Table 4 Details of the approved therapies based on viral vector technology
From: Current strategies employed in the manipulation of gene expression for clinical purposes
Trade name | Agency | First approval | First approved indication | Cargo | Delivery method | Admin | Pivotal clinical trial | Actual completion date if applicable |
|---|---|---|---|---|---|---|---|---|
Glybera® | EMA | 2012/10 (withdrawn 2017/10) | LPLD | LPL | AAV | in vivo | NCT00891306 | 2009/02—2011/04 |
Strimvelis® | EMA | 2016/04 | ADA-SCID | ADA | RVV | ex vivo | NCT00598481 | 2008/01—2019/06 |
Kymriah® | FDA | 2017/08 | r/r B-cell ALL | CD19 CAR | LVV | ex vivo | NCT02435849 | 2015/04—2020/01 |
Yescarta® | FDA | 2017/10 | r/r LBCL | CD19 CAR | RVV | ex vivo | NCT02348216 | 2015/04—2020/09 |
Luxturna® | FDA | 2017/12 | biallelic RPE65 mutation-associated IRD | RPE65 | AAV | in vivo | NCT00999609 | 2012/10—2015/07 |
Zolgensma ® | FDA | 2019/05 | SMA (type I) | SMN | AAV | in vivo | NCT03306277 | 2017/10—2019/11 |
Zynteglo™ | EMA | 2019/05 (withdrawn 2022/03) | TDT | HBB | LVV | ex vivo | NCT01745120 | 2013/08—2018/02 |
Tecartus™ | FDA | 2020/07 | r/r MCL | CD19 CAR | RVV | ex vivo | NCT02601313 | 2015/11—2019/07 |
Breyanzi® | FDA | 2021/02 | r/r LBCL | CD19 CAR | LVV | ex vivo | NCT02631044 | 2016/01—2022/12 |
Abecma® | FDA | 2021/03 | r/r MM | BCMA CAR | LVV | ex vivo | NCT03361748 | 2017/12—2024/11 |
Carvykti ™ | FDA | 2022/02 | r/r MM | BCMA CAR | LVV | ex vivo | NCT03548207 | 2018/06—2022/08 |