Table 1 These 12 genomic therapies were selected from the FDA’s list of 27 approved cellular and gene therapy (CGT) products, as of December, 16, 2022.
Therapy | Date Approved | Drug Description | Disease | Price |
|---|---|---|---|---|
KYMRIAH - Novartis Pharmaceuticals Corporation | 8/30/2017 | CD19-directed autologous CAR T-cells (ex vivo) | follicular lymphoma | $475,000 [28] |
YESCARTA - Kite Pharma, Inc. (Gilead) | 10/18/2017 | CD19-directed autologous CAR T-cells (ex vivo) | large B-cell lymphoma, a type of non-Hodgkin lymphoma | $373,000 [29] |
LUXTURNA - Spark Therapeutics, Inc. | 12/18/2017 | AAV carrying a functional RPE65 gene (in vivo) | biallelic RPE65 mutation-associated retinal dystrophy. | $425,000 [30] |
ZOLGENSMA - Novartis Gene Therapies, Inc. | 5/24/2019 | AAV carrying a functional SMN1 gene (in vivo) | spinal muscular atrophy | $2.125 M [31] |
TECARTUS - Kite Pharma, Inc. (Gilead) | 7/24/2020 | CD19-directed autologous CAR T-cells (ex vivo) | mantle cell lymphoma or acute lymphoblastic leukemia | $373,000 [32] |
BREYANZI - Juno Therapeutics, Inc., a Bristol-Myers Squibb Company | 2/5/2021 | CD19-directed autologous CAR T-cells (ex vivo) | large B-cell lymphoma | $410,300 [33] |
ABECMA - Celgene Corporation, a Bristol-Myers Squibb Company | 3/26/2021 | BCMA-directed autologous CAR T-cells (ex vivo) | multiple myeloma | $419,500 [34] |
CARVYKTI - Janssen Biotech, Inc. | 2/28/2022 | BCMA-directed autologous CAR T-cells (ex vivo) | multiple myeloma | $465,000 [35] |
ZYNTEGLO - bluebird bio, Inc. | 8/17/2022 | Autologous HSP cells modified with a LVV carrying a form of the ß-globin gene (ex vivo) | ß-thalassemia | $2.8 M (USA) [36]$1.8 M* (EU) |
SKYSONA - bluebird bio, Inc. | 9/16/2022 | Autologous HSP cells modified with a LVV carrying a functional ABCD1 gene (ex vivo) | cerebral adrenoleukodystrophy | $3.0 M [37] |
HEMGENIX - CSL Behring LLC | 11/22/2022 | AAV carrying a form of the factor IX gene (in vivo) | Hemophilia B | $3.5 M [38] |
ADSTILADRIN - Ferring Pharmaceuticals A/S | 12/16/2022 | AAV carrying a functional interferon alfa-2b gene (in vivo) | Non-Muscle Invasive Bladder Cancer | not available |
