Table 1.
Ex vivo gene therapy products that have acquired marketing authorization.
| Product | Developer | Approval | Indication | Mechanism of action | Clinical trials | Price |
|---|---|---|---|---|---|---|
| STRIMVELIS® | GlaxoSmithKline | May 2016 (EMA) | ADA-SCID | Autologous HSCs expressing ADA; γ-RV-ADA |
NCT00598481; NCT0059978 | 594,000 $ |
| KYMRIAH® Tisagenlecleucel | Novartis | Aug 2017 (FDA) |
B-cell acute lymphoblastic leukemia (ALL), r/r diffuse large B-cell lymphoma (DLBCL) | Autologous CAR T cell targeting CD19; SIN-LV-CAR |
NCT02435849; NCT02445248; NCT02228096; NCT02445222; NCT01626495; NCT01029366; NCT01747486; NCT02030847; NCT02030834; NCT02135406 | 475,000 $ |
| YESCARTA® Axicabtagene Ciloleucel | Kite Pharma (Gilead) |
Oct 2017 (FDA) |
Non-Hodgkin lymphoma, B-cell lymphoma, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma (FL) | Autologous CAR T cell targeting CD19; γ-RV-CAR |
NCT02348216 | 373,000 $ |
| ZYNTEGLOTM Betibeglogene Autotemcel | Bluebird bio | May 2019 (EMA) |
Transfusion dependent β-thalassemia (TDT) | Autologous HSCs expressing HBB; SIN-LV-HBB |
NCT01745120; NCT02151526 | ∼1.8 million $ |
| LIBMELDYTM Atidarsagene Autotemcel | Orchard Therapeutics | Dec 2020 (EMA) |
Early-onset metachromatic leukodystrophy (MLD) | Autologous HSCs expressing ARSA; SIN-LV-ARSA |
Eudract 2009-017349-77 (Study 201222) |
∼3.9 million $ |
| BREYANZI® Lisocabtagene Maraleucel |
Juno Therapeutics (Bristol-Myers Squibb) |
Feb 2021 (FDA) |
r/r large B cell lymphoma | Autologous CAR T cell targeting CD19; SIN-LV-CAR* |
NCT02631044; NCT03484702; NCT03744676; NCT03310619; NCT03483103; NCT03331198; NCT03743246; NCT03435796 | ∼ 432,055 $ |
| ABECMA® Idecabtagene Vicleucel | Celgene (Bristol-Myers Squibb) |
Mar 2021 (FDA) |
r/r multiple myeloma (MM) | Autologous CAR T cell targeting BCMA; SIN-LV-CAR |
NCT03361748; NCT03601078; NCT03651128; NCT03435796; NCT02658929; NCT02786511 | 419,500 $ |
| TECARTUS® Brexucabtagene Autoleucel | Kite Pharma (Gilead) |
Oct 2021 (FDA) |
r/r mantle cell lymphoma (MCL), r/r B-cell precursor ALL | Autologous CAR T cell targeting CD19; γ-RV-CAR# |
NCT02601313, NCT02614066, NCT02625480, NCT03624036, NCT04162756 | 373,000 $ |
| CARVYKTITM Ciltacabtagene Autoleucel | Janssen Biotech | Feb 2022 (FDA) |
r/r MM | Autologous CAR T cell targeting BCMA; SIN-LV-CAR |
NCT03548207; NCT04133636; NCT04181827 | 465,000 $ |
*The BREYANZI SIN-LV-CAR includes a nonfunctional truncated growth factor receptor (GFRt) that is co-expressed with CD19-specific CAR.
#The γ-RV-CAR applied in TECARTUS is identical to that in YESCARTA, with TECARTUS differing from YESCARTA in that T cell enrichment is performed in TECARTUS manufacture but not in YESCARTA.
γ-RV: Gamma-retroviral vector; SIN-LV: Self-inactivate lentiviral vector; CAR: Chimeric antigen receptor; ADA: Adenosine deaminase; SCID: Severe combined immunodeficiency; r/r: Relapsed or refractory; HBB: Hemoglobin subunit beta; ARSA: Arylsulfatase A; BCMA: B cell maturation antigen.