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AssigneeShire Human Genetic Therapies, Inc.
InventorsMichael F. Concino, Pericles Calias, Jing Pan, Kevin Holmes, Paolo Martini, Alla Romashko, Muthuraman Meiyappan, Bohong Zhang, Andrea Iskenderian, Dianna Lundberg, Angela Norton, Bettina Strack-Logue, Huang Yan, Mary Alessandrini, Richard Pfeifer
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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.
A method of treating Sanfilippo syndrome type B (San B) disease comprising a step of
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A therapeutic fusion protein comprising
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A therapeutic fusion protein comprising an amino acid sequence at least 80% identical to SEQ ID NO:6 (the full-length Naglu-IGF-II fusion protein), wherein, once administered, the therapeutic fusion protein is targeted to lysosomes and is therapeutically active in vivo.
Dependencies are inferred from the claim text.