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Drug Names(s): AAV Sanfilippo IIIA, adeno-associated virus vector serotype 9 expressing human sulfamidase, MPS IIIA Gene Therapy (Esteve), EGT101, EGT 101
Description: Esteve is developing a novel gene therapy treatment, based on an adeno-associated virus (AAV) vector containing the human sulfamidase gene (AAV-sulfamidase), for Sanfilippo syndrome type A (caused by the lack of heparan N-sulfatase or sulfamidase). AAV-sulfamidase is a genetically modified viral vector derived from a wild-type AAV, a non-pathogenic virus. The recombinant vector has been designed to maximise the sulfamidase therapeutic transgene expression.
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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