Table 2.
Status | Name/Trial | Trade Name | Disease | Reference |
---|---|---|---|---|
2017 FDA approved | Voretigene neparvovec | Luxterna | Retinal dystrophy | [43,44] |
2019 FDA approved | Onasemnogene abeparvovec | Zolgensma | SMA (Pediatric Patients, < 2 y/o) | [45,46] |
Currently evaluated in clinical trials | Lovotibeglogene autotemcel | LentiGlobin BB305 | SCD, Thalassemia, TDT | [47] |
Currently evaluated in clinical trials | CLIMB THAL-111, CLIMB SCD-111 | - | SCD, TDT | [48] |