Shire Human Genetic Therapies, Inc.

Huang Yan

Pericles Calias

Michael F Concino

Paolo Martini

Jing Pan

Angela Norton

Muthuraman Meiyappan

Bettina Strack-Logue

Richard Pfeifer

Bohong Zhang

Kevin Holmes

Andrea Iskenderian

Dianna Lundberg

Alla Romashko

Mary Alessandrini

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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Billerica, MA, United States of America

Huang Yan