Table 2-. Approved gene therapies for gene addition or gene replacement.
| Type | Name | Indication | Method | Immune-related issues* | Situation |
|---|---|---|---|---|---|
| in vivo | |||||
| Gendicine | Head and neck squamous cell carcinoma | Recombinant human serotype-5 adenovirus | Fever, chill, pain at the injection site, fatigue, nausea, and diarrhea (Zhang et al., 2018). | 2003 - Approved | |
| Oncorine | Nasopharyngeal carcinoma | Recombinant human Adenovirus type 5 injection, H101 | Fever, local pain at the injection site, and flu-like symptoms. (Ma et al., 2009). | 2005 - Approved | |
| Rexin-G | Metastatic pancreatic cancer | Retroviral vector | Chill, fatigue and headache. No serious drug-related adverse events (AE) were reported. (Chawla et al., 2010) | 2007 - Approved | |
| Neovasculgen | Atherosclerotic Peripheral Arterial Disease (PAD) | Plasmid DNA | Prophylactic treatment with acetylsalicylic acid to decrease risk of cardiovascular ischemic event. No AEs related. (Deev et al., 2015) | 2010 - Approved | |
| Glybera | Lipoprotein Lipase Deficiency (LPLD) | Adeno-associated virus serotype I | Immune response against AAV even with use of immunosuppressants. | 2012 - Approved 2014 - Withdrawn | |
| Imlygic | Melanoma | Genetically manipulated oncolytic herpes simplex virus type 1 (HSV) | Flu-like illness, fevers and chills. Muscle pain (myalgia), painful/swollen joints (arthralgia), limb pain, vasculitis and glomerulonephritis (very rare). Autoimmmune reactions. Plasmocytoma. | 2015 - Approved | |
| Luxturna | Inherited retinal dystrophies | Adeno-associated virus | Acute serious liver injury, acute liver failure, and elevated aminotransferases. | 2017 - Approved | |
| Zolgensma | Treatment of Spinal Muscular Atrophy (Type I) | Non-replicating recombinant AAV9 | Acute and chronic GvHD, febrile neutropenia, haemoglobin decreased, platelet count decreased. | 2019 - Approved | |
| Delytact | Residual or recurrent glioblastoma | Oncolytic virus therapy - replication-conditional Herpes simplex virus type 1 | Pyrexia, brain oedema, cytopenia, seizure, haemorrhage, infection, normal pressure hydrocephalus, and autoimmune diseases involving the central nervous system. | 2021 - Conditional approval in Japan | |
| ex vivo | |||||
| Strimvelis | Severe combined immunodeficiency | Autologous Hematopoietic stem cells retroviral vector GSK3336223 | Anaemia, neutropenia, autoimune haemolytic anaemia, autoimune aplastic anaemia, autoimune thrombocytopenia, autoimune thyroiditis, Guillain-Barré syndrome, rhinitis allergic, asthma, dermatites atopic, eczema. | 2016 - Approved | |
| Zalmoxis | Hematopoietic Stem Cell Transplantation | Allogeneic T cells genetically modified with a retroviral vector | Acute and chronic GvHD, febrile neutropenia, haemoglobin decreased, platelet count decreased, hepatic failure, bronchitis and hepatic failure. | 2016 - Approved 2019 - Withdrawn | |
| Kymriah | B-cell precursor acute lymphoblastic leukemia | Autologous T cells - CAR T cell therapy modified with lentiviral vector | Cytokine Release Syndrome. Neurological toxicities. Infections and Febrile Neutropenia. Prolonged Cytopenias. Hypogammaglobulinemia. Other manifestations included seizures, mutism and aphasia. | 2017 - Approved | |
| Yescarta | Refractory large B-cell lymphoma | Autologous T cells - CAR T cell therapy modified by retroviral transduction | Cytokine Release Syndrome. Neurologic toxicities, both including fatal or life-threatening reactions. | 2017 - Approved | |
| Invossa | Knee osteoarthritis | Allogeneic chondrocytes | Under Phase III for safety and efficacy evaluation. | 2017 - Phase III Clinical Trial approved in US |
* Information obtained from package insert, unless indicated otherwise.