Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Criteo S.a.

Bohong Zhang

Michael F Concino

Muthuraman Meiyappan

Angela Norton

Bettina Strack-Logue

Kevin Holmes

Andrea Iskenderian

Dianna Lundberg

Jing Pan

Alla Romashko

Pericles Calias

Paolo Martini

Richard Pfeifer

Lieh Yoon Low

Huang Yan

Mary Alessandrini

Thomas Allen Miller

Clark Qun Pan

Hicham Naimy

Omar L Francone

Jianwen Feng

Lin Guey

Bruce Tangarone

Matthew Traylor

Lenore Von Krusenstiern

Tracy Dowie

Jun Hu

Brian Dwyer

Paul McLean

Angela W Norton

Yan Huang

The invention relates to an affinity resin functionalized with small molecule inhibitors of glycoside-cleaving enzymes, e.g., &#x3b1;-galactosidase A (&#x3b1;-Gal A), glucocerebrosidase (GCB), &#x3b2;-galactosidase, and acid alpha-glucosidase (GAA), and a method for purifying glycoside-cleaving enzymes produced in a cell line using the small molecule inhibitor-functionalized affinity resin.

Affinity purification of glycosidase-cleaving enzymes

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

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

The invention provides compositions and methods for effective lysosomal targeting mediated by PCSK9. In particular, the compositions and methods provided by the invention may be used to treat lysosomal storage diseases such as Pompe Disease and Sanfilippo Syndrome Type B, and they may be used for targeting lysosomal enzymes to the various muscles of the human body.

Lysosomal targeting of enzymes, 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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Lexington, MA, United States of America

Bohong Zhang