List of Gene Therapy Clinical Study Protocols Approved in Japan
Up date at May 2015**Ref.: move to Japanese page. Sorry!
| Ref. |
Year of Approval | Hospital, Institute, Company |
Target Desease | Gene | Vactor/ Methods | Case number | |
| 51 | 2014 ++ |
kyushu Univ. Hospital | Intermittent claudication | Fibroblast growth factor-2（FGF-2) | Sendai viral vector／in vivo | (60) [Continue] |
|
| 50 | 2014 | Jichi Medical Univ. | Parkinson's disease | Aromatic L-amino acid Decarboxylase (AADC) | Adeno associated vector／ in vivo (intra-putamen） |
(6) [Continue] |
|
| 49 | 2014 | Jichi Medical Univ. | Aromatic L-amino acid Decarboxylase deficiency | Aromatic L-amino acid Decarboxylase (AADC) | Adeno associated vector type-2／ in vivo (intra-striatum） |
(4) [Continue] |
|
| 48 | 2014 ++ |
Tokyo Univ. Research Hospital | Glioblastoma | β-galactosidase (Lac-Z) （as a maker) |
*Oncolytic herpes simplex virusesG47Δin vivo (intra-tumoral） | [Continue] | |
| 47 | 2014 | Osaka Univ. Hospital | Chronic arterial occlusion (arteriosclerosis obliterans (ASO)；Buerger's disease） | Hepatocyte Growth Factor（HGF） | Plasmid vector／ in vivo （intra-muscular） |
[Continue] | |
| 46 | 2014 | Okayama Univ. Hospital | Malignant pleiral mesothelioma | REIC（Reduced Expression in Immortalized Cells）/Dickkopf-3（Dkk-3） | Adenoviral Type 5／ in vivo (intra-thoracic、intra-tumoral） |
[Continue] | |
| 45 | 2014 | Jichi Medical Univ. | Refractory B cell Non-Hodgkin Lymphoma | CD19-specific chimeric antigen receptor(CAR) | Retroviral vector/ ex vivo (Peripheral blood mononuclear cell) |
[Continue] | |
| 44 | 2013 | Tokyo Univ. Research Hospital | olfactory esthesioneuroblastoma | β-galactosidase (Lac-Z) （as a maker) |
*Oncolytic herpes simplex virusesG47Δin vivo(intra-tumoral） | (10) [Continue] |
|
| 43 | 2013 | Tokyo Univ. Research Hospital | Glioblastoma | β-galactosidase (Lac-Z) （as a maker) |
*Oncolytic herpes simplex virusesG47Δin vivo (intra-tumoral） | (21, Maximum 30) [Continue] |
|
| 42 | 2013 | Mie Univ. Hospital | Esophageal cance | MAGE-A4 antigen specific T cell receptor (TCR) α-chain and β-chain | Retroviral vector/ ex vivo (syngeneic T cell） |
(9) [Continue] |
|
| 41 | 2012 ++ |
Anges-MG | primary lymphedema | Hepatocyte Growth Factor（HGF） | Plasmid vector／ in vivo (intra-muscluar） |
[Terminated of proceeding] | |
| 40 | 2012 ++ |
Astellas pharma Inc. | Cytomegalovirus (protective vaccine) |
antigen of cytomegalovirus (2 kind) | Plasmid vector in vivo (intra-muscluar) |
[Terminated of proceeding] | |
| 39 | 2013 | Mie Univ. Ehime Univ. Nagoya Univ. Fujita Health Univ. Hospital (multiple collaboration study, 4 institution) |
Acute myeloid leukemia (AML) myelodysplastic syndrome |
WT1 antigen specific cell receptor(TCR) α-chain and β-chain. siRNA expression sequence against TCR. |
Retroviral vector/ ex vivo (Peripheral blood mononuclear cell) |
(9) [Continue] |
|
| 38 | 2012 | Okayama Univ. Hoospital | cervicofacial・thoracic malignant tumor | - | *Oncolytic Adenoviral Type 5, Telomelysin/ in vivo (intra-tumoral） |
(12, Maximum 24) [Continue] |
|
| 37 | － | Chiba Univ. Hospital | Malignant pleural mesothelioma | NK4 | Adenoviral vector/ in vivo (intra-thoracic） |
[Terminated of proceeding] | |
