In 2023, seven new cell and gene therapies were approved by the FDA. With so many therapies being developed and in clinical trials, we hope to see more life-saving and life-improving treatments to come in the future. To celebrate the accomplishments of the cell and gene therapy scientific and patient community, let’s reflect on all current FDA approved cell and gene therapies to date. The table below contains all licensed products listed as ‘Approved Cellular and Gene Therapy Products’ on fda.gov (last updated on 06/04/2024). Mirus Bio’s team of experts are adept at helping scientists improve their transfection results. Learn more about the transfection reagents and boosters used in cell and gene therapy by reading about TransIT-VirusGEN® Transfection Reagent and RevIT™ AAV Enhancer.
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| Name | Type | Manufacturer | Description |
|---|---|---|---|
| ABECMA (2021) |
Cell (autologous, modified) | Celgene Corporation, a Bristol-Myers Squibb Company | CAR T cells recognize the B-cell maturation antigen (BCMA) and kill BCMA-expressing tumor cells. A lentiviral vector, developed by bluebird bio, is used to engineer the CAR T cells. |
| ADSTILADRIN (2022) |
Adenovirus | Ferring Pharmaceuticals A/S | A recombinant adenovirus (rAd-IFNa/Syn3) delivers human interferon alfa-2b cDNA into the bladder epithelium. Treats patients with certain types of bladder cancer. |
| ALLOCORD (2013) |
Cell (allogeneic) | SSM Cardinal Glennon Children's Medical Center | Blood cell transplant derived from umbilical cord and placenta. |
| AMTAGVI (2024) |
Cell (modified) | Iovance Biotherapeutics, Inc. | Engineered T cells for the treatment of adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor. |
| BEQVEZ (2024) |
AAVRh74var | Pfizer, Inc. | AAV-mediated gene therapy for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid. |
| BREYANZI (2021) |
Cell (autologous, modified) | Juno Therapeutics, Inc., a Bristol-Myers Squibb Company | CAR T cells engineered with lentivirus to attack CD19-expressing tumor cells. Treats patients with certain kinds of large B-cell lymphoma. |
| CARVYKTI (2022) |
Cell (autologous, modified) | Janssen Biotech, Inc. | CAR T cells engineered with lentivirus to attack BCMA-expressing tumor cells. Treats patients with certain kinds of relapsed or refractory multiple myeloma. |
| CASGEVY (2023) |
Cell (autologous, modified) | Vertex Pharmaceuticals, Inc. | CD34+ hematopoietic stem cells engineered with electroporation of CRISPR/Cas9 RNP complexes to decrease expression of BCL11A to increase fetal hemoglobin production. Treats patients with certain kinds of sickle cell disease. |
| CLEVECORD (2016) |
Cell (allogeneic) | Cleveland Cord Blood Center | Transplant of hematopoietic progenitor cells (HPCs) from human cord blood. |
| DUCORD (2012) |
Cell (allogeneic) | Duke University School of Medicine | Transplant of HPCs from human cord blood. |
| ELEVIDYS (2023) |
AAVrh74 | Sarepta Therapeutics, Inc. | Recombinant AAVrh74 delivers a micro-dystrophin gene to treat patients with certain types of Duchenne muscular dystrophy. |
| GINTUIT (2012) |
Cell (allogeneic) | Organogenesis Inc. | Cellularized scaffold for topical application to a surgically created wound bed to treat certain mucogingival conditions. |
| HEMACORD (2011) |
Cell (allogeneic) | New York Blood Center | Transplant of HPCs from human cord blood. |
| HEMGENIX (2022) |
AAV5 | CSL Behring LLC, UniQure | Recombinant AAV5 delivers F9 to treat patients with certain kinds of Hemophilia B. |
| HPC, Cord Blood (2012) |
Cell (allogeneic) | Clinimmune Labs, University of Colorado Cord Blood Bank | Transplant of HPCs from human cord blood. |
| HPC, Cord Blood - MD Anderson Cord Blood Bank (2018) |
Cell (allogeneic) | MD Anderson Cord Blood Bank | Transplant of HPCs from human cord blood. |
| HPC, Cord Blood - LifeSouth (2016) |
Cell (allogeneic) | LifeSouth Community Blood Centers, Inc. | Transplant of HPCs from human cord blood. |
| HPC, Cord Blood - Bloodworks (2016) |
