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Drug Names(s): AAV Sanfilippo IIIB, adeno-associated viral vector serotype 9 containing human N-acetylglucosaminidase alpha gene, MPS IIIB Gene Therapy (Esteve), EGT201, EGT 201
Description: Esteve is developing a viral gene therapy that contains the human N-acetylglucosaminidase alpha gene for the treatment of mucopolysaccharidosis III type B.
Deal Structure: The project Sanfilippo is developed under a Public-Private Partnership (PPP) between Esteve and the UAB (Universitat Autnoma de Barcelona) for the development of gene therapies for mucopolysaccharidosis.
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