| 36 | 2012 | National Center of Child Health and Development | X-linked （X-Chronic granulomatous disease） | human cytochrome b heavy chain（CYBB） | Retroviral vector/ ex vivo （Hematopoietic stem cell） |
(5) [Continue] |
|
| 35 | 2012 | Tokyo Univ. Hospital | Prostate cancer | β-galactosidase (Lac-Z) （as a maker) |
*Oncolytic herpes simplex virusesG47Δin vivo (intra-tumoral） |
[Continue] | |
| 34 | － | Kitano Hosp. The tazuke kofukai, Osaka Univ. Hospital (28：collaborating with Mie Univ. Hospital) |
Esophageal cancer | MAGE-A4 antigen specific T cell receptor (TCR) α-chain and β-chain | Retroviral vector/ ex vivo (Syngeneic T cell) |
[Terminated of proceeding] (Dismissal) |
|
| 33 | － | ||||||
| 32 | 2012 | kyushu Unuv. Hospital | Retinitis pigmentosa | Pigment epithelium-derived factor（hPEDF） | simian immunodeficiency viral vector (SIV)／ in vivo (subretinal injection） | (20) [Continue] |
|
| 31 | 2013 | Chiba Univ. Hospital | familial LCATdeficiency | Lecithin cholesterol acyltransderase（LCAT） | Retroviral vector／ ex vivo (pre-adipocyte） | [Continue] | |
| 30 | 2011 | Okayama Univ. Hospital | Prostate cancer | REIC（Reduced Expression in Immortalized Cells）/Dickkopf-3（Dkk-3） | Adenoviral vector／ in vivo (intra-tumoral） |
(24, Maximum 36) [Continue] |
|
| 29 | 2009 | Kyoto Prefectural Univ. of Medicine | Renal cell carcinoma | Interferon-β | Positive charge liposome／ in vivo (intra-tumoral） |
(5) [Continue] |
|
| 28 | 2009 | Mie Univ. Hospital | Esophageal Cancer | MAGE-A4 antigen specific T cell receptor (TCR) α-chain and β-chain | Retroviral vector/ ex vivo (syngeneic T cell) |
(15) 2014. 5 [Terminated] |
|
| 27 | 2009 | National Cancer Center Hospital | Hematopoietic malignancy［Protection from Severe Graft Versus Host Disease (GVHD)］ | Herpes Simplex Virus Thymidine Kinase (HSV-tk)/truncated version of the low affinity nerve growth factor receptor (delta LNGFR) |
Retroviral vector/ ex vivo (Peripheral blood mononuclear cell) |
(10, minimum 5) [Continue] |
|
| 26 | 2009 | Tokyo Univ. Hospital | Glioblastoma | β-galactosidase (Lac-Z) （as a maker) |
*Oncolytic herpes simplex viruses G47Δ/ in vivo (intra-tumoral） |
(21, Maximum 30) [Continue] |
|
| 25 | 2008 | Okayama Univ. Hospital | Prostate cancer | Interleukin-12（IL-12） | Adenoviral vector/ in vivo (intra-tumoral） |
(21, Maximum 36) [Continue] |
|
| 24 | 2007 ++ |
Sanofi aventis | Peripheral arterial occlusive disease | Fibroblast growth factor-1（FGF-1） | Plasmid vector/ in vivo (intra-muscular） |
[Continue] | |
| 23 | 2007 ++ |
Takarabio Inc. | Leukemia ［Protection from Severe Graft Versus Host Disease (GVHD)］ | Herpes Simplex Virus Thymidine Kinase (HSV-tk)/truncated version of the low affinity nerve growth factor receptor (delta LNGFR) |
Retro viral vector/ ex vivo (Peripheral blood mononuclear cell) |
(9) [Continue] |
|
| 22 | 2007 | Kitasato Univ. Hospital | Prostate cancer | Herpes Simplex Virus Thymidine Kinase （HSV-tk） |
Adenoviral vector/ in vivo (intra-tumoral） |
5 [Terminated] |
|
| 21 | 2006 | Jichi Medical Hospital | Parkinson's disease | Aromatic L-amino acid decarbocylase (hAADC) | Adeno associated viral vector/ in vivo（intra-striatum) |
6(6) [Terminated]] |
|
| 20 | 2006 | Kyusyu Univ. Hospital | Arteriosclerotic Obliterans (ASO)／Buerger's disease | Fibroblast growth factor-2（FGF-2） | Sendai viral vector／ in vivo (intra-muscular） |
12 [Terminated] |
|
| 19 | 2003 ++ |
