Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Shire-nps Pharmaceuticals, Inc.

Kevin Holmes

Angela Norton

Michael F Concino

Muthuraman Meiyappan

Bettina Strack-Logue

Bohong Zhang

Jing Pan

Andrea Iskenderian

Dianna Lundberg

Clark Qun Pan

Pericles Calias

Paolo Martini

Richard Pfeifer

Alla Romashko

Sujit Jain

Chuan Shen

Rongxin Shi

Mary Alessandrini

Huang Yan

Omar L Francone

Lieh Yoon Low

Jianwen Feng

Lin Guey

Bruce Tangarone

Matthew Traylor

Lenore Von Krusenstiern

Tracy Dowie

Bryan James Goodwin

David Bruce Lloyd

Bryan Goodwin

David Lloyd

Angela W Norton

Yan Huang

The invention relates to parathyroid hormone (PTH) variants and pharmaceutical compositions comprising same. The invention further relates to PTH compositions with improved serum half-life and peak-trough ratios, and methods of controlling serum calcium levels with the PTH variants and compositions of the invention. The invention further relates to methods of treating hypoparathyroidism and/or hypocalcemia due to hypoparathyroidism with the PTH variants and compositions of the invention.

Parathyroid hormone variants

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

The invention provides compositions and methods for effective lysosomal targeting mediated by SORT1. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Sanfilippo syndrome type B.

Lysosomal targeting and uses thereof

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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Belmont, MA, United States of America

Kevin Holmes