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 |