Table 1.
Gene therapy products launched around the world.
| Product (Company) | Vector | Delivery Gene | Target Disease | Approved Country (Year) | Reference |
|---|---|---|---|---|---|
| Gendicine (Shenzhen SiBiono GeneTech, Shenzhen, China) | Adenovirus (in vivo, IT) |
Human wild-type p53 | Head and neck squamous cell carcinoma | China (2003) | |
| Oncorine (Shanghai Sunway Biotech, Shanghai, China) | Oncolytic adenovirus (in vivo, IT) |
Head and neck cancer | China (2006) | [26] | |
| Rexin G (Epeius Biotechnology, Monrovia, CA, USA) | Retrovirus (in vivo, IV) |
Mutant cyclin G1 | Chemoresistant solid tumor | Philippines (2007) | |
| Neovagulgen (Human Stem Cell Institute, Moscow, Russia) | Plasmid (in vivo, IM) |
Vascular endothelial growth factor (VEGF) | Peripheral artery disease | Russia (2011) Ukraine (2013) |
|
| Glybera (Uniqure, Amsterdam, Netherlands) Discontinued |
AAV1 (in vivo IM) |
Lipoprotein lipase | Lipoprotein lipase deficiency | Europe (2012) | [27] |
| Imlygic (Amgen, Thousand Oaks, CA, USA) | Oncolytic HSV1 (in vivo, IT) |
Granulocyte macrophage colony-stimulating factor (GM-CSF) | Malignant melanoma | America (2015) Europe (2015) |
[28] |
| Strimveils (GSK, Brentford, United Kingdam) | Retrovirus (ex vivo) |
Adenosine deaminase (ADA) | ADA deficiency | Europe (2016) | [29] |
| Zalmoxis (MolMed S.p.A, Milano, Italy) | Retrovirus (ex vivo) |
Herpesvirus thymidine kinase | Graft versus host disease | Europe (2016) | |
| Kymriah (Novartis, Basel, Switzerland) | Retrovirus (ex vivo) |
Chimeric antigen receptor (CAR) against CD19 | Acute lymphoblastic leukemia | America (2017) Europe (2018) Japan (2019) |
[30] |
| Yescarta (Kite Pharma, Santa Monica, CA, USA) | Retrovirus (ex vivo) |
CAR against CD19 | Large B cell lymphoma | America (2017) Europe (2018) |
[31] |
| Luxturna (Spark Therapeutics, Philadelphia, PA, USA) | AAV2 (in vivo, SR) |
RPE65 | Retinal dystrophy | America (2017) Europe (2018) |
|
| Zynteglo (Bluebird Bio, Cambridge, MA, USA) | Lentivirus (ex vivo) |
Beta-globin | Beta thalassemia | Europe (2019) | [32] |
| Zolgensma (Novartis, Basel, Switzerland) | AAV9 (in vivo, IV) |
SMN1 | Spinal mascular atrophy (SMA) | America (2019) Europe (2019) |
[25] |
| Collategene (Mitsubishi Tanabe Pharma Corporation, Osaka, Japan) | Plasmid (in vivo, IM) |
HGF | Critical limb ischemia | Japan (2019) |
IT: intra-tumor, IV: intravenous, IM: intra-muscular, SR: sub-retinal.