Table 2. Current list of approved AAV gene therapy products for clinical use.
| Therapeutic name | Year of approval | Approving agency | Indication | Type of therapy | Vector | Dose | Route of administration | Manufacturer |
|---|---|---|---|---|---|---|---|---|
| Glybera* (alipogene tiparvovec) | 2012 | EMA | Lipoprotein lipase deficiency | AAV gene therapy | AAV1-LPL | 1 × 1012 vg/kg body weight | Intramuscular injection | UniQure (Amsterdam, Netherlands) |
| Luxturna (voretigene neparvovec-rzyl) | 2017/2018 | FDA/EMA | Retinal dystrophy (biallelic RPE65 mutation) | AAV gene therapy | AAV2-RPE65 | 1.5 × 1011 vg/eye | Subretinal injection | Spark Therapeutics, Inc (Philadelphia, Pennsylvania, U.S.A.) |
| Zolgensma (onasemnogene aberparvovec xioi) | 2019 | FDA | Spinal muscular atrophy | AAV gene therapy | AAV9- SMN1 | 1.1 × 1014 vg/kg body weight | Intravenous infusion | AveXis Inc (Chicago, Illinois, U.S.A.) |
EMA, European Marketing Authorization; FDA, Food and Drug Administration.
*
prohibitive cost of regulatory body precluding commercial viability.