Table 3.
FDA/EMA approved gene therapy product for rare monogenic diseases
| Disease | Product | Vector | Gene | Approach | Year of approval |
|---|---|---|---|---|---|
| Lipoprotein lipase deficiency | Glybera (alipogene tiparvovec) | AAV1 | Lipoprotein lipase | In vivo | 2012 |
| SCID due to adenosine deaminase deficiency | Strimvelis (GSK2696273) | Retrorvirus | Adenosine deaminase | Ex vivo (CD34+ cells) | 2016 |
| Retinal dystrophy due to RPE65 mutation | Luxturna (voretigene neparvovec-rzyl) | AAV2 | RPE65 | In vivo | 2017 |
| Spinal muscular atrophy | Zolgensma (onasemnogene abeparvovec-xioi) | AAV9 | SMN1 | In vivo | 2019 |
| β-Thalassemia | Zynteglo (betibeglogene autotemcel) | Lentivirus | Modified β-globin gene | Ex vivo (CD34+ cells) | 2019 |
| Metachromatic leukodystrophy | Libmeldy (atidarsagene autotemcel) | Lentivirus | ARSA | Ex vivo (CD34+ cells) | 2020 |
| Adrenoleukodystrophy | Skysona (elivaldogene autotemcel/lenti-D) | Lentivirus | ABCD1 | Ex vivo (CD34+ cells) | 2021 |
| Hemophilia B | Hemgenix (etranacogene dezaparvovec) | AAV5 | Padua variant of factor IX (FIX-Padua) | In vivo | 2022 |
FDA/EMA, U.S. Food and Drug Administration/European Medicines Agency; SCID, severe combined immunodeficiency; AAV, adeno-associated virus; ARSA, arylsulfatase A.