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https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products

https://www.whatisbiotechnology.org/index.php/science/summary/gene-therapy/		Date of approvalDiseaseGene Therapy Agent# Individuals AffectedGene Therapy StrategyCitationSide Effects
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Retinal Degeneration12/19/2017Retinal dystrophy (bi-allelic mutation of RPE65)LUXTURNA (voretigene neparvovec-rzyl)2–3 per 100,000 individualsSubretinal injection adeno-associated virus vector-based gene therapy (AAV2)https://www.fda.gov/media/109906/download?attachment

https://www.cell.com/molecular-therapy-family/molecular-therapy/pdf/S1525-0016(20)30655-9.pdf#page=17&zoom=100,65,272

https://www.drugs.com/sfx/luxturna-side-effects.html		Conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula)
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Spinal Muscular Atrophy5/24/2019Spinal muscular atrophy (pediatric patients < 2 years old with bi-allelic mutations in SMN1)ZOLGENSMA (onasemnogene abeparvovec-xioi)1/10,000 live birthsIn-vivo adeno-associated virus vector-based gene therapy (AAV9)https://www.fda.gov/media/126109/download?attachment

https://www.cdc.gov/nceh/dls/nsmbb_sma.html#:~:text=Spinal%20Muscular%20Atrophy%20(SMA)%20is,being%20unable%20to%20make%20protein.

https://journals-sagepub-com.proxy.cc.uic.edu/doi/full/10.1177/1060028020914274

https://www.drugs.com/sfx/zolgensma-side-effects.html		Thrombocytopenia, Respiratory insufficiency, thrombotic microangiopathy, Elevated Liver Enzymes, vomiting, elevated troponin-I levels, fevers
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Beta-Thalassemia8/17/2022Transfusion-dependent ß-thalassemiaZYNTEGLO (betibeglogene autotemcel)Rare in USA (exact number is unknown, estimated that 1,300 people in U.S. are affected)Ex-vivo Lenti-D lentiviral vector gene therapy (LVV)https://www.fda.gov/media/160991/download?attachment

https://www.bluebirdbio.com/our-focus/beta-thalassemia#:~:text=As%20a%20rare%20disease%2C%20it,Asian%2C%20and%20Middle%20Eastern%20descent.

https://www.drugs.com/zynteglo.html		Tachycardia, pain in arm or legs, Alopecia, rash, mucositis, vomiting, abdominal pain, diarrhea, nausea, constipation, leukopenia, thrombopenia
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X-linked Adrenoleukodystrophy9/16/2022Early Cerebral AdrenoleukodystrophySKYSONA (elivaldogene autotemcel)1/10,000 - 1/17,000Ex-vivo Lenti-D lentiviral vector gene therapy (LVV)https://www.fda.gov/media/161640/download

https://rarediseases.org/rare-diseases/adrenoleukodystrophy/

https://www.drugs.com/skysona.html		Tachycardia, Alopecia, pruritus, rash, skin hyperpigmentation, fever, febrile neutropenia, hypertension, transfusion reaction, decreased appetite, anxiety, headache, vision blurred, cough, epistaxis, oropharyngeal pain
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Hemophilia B11/22/2022Hemophilia BHEMGENIX (etranacogene dezaparvovec-drlb)1/25,000 Male BirthsIn-vivo adeno-associated virus vector-based gene therapy (AAV5)https://www.fda.gov/vaccines-blood-biologics/vaccines/hemgenix

https://rarediseases.org/rare-diseases/hemophilia-b/#affected

https://www.drugs.com/sfx/hemgenix-side-effects.html		Increased Liver Enzymes, headache, hypersensitivity, flu-like symptoms, fatigue, nausea, malaise
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Bladder Cancer (without invasion of the muscle wall)12/16/2022Non-muscle invasive bladder cancer (NMIBC) (unresponsive to BCG treatment)ADSTILADRIN (nadofaragene firadenovec-vncg)NMIBC incidence =75% of 81,190 estimated newly diagnosed bladder cancer cases in USA. This is about 1/5,000 - 1/6,000.In-vivo Intravesical Instillation adeno-associated virus vector-based gene therapy (AAV5)https://www.fda.gov/media/164029/download?attachment

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6615913/

https://www.drugs.com/sfx/adstiladrin-side-effects.html		Glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination)
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Hemophilia A6/30/2023Hemophilia AROCTAVIAN (valoctocogene roxaparvovec-rvox)1/5,000 Male BirthsIn-vivo adeno-associated virus vector-based gene therapy (AAV5)https://www.fda.gov/vaccines-blood-biologics/roctavian

https://rarediseases.org/rare-diseases/hemophilia-a/

https://www.drugs.com/roctavian.html		Increased Liver Enzymes, Nausea, fatigue, headache, infusion-related reactions, vomiting, abdominal pain, hepatotoxicity, thromboembolic events.
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Acute Lymphoblastic Leukemia (and two types of non-Hodgkin lymphomas (FL and DLBCL))08/30/2017 (ALL)
ALL/DLBCL/FL (in patients that are refractory or in second or later relapse)KYMRIAH (tisagenlecleucel)34 per 1 million (pediatric ALL)Ex-vivo CD19-directed CAR-T cell therapyhttps://www.fda.gov/media/107296/download?attachment

https://www.cdc.gov/mmwr/volumes/66/wr/mm6636a3.htm#:~:text=Acute%20lymphoblastic%20leukemia%20(ALL)%20is%20the%20most%20prevalent%20cancer%20among,cases%20each%20year%20(1).

https://www.drugs.com/sfx/kymriah-side-effects.html		Cytokine release syndrome, infections-pathogen unspecified, hypogammaglobulinemia, fever, decreased appetite, viral infectious disorders, headache, febrile neutropenia, hemorrhage, musculoskeletal pain, vomiting, encephalopathy, diarrhea, hypotension, cough, nausea, bacterial infectious disorders, pain, hypoxia, tachycardia, edema, fatigue, and acute kidney injury
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SCIDClinical TrialsADA and X-linked are major ones of notehttps://clinicaltrials.gov/study/NCT05432310

https://primaryimmune.org/understanding-primary-immunodeficiency/treatment/gene-therapy
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Sickle cell diseaseClinical Trials
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