Investigational lentiviral vector–based autologous cell therapy products for sickle cell disease
| Product . | Sponsor . | Technology . | Effect . | Activation . | Status . |
|---|---|---|---|---|---|
| Lovotibeglogene autotemcel | Bluebird bio | Lentiviral gene addition | BB305 LVV/bA-T87Q (antisickling) | 2014 | Submitted BLA spring 2023 |
| ARU-1801 | Aruvant Sciences GmbH | Lentiviral gene addition | LVV/γ-globin (G16D) (antisickling) | 2014 | Active, not recruiting |
| DREPAGLOBE | Assistance Publique–Hopitaux de Paris | Lentiviral gene addition | GLOBE LVV/bA (nonsickling) | 2019 | Active, not recruiting |
| BCH-BB694 | Boston Children's Hospital | Lentiviral gene addition | shRNA targeting BCL11A (antisickling) | 2018 | Phase 3 |
| Product . | Sponsor . | Technology . | Effect . | Activation . | Status . |
|---|---|---|---|---|---|
| Lovotibeglogene autotemcel | Bluebird bio | Lentiviral gene addition | BB305 LVV/bA-T87Q (antisickling) | 2014 | Submitted BLA spring 2023 |
| ARU-1801 | Aruvant Sciences GmbH | Lentiviral gene addition | LVV/γ-globin (G16D) (antisickling) | 2014 | Active, not recruiting |
| DREPAGLOBE | Assistance Publique–Hopitaux de Paris | Lentiviral gene addition | GLOBE LVV/bA (nonsickling) | 2019 | Active, not recruiting |
| BCH-BB694 | Boston Children's Hospital | Lentiviral gene addition | shRNA targeting BCL11A (antisickling) | 2018 | Phase 3 |
BLA, biologic license application; LVV, lentiviral vector; shRNA, short hairpin RNA.