endocrine/ Neuroendocrine, Non-Small Cell Lung Cancer, Breast Cancer, Gastrointestinal/ Genitourinary Cancers, Ovarian Cancer |
Drug: Fludarabine Drug: Cyclophosphamide Drug: Aldesleukin Biological: Sleeping Beauty Transposed PBL |
2 | NCT04102436 |
| 8. | Phase I/II Study of Autologous T Cells Engineered Using the Sleeping Beauty System to Express T-Cell Receptors (TCRs) Reactive Against Cancer-specific Mutations in Subjects with Solid Tumors | Gynecologic Cancer, Colorectal Cancer, Pancreatic Cancer, Non-small Cell Lung Cancer, Cholangiocarcinoma, Ovarian Cancer |
Biological: Neoantigen specific TCR-T cell drug product Biological: Aldesleukin (IL-2) |
1/2 | NCT05194735 |