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Gene therapy treatments for rare diseases
Diseases of the muscles, immune system, blood, retina…
An integrated structure: from research to clinical trials
Globally unrivalled bioproduction capacities
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Pre-clinical phase
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Gamma-sarcoglycanopathy (IM administration)
Wiskott-Aldrich Syndrome (WAS)
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Leber’s Hereditary Optic Neuropathy
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Bioproduction
Genethon Bioprod
, the world’s largest center for the production of clinical grade gene therapy products
