Table 2.
The currently approved viral vector-based gene therapy products.
| Trade Name | Generic Name | Locations Approved | Price * | Indication | Manufacturer | Vector |
|---|---|---|---|---|---|---|
| ABECMA | Idecabtagene vicleucel | US, EU, JP, CAN | $419,500 | Multiple myeloma. | Celgene Corporation, a Bristol Myers Squibb Company. | Lentiviral |
| ADSTILADRIN | Nadofaragene firadenovec | US | $158,600–$262,000 | High-risk Bacillus Calmette-Guérin (BCG)—unresponsive. Non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) and with or without papillary tumors | Ferring Pharmaceuticals A/S. | Adenoviral |
| BREYANZI | Lisocabtagene maraleucel | US, EU, CAN, JP, UK, CH | $470,939.53 | Large B-cell lymphoma (LBCL). | Juno Therapeutics, Inc., a Bristol Myers Squibb Company. | Lentiviral |
| CARVYKTI | Ciltacabtagene autoleucel | US, EU, UK, JP, CAN | $465,000 | Relapsed or refractory multiple myeloma. | Janssen Biotech, Inc. | Lentiviral |
| HEMGENIX | Etranacogene dezaparvovec | US | $3,500,000 | Hemophilia B (congenital Factor IX deficiency). | CSL Behring LLC. | Adeno-associated virus |
| IMLYGIC | Talimogene laherparepvec | US, EU, CHN, UK, AUS | $65,000 | Unresectable cutaneous, subcutaneous, and nodal lesions in patients with recurrent melanoma after the initial surgery. | BioVex Inc., a wholly owned subsidiary of Amgen, Inc. | Herpes simplex virus –1 |
| KYMRIAH | Tisagenlecleucel | US, EU, UK, CAN, JP, AUS, KR, CH | $475,000 | Relapsed or refractory follicular lymphoma. | Novartis Pharmaceuticals Corporation. | Lentiviral |
| LUXTURNA | Voretigene neparvovec | US, EU, UK, AUS, CAN, KR | $850,000 | Confirmed biallelic RPE65 mutation-associated retinal dystrophy | Spark Therapeutics, Inc. | Adeno-associated virus |
| SKYSONA | Elivaldogene autotemcel | US, EU, UK # | $3,000,000 | Neurological dysfunction in boys aged 4–17 years with early, active cerebral adrenoleukodystrophy (CALD). | Bluebird Bio, Inc. | Lentiviral |
| TECARTUS | Brexucabtagene autoleucel | US, EU, UK, CAN | $373,000 | Relapsed or refractory mantle cell lymphoma (MCL) and B-cell precursor acute lymphoblastic leukemia (ALL). | Kite Pharma, Inc. | Retroviral |
| YESCARTA | Axicabtagene ciloleucel | US, EU, UK, JP, CHN, CAN | $373,000 | Large B-cell lymphoma. | Kite Pharma Inc. | Retroviral |
| ZYNTEGLO | Betibeglogene autotemcel | US, CAN, EU # | $2,800,000 | Adult and pediatric patients with ß-thalassemia who require regular red blood cell (RBC) transfusions. | Bluebird bio Inc. | Lentiviral |
| ZOLGENSMA | Onasemnogene abeparvovec | US, EU, JP, AUS, CAN, BRA, TWN, KR, ISL, NO, LIE, UK | $2,125,000 | Spinal muscular atrophy (Type I) | Novartis Gene Therapies, Inc. | Adeno-associated virus |
| GLYBERA | Alipogene tiparvovec | EU # | $1,000,000 | Lipoprotein lipase deficiency. | uniQure biopharma. | Adeno-associated virus |
| LIBMELDY | Atidarsagene autotemcel | EU, UK, ISL, NO | $3,780,000 | Metachromatic leukodystrophy (MLD). | Orchard Therapeutics (Netherlands). | Lentiviral |
| ROCTAVIAN | Valoctocogene roxaparvovec | EU, UK | Around $2,500,000 | Hemophilia A (congenital factor VIII [FVIII] deficiency). | BioMarin International Limited. | Adeno associated virus |
| STRIMVELIS | autologous CD34+ enriched cells | EU, UK | $665,000 | Severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). | Orchard Therapeutics (Netherlands). | Retroviral |
| UPSTAZA | Eladocagene exuparvovec | EU, UK | more than $3,600,000 |
Severe deficiency of aromatic L-amino acid decarboxylase (AADC). | PTC Therapeutics International Limited. | adeno associated virus |
| GENDICINE | Recombinant p53 gene | CHN | N/A | Head and neck cancer. | Shenzhen SiBiono GeneTech Co. Ltd. | Adenoviral |
| ONCORINE | E1B/E3 deficient adenovirus | CHN | N/A | Head and neck cancer; Nasopharyngeal cancer. | Shanghai Sunway Biotech Co. Ltd. | Adenoviral |
| REXIN | G mutant cyclin-G1 gene | PHL | N/A | Solid tumors. | Epeius Biotechnologies. | Retroviral |
| DELYTACT | Teserpaturev | JP | $12,500 | Malignant glioma. | Daiichi Sankyo. | Herpes simplex virus –1 |
| RELMA-CEL | Relmacabtagene autoleucel | CHN | N/A | Diffuse large B-cell lymphoma | JW Therapeutics. | Lentiviral |
| ZALMOXIS | N/A | EU # | $263,000 | Haploidentical hematopoietic stem cell transplantation (HSCT) of adult patients at a high risk of hematological malignancies. | MolMed S.p.A. | Retroviral |
| INVOSSA-K | N/A | KR # | N/A | Osteoarthritis. | Kolon Life Science. | Retroviral |
| RIGVIR | N/A | LV, EE, PL, AM, BLR | N/A | Local treatment of skin and subcutaneous metastases of melanoma. | SIA Latima. | Echovirus 7 |
Data statistics as of 2023/01. #: Withdrawal of the marketing authorization. *: Pricing varies depending on the country. N/A: Not available.