Approved Therapies

In the table below you will find an overview of gene and cell therapy products approved by EMA and/or FDA, with the exception of cord blood stem cell products. This table will be updated on a yearly basis. For inconsistencies, please contact us.

Brand NameClassificationINN/descriptionIndicationCompanyEMA/FDA approvedMonth/Year approved
Imlygic™In vivo GTMPTalimogene laherparepvec / HSV-1 oncolytic virus containing the cDNA of human GM-CSFAdvanced metastatic melanomaAmgenYes/YesDecember 2015/ October 2015
Luxturna™In vivo GTMPVoretigene neparvovec / AAV2 vector containing human RPE65 cDNALeber congenital amaurosisSpark TherapeuticsYes/YesNovember 2018/December 2017
Zolgensma™In vivo GTMPOnasemnogene abeparvovec / AAV9 vector containing the SMN1 transgeneSpinal muscular dystrophy (SMA)AveXis/ NovartisYes/YesMay 2020/May 2019
Upstaza™In vivo GTMPEladocagene exuparvovec / AAV2 vector containing the cDNA of human dopa decarboxylase (DCC)Patients > 18 months with aromatic L-amino acid decarboxylase deficienciesPTC TxYes/NoJuly, 2022
Roctavian™In vivo GTMPValoctocogene roxaparvovec /AAV5 vector containing the cDNA of the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ)  Adult patients with severe hemophilia ABioMarinYes/NoAugust, 2022
Hemgenix™In vivo GTMPEtranacogene dezaparvovec / AAV5 vector containing the cDNA of human coagulation factor IXAdult patients with hemophilia BCSL Behring/ UniQureNo/YesNovember, 2022
Strimvelis™Ex vivo GTMPAutologous CD34+ cells transduced with a retroviral vector containing the ADA cDNA encoding human adenosine deaminase (ADA)Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)Orchard TxYes/NoMay 2016
Zalmoxis™Ex vivo GTMPAllogeneic T cells genetically modified with a retroviral vector encoding for a truncated form of the human low affinity nerve growth factor receptor (ΔLNGFR) and the herpes simplex I virus thymidine kinase (HSV-TK Mut2)Add on treatment for patients who have received a haploidentical hematopoietic stem cell transplantMolMed S.p.A.Withdrawn/NoAugust 2016
Kymriah™Ex vivo GTMPTisagenlecleucel / autologous CAR T cells directed to CD19Acute Lymphoblastic Leukemia (ALL)NovartisYes/YesAugust 2018/August 2017
Yescarta™Ex vivo GTMPAxicabtagen ciloleucel /  autologous CAR T cells directed to CD19Non-Hodgkin lymphoma: -Large B cell lymphoma -Follicular lymphoma Kite PharmaYes/YesAugust 2018/October 2017
Zynteglo™Ex vivo GTMPBetibeglogene autotemcel / Lentiviral vector containing the cDNA of the human HBB gene.Transfusion-dependent beta thalassemiaBluebird BioYes/YesMay 2019/August 2022
Libmeldy™Ex vivo GTMPAtidarsagene autotemcel / Lentiviral vector containing the human ARSA cDNA encoding arylsulfatase AMetachromatic Leukodystrophy (MLD)Orchard TherapeuticsYes/NoDecember 2020/
Tecartus™Ex vivo GTMPBrexucabtagene autoleucel / autologous CAR T cells directed to CD19Mantle Cell Lymphoma Acute Lymphoblastic Leukemia (ALL)Kite PharmaYes/YesDecember 2020/July 2020
Breyanzi™Ex vivo GTMPLisocabtagen maraleucel / autologous CAR T cells directed to CD19Relapsed or Refractory Large B-cell lymphoma Non-Hodgkin lymphoma Follicular lymphomaBris­tol Mey­ers SquibbYes/YesApril 2022/February 2021
Abecma™Ex vivo GTMPIdecabtagene vicleucel / autologous CAR T -cellsyu76hRelapsed or Refractory Multiple MyelomaCelgeneYes/YesAugust 2021/ March 2021
Carvykti™Ex vivo GTMPCiltacabtagene autoleucel / autologous CAR T cells directed to B cell maturation antigen (BCMA)Relapsed or Refractory Multiple MyelomaJanssen BiotechYes/YesMay 2022/February 2022
Skysona™Ex vivo GTMPElivaldogene autotemcel / autologous CD34+ cells tranduced with retroviral vector containing The cDNA of the human ABCD1Early, Active Cerebral Adrenoleukodystrophy  Bluebird BioWithdrawn/YesSeptember 2022
Provenge™SCTMPSipuleucel-T / autologous dendritic cellsMetastatic castrate-resistant hormone-refractory prostate cancerDendreon PharmaNo/YesWithdrawn/April 2010
Holoclar™SCTMPEx vivo expanded autologous human corneal epithelial cells containing stem cellsPatients with corneal damage due to (chemical) burnsHolostem Advanced TherapiesYes/NoFebruary 2015
Spherox™SCTMPSpheroids of human autologous matrix-associated chondrocytesSymptomatic articular cartilage defects of the femoral condyle and the patella of the kneeCO.DON AgYes/NoJuly 2017
Alofisel™SCTMPDarvadstrocel / Mesenchymal stem cells from fat tissue of adult donorsComplex perianal fistulas in patients with Crohn’s diseaseTakeda PharmaYes/NoMarch 2018
Ebvallo™SCTMPTabelecleucel / allogeneic, EBV-specific T-cells from matched donorSecond-line treatment for transplant recipients who develop, as a result of immunosuppression treatment, Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disease (PTLD)Atara BiotherapeuticsYes/NoOctober 2022
GINTUIT™TEPAllogeneic cultured keratinocytes and dermal fibroblasts in murine collagenTopical (non-submerged) application to a surgically created vascular wound bed in the treatment of mucogingival conditions in adults (venous leg ulcers)Organogenesis Inc.No/YesMarch 2012
MACI™TEPMatrix-applied characterised autologous cultured chondrocytesThe repair of single or multiple symptomatic, full-thickness cartilage defects of the knee with or without bone involvement in adultsVericelWithdrawn/YesDecember 2016
StrataGraft™TEPAllogeneic cultured keratinocytes and dermal fibroblasts in murine collagenAdults with thermal burns containing intact dermal elements for which surgical intervention is clinically indicatedMallinckrodt plcNo/YesJune 2021
Rethymic™TEPHuman allogeneic processed thymus tissue-agdcPediatric patients with Congenital AthymiaEnzyvantNo/YesOctober 2021
Table last updated December 10, 2022