Anges-MG | Arteriosclerotic Obliterans (ASO)／Buerger's disease | Hepatocyte Growth Factor（HGF） | Plasmid vector／ in vivo (intra-muscular） |
[Terminated] |
|
| 18 | 2003 | Shinshu Univ. Hospital | malignant melanoma | Interferon-β | Positive charge liposome／ in vivo (intra-tumoral） |
5（5） [Terminated] |
|
| 17 | 2003 | Kobe Univ. Hospital | Prostate Cancer | Herpes Simplex Virus Thymidine Kinase （HSV-tk） |
Adenoviral vector／ in vivo (intra-tumoral） |
6（6） [Terminated] |
|
| 16 | 2002 | Tohoku Univ. Hospital | X-linked severe combined immunodeficiency （X-SCID） |
Common Cytokine Receptor chain γ | Retroviral vector／ ex vivo （Hematopoietic stem cel） |
0 (5) [Terminated] |
|
| 15 | 2002 | Hokkaido Univ. | Adenosine deaminase（ADA）Deficiency ［Immunodeficiency］ |
Adenosine deaminase (ADA)） | Retroviral vector／ ex vivo (Hematopoietic stem cell） |
2（4） [Continue] |
|
| 14 | 2002 | Tokyo Univ. Research Hospital | Neuroblastoma | Interleuin-2 (IL-2)／Lymphotactin | Adenoviral vector／ ex vivo (neuroblastoma cell） |
0 (6) [Terminated] |
|
| 13 | 2002 | Tsukuba Univ. Hospital | Leukemia ［Protection from Severe Graft Versus Host Disease (GVHD)］ | Herpes Simplex Virus Thymidine Kinase （HSV-tk）／truncated version of the low affinity nerve growth factor receptor (delta LNGFR) |
Retroviral vector／ ex vivo (Peripheral blood mononuclear cell) |
（10） [Continue] |
|
| 12 | 2001 | Osaka Univ. Hospital | Arteriosclerotic Obliterans (ASO)／ Burtger's disease |
Hepatocyte Growth Factor（HGF） | Plasmmid Vector／ in vivo (intra-muscular) |
22（22） [Terminated] |
|
| 11 | 2000 | Okayama Univ. Hospital | Prostate Cancer | Herpes Simplex Virus Thymidine Kinase （HSV-tk） |
Adenoviral vector／ in vivo (intra-tumoral) |
9 （9） [Terminated] |
|
| 10 | 2000 ++ |
Chiba Univ. Hospital／ RPR gencell |
Esophageal cancer | p53 | Adenoviral vector／ in vivo (intra-tumoral) |
10（10） [Terminated] |
|
| 9 | 2002 | Center ofJapanease Foundation for cancer research | Brest cancer［Protection from Bone Marrow Suppression on dose intensive chemotherapy］ | Multiple drug resistant gene; MDR1 | Retroviral vector／ ex vivo （Hematopoietic stem cell） |
3（10） [Continue] |
|
| 8 | 2000 | Nagoya Univ. Hospital | Malignant Glioma | Interferon-β | Positive charge liposome／ in vivo (intra-tumoral) |
2(25) [Terminated] |
|
| 7 | 2000 | Jikei Univ. Hospital | NSCLC | p53 | Adenoviral vector／ in vivo (intra-tumoral) |
1 [Terminated] |
|
| 6 | 2000 | Tohoku Univ. Hospital | NSCLC | p53 | Adenoviral vector／ in vivo (intra-tumoral） |
2 [Terminated] |
|
| 5 | 2000 | Tokyo medical Univ. | NSCLC | p53 | Adenoviral vector／ in vivo (intra-tumoral） |
3 [Terminated] |
|
| 4 | 1998 | Okayama Univ. Hospital／ RPR Gencell |
NSCLC | p53 | Adenoviral vector／ in vivo (intra-tumoral） |
9 [Terminated] |
|
| 3 | 1998 | Tokyo Univ. Hospital | Renal carcinoma cell | Granulocyte-macrophage colony-stimulating factor（GM-CSF） | Retrovial vector／ ex vivo （renal carcinoma cell） |
4 (6) [Terminated] |
|
| 2 | 1997 ++ |
Midori Juji | Human Immunodeficiency Virus（HIV）Infectious disease | HIV env/rev | Retroviral vector／ in vivo (intra-muscular） |
0 (4) [Terminated] |
|
| 1 | 1995 | Hokkaido Univ. Hospital | Adenosine deaminase（ADA）Deficiency ［Immunodeficiency］ |
Adenosine deaminase (ADA) | Retroviral vector／ ex vivo (syngeneic T cell) |
1 [Terminated] |
|
＊clinical Trials; ++