Shire Human Genetic Therapies, Inc.

Mary Alessandrini

Pericles Calias

Michael F Concino

Paolo Martini

Jing Pan

Muthuraman Meiyappan

Angela Norton

Bettina Strack-Logue

Richard Pfeifer

Bohong Zhang

Kevin Holmes

Andrea Iskenderian

Alla Romashko

Dianna Lundberg

Huang Yan

Yan Huang

Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.

Therapeutic fusion protein comprising an alpha-n-acetylglucosaminidase and a lysosomal targeting moiety

Treatment of Sanfilippo syndrome type B by intrathecal administration of alpha-n-acetylglucosaminidase

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Clinton, MA, United States of America

Mary Alessandrini