Shire Human Genetic Therapies, Inc.

Translate Bio, Inc.

Takeda Pharmaceutical Company Limited

Bettina Strack-Logue

Michael F Concino

Angela Norton

Muthuraman Meiyappan

Bohong Zhang

Michael W Heartlein

Kevin Holmes

Andrea Iskenderian

Dianna Lundberg

Paolo Martini

Jing Pan

Alla Romashko

Frank DeRosa

Pericles Calias

Richard Pfeifer

Dennis Keefe

Serene Josiah

Huang Yan

Mary Alessandrini

Clark Qun Pan

Marcia Sellos-Moura

Frank Derosa

Lieh Yoon Low

Juan Ruiz

Hicham Naimy

Omar L Francone

Luying Pan

Clément Olivier

Jianwen Feng

Lin Guey

Bruce Tangarone

Matthew Traylor

Lenore Von Krusenstiern

Tracy Dowie

Pan Luying

Scott Alderucci

Angela W Norton

Yan Huang

GLP-2 analogs, such as teduglutide, and fusion proteins of GLP-2 with immunoglobulin are administered before, during or after surgery, particularly resection of the small intestine. When short bowel syndrome develops after surgical resection of the small intestine, parenteral nutrition is usually necessary to compensate for reduced absorption of water and nutrients across the small intestine. GLP-2 analogs and GLP-2 fusion proteins promote growth of small intestine, improve nutrient absorption and can reduce the need for parenteral nutrition after surgery.

GLP-2 analogs and peptibodies for administration before, during or after surgery

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 present invention provides, among other things, methods and compositions for large-scale production of capped mRNA using SUMO-Guanylyl Transferase fusion protein.

Modification of RNA-related enzymes for enhanced production

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 present invention provides, among other things, compositions and methods for treatment of Friedrich's Ataxia based on effective targeting of a therapeutic moiety to mitochondria that can substitute for natural FXN protein activity or rescue one or more phenotypes or symptoms associated with frataxin-deficiency. In some embodiments, the present invention provides a targeted therapeutic comprising a therapeutic moiety, which is a polypeptide having an N-terminus and a C-terminus, a mitochondrial targeting sequence associated with the therapeutic moiety at the N-terminus, and a mitochondrial membrane-penetrating peptide associated with the therapeutic moiety at the C-terminus, wherein the therapeutic moiety is targeted to mitochondria upon cellular entry.

Mitochondrial targeting and therapeutic use 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

Described herein are isolated nucleic acid molecules comprising nucleotide sequence encoding mannose receptor, C type 1 (MRC1) wherein the 5' region of the nucleotide sequence encoding MRC1 is codon optimized; cells comprising such nucleic acid molecules; and methods of detecting antibody production, e.g., neutralizing antibody production, in a subject being treated for Gaucher disease using such cells.

Mannose receptor C type 1 (MRC1) codon optimized cell line and uses thereof

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Somerville, MA, United States of America

Bettina Strack-Logue