Luxturna (voretigene neparvovec-rzyl) is currently the only approved gene therapy available in the US. It is the first medication approved for an inherited genetic disease ever. Luxturna was approved by the FDA at December 19, 2017 for the United States and it is currently under supervision at the EMA for approval in Europe. It is developed by Spark Therapeutics and outlicensed commercial rights outside the United States to Novartis.

Luxturna is used for patients with an inherited form of retinal dystrophy. Retinal dystrophy is an umbrella term for a wide range of progressive eye diseases. ‘Retinal’ means that the condition is related to the retina, which is the back layer of the eye which converts light into an understandable message to the brain. ‘Dystrophy’ is a degenerative condition. Retinal dystrophy causes progressive reduction or deterioration of vision which eventually can result in complete blindness. Luxturna can be used for patients who have mutations in the RPE65 gene in both chromosomes.

Luxturna is an adeno-associated virus type 2 (AAV2)-based treatment where the correct copy of the RPE65 gene is delivered without disturbing the genome. Luxturna is injected directly into the retina so it can infect the retinal cells. When RPE65 is expressed in those cells, it can perform its function and in principle, halt the progression of the disease.


Imlygic (talimogene laherparepvec) is a genetically modified herpes virus used to treat melanoma. Imlygic was approved by the FDA at October 27, 2015 and by the EMA at December 17, 2015. It was originally developed by BioVex and continued by Amgen after the acquisition of BioVex by Amgen in 2011. Although melanomas shrink due to Imygic, there is no statistically significant benefit in overall survival. In other words, Imlygic does not extend lifes of patients with melanoma. The lack of benefits in combination with the price tag of approximately $65,000 makes Imlygic unpopular to prescribe for doctors.

Imlygic is an oncolytic viral therapy with attenuated life herpes simplex virus type 1 (HSV-1). In HSV-1, two genes are removed and one gene is added. The genes who are removed originally code for the proteins infected cell protein 34.5 (ICP34.5) and infected cell protein 47 (ICP47). ICP34.5 blocks the response of healthy cells to stop replicating and die after viral infection. However, cancer cells lack this system. By removing ICP34.5, HSV-1 can not replicate and kill normal cells, but in cancer cells they can. ICP47 suppresses an immune response to viral infection. Removing ICP47 should trigger an immune response, in theory. A gene coding for granulocyte colony-stimulating factor (GM-CSF) is inserted to promote an immune response to cancer cells infected by Imlygic. Despite these efforts, clinical evidence for an immune response to cancer cells due to Imlygic is not available.

Note: this page is under construction. New gene and cell therapy products will be added soon.