Available Treatments

Several cell and gene therapies have been approved in the United States and in Europe:

NAME OF CONDITION
THERAPY
NAME
BRAND NAME
MANUFACTURER
LOCATION OF APPROVED USE
Heart Abnormality
Tissue Engineered Medical Product Bio-scaffold material
Intracardiac Patch
CardioCel
Admedus
USA, Europe, Canada, Singapore
Cancer – leukemia
CAR -T cell therapy (CD19-directed genetically modified autologous T-cell immunotherapy)
tisagenlecleucel
Kymriah
Novartis Pharmaceuticals Corporation
USA, Europe, Canada, Japan
Cancer – B Cell Malignancies
CAR -T cell therapy (CD19-directed genetically modified autologous T-cell immunotherapy)
axicabtagene ciloleucel
Yescarta
Kite Pharma, Incorporated
USA, Europe, Canada
Retinal dystrophy
Gene therapy (Adeno-associated virus vector-based gene therapy)
voretigene neparvovecrzyl
Luxturna
Spark Therapeutics, Inc.
USA, Europe
Cartilage defects in the knee
Tissue engineered medical product — Autologous cellularized scaffold product
Autologous Cultured Chondrocytes on a Porcine Collagen Membrane
MACI
Vericel Corporation
USA
Severe burns
Tissue Engineered medical product — autologous keratinocytes
Cultured Epidermal Autografts
Epicel
Vericel
USA
Cancer – Prostate cancer
Autologous cellular immunotherapy
Autologous Cellular Immunotherapy
Provenge
Dendreon Corp
USA
Cancer – melanoma
Gene therapy directed at the tumor itself
Talimogene laherparepvec
Imlygic
Amgen
USA, Europe, Australia
Spinal Muscular Atrophy
Adeno-associated virus vestor based gene therapy
Onasemnogene abeparvovec- xioi
Zolgensma
AveXis, Inc. (Novartis Gene Therapy)
Europe
Oral soft tissue
Tissue engineered medical product – Allogeneic cellular sheet with other factors
Allogeneic cultured keratinocytes and fibroblasts in bovine collagen
Gintuit
Organogenesis Inc.
USA
Sickle Cell Disease
Gene modified autologous CD34+ cells that contains HSC transduced with lentiviral vector (LVV) encoding the βA-T87Q -globin gene
Betibeglogene autotemcel
Zynteglo
Bluebird bio
Europe
Spinal Muscular Atrophy
survival motor neuron-2 (SMN2)-directed antisense oligonucleotide
Nusinersen
Spinraza
Biogen
USA, Europe
Facial Wrinkles
Autologous cellular product
Laviv
Azficel-T
Fibrocell Technologies, Inc.
USA
Diabetic Foot ulcers
tissue engineered medical product — Bilayer dermal regeneration matrix
bilayer matrix for dermal regeneration
Omnigraft
Integra
USA