Table 2.
Ongoing clinical trials of gene therapy in China.
| Number | Company/Institute | Product | Route of administration | Target | Indication/disease | Clinical trial | Trial ID | Initiate year | Status |
|---|---|---|---|---|---|---|---|---|---|
| 1 | Shenzhen Geno-immune Medical Institute, Shenzhen | YUVA-GT-F801 (lentiviral gene-modified autologous stem cells) | Intravenous infusion | FVIII | Hemophilia A | Phase I | NCT03217032 | 2017 | Not yet recruiting |
| 2 | Institute of Hematology & Blood Diseases Hospital, Tianjin | BBM-H901 (AAV) | Single-dose intravenous injection | FIX | Hemophilia B | Phase I | NCT04135300 | 2019 | Recruiting |
| 3 | Nanfang Hospital, Guangzhou | Autologous CD34+ cells genetically modified (lentiviral vector) | Intravenous infusion/Auto-HSCT | HBB | β-thalassemia major | Phase I/II | NCT03276455 | 2017 | Not yet recruiting |
| 4 | Shenzhen Geno-immune Medical Institute, Shenzhen | Gene-modified autologous stem cells (lentiviral Vector) | Intravenous infusion | HBB | β-thalassemia | Phase I/II | NCT03351829 | 2017 | Not yet recruiting |
| 5 | Shanghai Bioray laboratory Inc,Shanghai | γ-globin reactivated autologous hematopoietic stem cells (CRISPR/Cas9 gene editing system) | Intravenous infusion/Auto-HSCT | NA | β-thalassemia major | Phase I/II | NCT04205435 | 2019 | Not yet recruiting |
| 6 | Sichuan University | PD-1 Knockout T Cells | Intravenous infusion | PD-1 | Metastatic non-small cell lung cancer | Phase I | NCT02793856 | 2016 | Active, not recruiting |
| 7 | Hangzhou Cancer Hospital | PD-1 Knockout T Cells | Intravenous infusion | PD-1 | Esophageal cancer | Phase I | NCT03081715 | 2017 | Completed |
| 8 | Huazhong University of Science and Technology | rAAV2-ND4 | Intravitreal Injection (IVT injection) | ND4 (complex I) | LHON | NA | NCT01267422 | 2010 | Completed |
| 9 | Huazhong University of Science and Technology | rAAV2-ND4 | IVT injection | ND4 (complex I) | LHON | Phase II/III | NCT03153293 | 2017 | Active, not recruiting |
| 10 | Huazhong University of Science and Technology | rAAV2-ND4 | IVT injection | ND4 (complex I) | Acute LHON | NA | NCT03428178 | 2018 | Recruiting |
| 11 | OrienGene Biotechnology Ltd. | OrienX010 (recombinant human GM-CSF HSV-1) | Tumor site local injections | NA | Melanoma | Phase I | NCT03048253 | 2017 | Unknown |
| 12 | Wuhan Binhui Biotechnology Co., Ltd. | OH2 Oncolytic Virus (HSV-2 strain HG52) | Intratumorally injection | NA | Malignant solid tumors (gastrointestinal cancers, head and neck cancers, soft tissue sarcomas) | Phase I | NCT03866525 | 2019 | Recruiting |
| 13 | Wuhan Binhui Biotechnology Co., Ltd. | OH2 Oncolytic Virus (HSV-2 strain HG52) | Intratumorally injection | NA | Malignant solid tumors (melanoma) | Phase I/II | NCT04386967 | 2020 | Recruiting |
| 14 | Shenzhen Geno-Immune Medical Institute | TYF-IL2RG gene-modified autologous stem cells | Intravenous infusion | IL2RG | SCID-X1 | Phase I/II | NCT03217617 | 2017 | Recruiting |
| 15 | Children’s Hospital of Chongqing Medical University | Lentiviral vector transduced bone marrow stem cells | Intravenous infusion | IL2RG | SCID-X1 | NA | NCT04286815 | 2020 | Recruiting |
| 16 | Shenzhen Geno-Immune Medical Institute | Gene-modified autologous stem cells (lentiviral vector) | Intravenous infusion | FANCA | Fanconi anemia | Phase I/II | NCT03351868 | 2017 | Recruiting |
| 17 | Shenzhen Geno-Immune Medical Institute | TYF-ADA gene-modified autologous stem cells (lentiviral vector) | Intravenous infusion | ADA | ADA-SCID | NA | NCT03645460 | 2018 | Recruiting |
| 18 | Shenzhen Geno-Immune Medical Institute | Lentiviral TYF-CGD-modified autologous stem cells | Intravenous infusion | CYBB/NCF1 | CGD | Phase I/II | NCT03645486 | 2018 | Recruiting |
| 19 | Shenzhen Geno-Immune Medical Institute | Lentiviral vector TYF-ARSA | Intracerebral injection | ARSA | MLD | Phase I/II | NCT03725670 | 2018 | Recruiting |
| 20 | Shenzhen Geno-Immune Medical Institute | Lentiviral vector carrying ABCD1 gene | Intracerebral injection | ABCD1 | X-ALD | Phase I/II | NCT03727555 | 2018 | Recruiting |
FVIII factor VIII, FIX factor IX, HSCT hematopoietic stem cell transplantation, HBB hemoglobin subunit beta, NA not available, PD-1 programmed death-1, ND4 NADH dehydrogenase, subunit 4, LHON Leber’s hereditary optic neuropathy, HSV Herpes simplex virus, SCID-X1 X-linked severe combined immunodeficiency, FANCA Fanconi anemia complementation group A, ADA adenosine deaminase, ADA-SCID adenosine deaminase severe combined immune deficiency, CYBB X-linked gp91phox gene, NCF1 gene coding for p47phox, CGD chronic granulomatous disease, ARSA Arylsulfatase A, MLD metachromatic leukodystrophy, ABCD1 ATP-binding cassette transporter, X-ALD X-linked adrenoleukodystrophy.