Cell (allogeneic) | Bloodworks | Transplant of HPCs from human cord blood. |
| IMLYGIC (2015) |
HSV-1 | BioVex, Inc., a subsidiary of Amgen Inc. | Recombinant HSV-1 delivers GM-CSF to treat patients with certain unresectable melanoma lesions. |
| KYMRIAH (2017) |
Cell (autologous, modified) | Novartis Pharmaceuticals Corporation | CAR T cells engineered with lentivirus to attack CD19-expressing tumor cells. Treats patients with certain kinds of relapsed or refractory follicular lymphoma. |
| LANTIDRA (2023) |
Cell (allogeneic) | CellTrans Inc. | Pancreatic islet cells from a deceased donor are transplanted to treat patients with certain types of type I diabetes. |
| LENMELDY (2024) |
Gene (ex vivo, autologous) | Orchard Therapeutics (Europe) Limited | Ex vivo gene therapy for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD). |
| LAVIV (2011) |
Cell (autologous, expanded) | Fibrocell Technologies | Fibroblasts to improve the appearance of moderate to severe nasolabial fold wrinkles. |
| LUXTURNA (2017) |
AAV2 | Spark Therapeutics, Inc. | Recombinant AAV2 delivers RPE65 to treat patients with RPE65-mutation-associated retinal dystrophy. |
| LYFGENIA (2023) |
Cell (autologous, modified) | bluebird bio, Inc. | Hematopoietic stem cells engineered with a lentivirus, BB305, expressing modified ßA-globin gene. Treats patients with certain kinds of sickle cell disease. |
| MACI (2016) |
Cell (autologous, expanded) | Vericel Corp. | Cellularized scaffold to repair cartilage defects of the knee. |
| OMISIRGE (2023) |
Cell (allogeneic, modified) | Gamida Cell Ltd. | Transplant of nicotinamide-modified HPCs from human cord blood. |
| PROVENGE (2010) |
Cell (autologous, activated/expanded) | Dendreon Corp. | Antigen presenting cells are activated and used to treat certain types of prostate cancer. |
| RETHYMIC (2021) |
Cell (allogeneic) | Enzyvant Therapeutics GmbH | Thymus tissue implant to improve immune function in patients with certain types of congenital athymia. |
| ROCTAVIAN (2023) |
AAV5 | BioMarin Pharmaceutical Inc. | Recombinant AAV5 delivers a B-domain deleted SQ form of F8 to treat patients with certain types of hemophilia A. |
| SKYSONA (2022) |
Cell (autologous, modified) | bluebird bio, Inc. | Hematopoietic stem cells engineered with Lenti-D vectors that express the ABCD1 gene. Treats patients with a kind of cerebral adrenoleukodystrophy. |
| STRATAGRAFT (2021) |
Cell (allogeneic) | Stratatech Corporation | Cellularized scaffold of keratinocytes and dermal fibroblasts in murine collagen to treat patients with some types of thermal burns. |
| TECARTUS (2020) |
Cell (autologous, modified) | Kite Pharma, Inc. | CAR T cells engineered with gammaretrovirus to attack CD19-expressing tumor cells. Treats patients with certain kinds of relapsed or refractory mantle cell lymphoma. Differs from YESCARTA in that the engineered T cells are enriched for activity against certain B cells. |
| VYJUVEK (2023) |
HSV-1 | Krystal Biotech, Inc. | Recombinant HSV-1 delivers the collagen type VII gene (COL7) to treat certain wounds of patients with dystrophic epidermolysis bullosa with mutations in the COL7A1 gene. |
| YESCARTA (2017) |
Cell (autologous, modified) | Kite Pharma, Inc. | CAR T cells engineered with gammaretrovirus to attack CD19-expressing tumor cells. Treats patients with certain kinds of relapsed or refractory large B-cell lymphoma. |
| ZYNTEGLO (2022) |
Cell (autologous, modified) | bluebird bio, Inc. | Hematopoietic stem cells engineered with a lentivirus, LentiGlobin BB305, to express HBB. Treats patients with certain types of ß-thalassemia. |
| ZOLGENSMA (2019) |
AAV9 | Novartis Gene Therapies, Inc. | Recombinant AAV9 delivers SMN1 to treat patients with certain types of spinal muscular atrophy. |
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As of this writing, that makes 37 different cell and gene therapies that are licensed for use by the United States FDA. We will continue to update throughout 2024!
The TransMission
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