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The TransMission: News

Current FDA Approved Gene and Cell Therapies

Last Updated: January 10, 2023

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Happy New Year from Mirus Bio!

In 2022, five 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 ring in the new year and celebrate the accomplishments of the gene and cell therapy scientific and patient community, let's reflect on all current FDA approved gene and cell therapies to date. The table below contains all licensed products listed as 'Approved Cellular and Gene Therapy Products' on fda.gov (last updated on 12/16/2022).

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.
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.
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.
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.
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.
MACI
(2016)
Cell (autologous, expanded) Vericel Corp. Cellularized scaffold to repair cartilage defects of the knee.
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.
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.
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. 

 

As of this writing, that makes 27 different cell and gene therapies that are licensed for use by the United States FDA. Cheers to 2023!

 

Are you developing a novel cell or gene therapy? We'd love to hear your story in the Comments section below or via email to techsupport@mirusbio.com.

 

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