US Patent:
20110318327, Dec 29, 2011
Inventors:
Michael F. Concino - Bolton MA, US
Pericles Calias - Melrose MA, US
Jing Pan - Boxborough MA, US
Kevin Holmes - Belmont MA, US
Paolo Martini - Boston MA, US
Alla Romashko - Lexington MA, US
Muthuraman Meiyappan - Jamaica Plain MA, US
Bohong Zhang - Newton MA, US
Andrea Iskenderian - Arlington MA, US
Dianna Lundberg - Brentwood NH, US
Angela Norton - Reading MA, US
Bettina Strack-Logue - Somerville MA, US
Yan Huang - Billerica MA, US
Mary Alessandrini - Clinton MA, US
Richard Pfeifer - North Granby CT, US
Assignee:
SHIRE HUMAN GENETIC THERAPIES, INC. - Cambridge MA
International Classification:
A61K 38/47
A61P 43/00
C12N 9/24
Abstract:
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.