Shire Human Genetic Therapies, Inc.

Dianna Lundberg

Michael F Concino

Muthuraman Meiyappan

Angela Norton

Bettina Strack-Logue

Bohong Zhang

Kevin Holmes

Andrea Iskenderian

Alla Romashko

Pericles Calias

Paolo Martini

Jing Pan

Richard Pfeifer

Mary Alessandrini

Huang Yan

Lieh Yoon Low

Hicham Naimy

Omar L Francone

Lin Guey

Bruce Tangarone

Matthew Traylor

Lenore Von Krusenstiern

Tracy Dowie

Angela W Norton

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

A targeted therapeutic including a lysosomal enzyme and a lysosomal targeting moiety that is a peptide containing at least one N-linked glycosylation site. Methods of producing the targeted therapeutic may include nucleotide acids encoding the same and host cells co-expressing GNPT. Pharmaceutical compositions comprising the targeted therapeutic and methods of using the same to treat a lysosomal storage disease.

Mannose-6-phosphate bearing peptides fused to lysosomal enzymes

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

A lipoprotein lipase (LPL) protein for treating and/or preventing HTG and its associated diseases, including but not limited to acute pancreatitis (AP), and in particular, acute pancreatitis secondary to or exacerbated by hypertriglyceridemia, and hypertriglyceridemia and its associated diseases in general, including cardiovascular and metabolic diseases, endocrine disorders, and fat embolism syndrome.

Lipoprotein lipase for treatment of hypertriglyceridemic-related conditions including acute pancreatitis

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Brentwood, NH, United States of America

Dianna Lundberg