Today, researchers and clinicians are under great pressure to develop therapies capable of reversing or significantly impacting the progression of disease. In recent years, the arrival of the cell and gene therapy industries have introduced the possibility of providing transformative, durable and potentially curative outcomes for a diverse range of life-threatening conditions, as well as injuries, degenerative diseases, genetic disorders, and cancers.
Cell therapy is the utilization of living cells for the treatment or cure of disease. It can involve both native (unmodified), as well as engineered or gene-edited cells. It also encompasses the exosomes and extracellular vesicles that are secreted by living cells.
Gene therapy is involves the insertion of functional genes into cells to replace faulty genes for the purpose of treating genetic diseases. Usually, a virus is genetically engineered to carry and deliver the therapeutic gene into the cells of the patient’s body. When the treatment is successful, the new gene delivered by the vector produces a functioning protein. There are several techniques to perform gene therapies, which include gene augmentation therapy, gene inhibition therapy, and suicide gene insertion, as well as other methods.
FDA Approved Cell and Gene Therapies
Below is a list of all known cell and gene therapy products that have been licensed by the Office of Tissues and Advanced Therapies (OTAT), a division of the U.S. FDA. At present, 32 cell and gene therapies have been approved within the United States. To date, no exosome therapies have received FDA approval, although the clinical trial pipeline looks promising.
The majority of these products are umbilical cord blood derivatives, representing 8 of the 32 cell and gene therapy approvals to date (25.0%).
CAR-T cell therapies represent the next largest segment, composing 6 of the 32 cell and gene therapy approvals (18.9%). The approved CAR-T cell therapies in the U.S. include: Abcema, Breyanzi, Carvykti, Kymriah, Tecartus, and Yescarta (listed in alphabetical order).
For each product, the list below presents its brand name, clinical name, and importantly, the company who developed it.
- ABECMA (idecabtagene vicleucel)
Celgene Corporation, a Bristol-Myers Squibb Company - ADSTILADRIN
Ferring Pharmaceuticals A/S - ALLOCORD (HPC, Cord Blood)
SSM Cardinal Glennon Children’s Medical Center - BREYANZI
Juno Therapeutics, Inc., a Bristol-Myers Squibb Company - CARVYKTI (ciltacabtagene autoleucel)
Janssen Biotech, Inc. - CLEVECORD (HPC Cord Blood)
Cleveland Cord Blood Center - Ducord, HPC Cord Blood
Duke University School of Medicine - ELEVIDYS
delandistrogene moxeparvovec - GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen)
Organogenesis Incorporated - HEMACORD (HPC, cord blood)
New York Blood Center - HEMGENIX
CSL Behring LLC - HPC, Cord Blood
Clinimmune Labs, University of Colorado Cord Blood Bank - HPC, Cord Blood – MD Anderson Cord Blood Bank
MD Anderson Cord Blood Bank - HPC, Cord Blood – LifeSouth
LifeSouth Community Blood Centers, Inc. - HPC, Cord Blood – Bloodworks
Bloodworks - IMLYGIC (talimogene laherparepvec)
BioVex, Inc., a subsidiary of Amgen Inc. - KYMRIAH (tisagenlecleucel)
Novartis Pharmaceuticals Corporation - LANTIDRA (donislecel)
CellTrans Inc. - LAVIV (Azficel-T)
Fibrocell Technologies - LUXTURNA
Spark Therapeutics, Inc. - MACI (Autologous Cultured Chondrocytes on a Porcine Collagen Membrane)
Vericel Corp. - OMISIRGE (omidubicel-onlv)
Gamida Cell Ltd. - PROVENGE (sipuleucel-T)
Dendreon Corp. - RETHYMIC
Enzyvant Therapeutics GmbH - ROCTAVIAN (valoctocogene roxaparvovec-rvox)
BioMarin Pharmaceutical Inc - SKYSONA (elivaldogene autotemcel)
bluebird bio, Inc. - STRATAGRAFT
Stratatech Corporation - TECARTUS (brexucabtagene autoleucel)
Kite Pharma, Inc. - VYJUVEK
Krystal Biotech, Inc. - YESCARTA (axicabtagene ciloleucel)
Kite Pharma, Incorporated - ZYNTEGLO (betibeglogene autotemcel)
bluebird bio, Inc. - ZOLGENSMA (onasemnogene abeparvovec-xioi)
Novartis Gene Therapies, Inc.