Table 1.
The list of approved gene therapy products worldwide.
| Number | Company/institute | Product | Clinical trial | Country | Year | Indication/disease |
|---|---|---|---|---|---|---|
| 1 | Shenzhen SiBiono GeneTech | Gendicine (recombinant human p53 oncolytic adenovirus) | Commercial market | China | 2003 | Head and neck cancer |
| 2 | Shanghai Sunway Biotech | Oncorine (Recombinant Human Adenovirus Type 5 Injection) | Commercial market | China | 2005 | Head and neck and esophagus cancer, Nasopharyngeal cancer, etc. |
| 3 | UniQure | Glybera (AAV1) | Withdrawn | EU | 2012 | LPLD |
| 4 | Amgen | Imlygic (talimogene laherparepvec, T-Vec) | Commercial market | USA | 2015 | Melanoma |
| 5 | GlaxoSmithKline | Strimvelis (autologous CD34+ cells transduced with ADA) | Commercial market | EU | 2016 | ADA-SCID |
| 6 | Novartis | Kymriah (tisagenlecleucel) | Commercial market | Switzerland/USA | 2017 | B-ALL |
| 7 | Gilead/Kite Pharma | Yescarta (axicabtagene ciloleucel) | Commercial market | USA | 2017 | DLBCL |
| 8 | Roche/Spark Therapeutics | Luxturna (voretigene neparvovec-rzyl) | Commercial market | USA | 2017 | Biallelic RPE65 mutation-associated retinal dystrophy |
| 9 | Novartis/AveXis | Zolgensma (onasemnogene abeparvovec-xioi) | Commercial market | Switzerland/USA | 2019 | SMA |
| 10 | Bluebird bio | Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) | Delayed commercial | EU | 2019 | TDT |
| 11 | BioMarin Pharmaceutical | Valoctocogene roxaparvovec (valrox) | Waiting for commercial | USA | 2019-2020 | Hemophilia A |
EU European Union, LPLD lipoprotein lipase deficiency, AAV1 adeno-associated virus type-1, ADA-SCID Adenosine deaminase deficiency-severe combined immune deficiency, B-ALL B-cell acute lymphoblastic leukemia, DLBCL diffuse large B-cell lymphoma, SMA spinal muscular atrophy, TDT transfusion-dependent β-thalassemia.