Treatment of sanfilippo syndrome type b by intrathecal administration of alpha-n-acetylglucosaminidase

US9814764B2 · kind B2 · utility

Assignee

• SHIRE HUMAN GENETIC THERAPIES, INC. · US

Inventors

• Michael F. Concino · Bolton, US
• Pericles Calias · Melrose, US
• Jing Pan · Newark, US
• Kevin Holmes · Belmont, US
• Paolo Martini · Boston, US
• Alla Romashko · Lexington, US
• Muthuraman Meiyappan · Lexington, US
• Bohong Zhang · Lexington, US
• Andrea Iskenderian · Lexington, US
• Dianna Lundberg · Brentwood, US
• Angela Norton · Reading, US
• Bettina Strack-Logue · Somerville, US
• Huang Yan · Billerica, US
• Mary Alessandrini · Clinton, US
• Richard Pfeifer · Granby, US

Key dates

• Filing date: May 10, 2013
• Grant date: Nov 14, 2017
• Expiry date: Jun 25, 2033

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12Y310/01001
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.