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., 2018Zhang W-W, Li L, Li D, Liu J, Li X, Li W, Xu X, Zhang MJ, Chandler LA, Lin H et al. (2018) The first approved gene therapy product for cancer Ad-p53 (Gendicine): 12 years in the clinic. Hum Gene Ther 29:160-179.). |
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., 2010Chawla SP, Chua VS, Fernandez L, Quon D, Blackwelder WC, Gordon EM and Hall FL (2010) Advanced phase I/II studies of targeted gene delivery in vivo: Intravenous Rexin-G for gemcitabine-resistant metastatic pancreatic cancer. Mol Ther 18:435-441.) |
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., 2015Deev RV, Bozo IY, Mzhavanadze ND, Voronov DA, Gavrilenko AV, Chervyakov YV, Staroverov IN, Kalinin RE, Shvalb PG and Isaev AA (2015) pCMV-vegf165 intramuscular gene transfer is an effective method of treatment for patients with chronic lower limb ischemia. J Cardiovasc Pharmacol Ther 20:473-482.) |
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2012 - Approved 2014 - Withdrawn |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 |