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Up-to-date list of FDA and EMA approved gene therapy drugs. All information is verified from official sources.
onasemnogene abeparvovec
Spinal Muscular Atrophy
voretigene neparvovec-rzyl
Leber Congenital Amaurosis
exagamglogene autotemcel
Sickle Cell Disease / Beta-Thalassemia
lovotibeglogene autotemcel
Sickle Cell Disease
etranacogene dezaparvovec
Hemophilia B
valoctocogene roxaparvovec
Hemophilia A
delandistrogene moxeparvovec
Duchenne Muscular Dystrophy
elivaldogene autotemcel
Cerebral Adrenoleukodystrophy
nadofaragene firadenovec
Bladder Cancer (BCG-unresponsive)
betibeglogene autotemcel
Beta-Thalassemia
autologous CD34+ enriched cell fraction
ADA-SCID (Severe Combined Immunodeficiency)
beremagene geperpavec
Dystrophic Epidermolysis Bullosa
atidarsagene autotemcel
Metachromatic Leukodystrophy (MLD)
fidanacogene elaparvovec
Hemophilia B
talimogene laherparepvec
Melanoma (Unresectable)
All drugs containing "gene therapy" in FDA drug labels — fetched automatically.
Data Sources: OpenFDA API, FDA CBER, EMA