Abstract: 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.

Abstract: Described are fusion proteins of GLP-2 with an Fc region of immunoglobulin. The GLP-2 and Fc regions are separated by a linker comprised of amino acids. The fusion proteins persist and remain active in the body for longer periods of time than GLP-2 itself. Methods are disclosed of using the fusion proteins to treat and prevent enterocutaneous fistulae, radiation damage to the gastrointestinal tract, obstructive jaundice, and short bowel syndrome.

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.

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

Abstract: Described are fusion proteins of GLP-2 with an Fc region of immunoglobulin. The GLP-2 and Fc regions are separated by a linker comprised of amino acids. Methods are disclosed of using the fusion proteins to treat and prevent enterocutaneous fistulae, radiation damage to the gastrointestinal tract, obstructive jaundice, and short bowel syndrome.

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.

Abstract: 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.

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

Abstract: 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.

Abstract: 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.

Abstract: Described are fusion proteins of GLP-2 with an Fc region of immunoglobulin. The GLP-2 and Fc regions are separated by a linker comprised of amino acids. The fusion proteins persist and remain active in the body for longer periods of time than GLP-2 itself. Methods are disclosed of using the fusion proteins to treat and prevent enterocutaneous fistulae, radiation damage to the gastrointestinal tract, obstructive jaundice, and short bowel syndrome.

Abstract: 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.

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

Abstract: Multi-specific molecules (e.g., engineered antibodies) that specifically bind to one or more targets, e.g., in a pH sensitive manner, are described, as well as methods of making and using such multi-specific molecules (e.g., engineered antibodies).

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

Abstract: 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.

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.

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.

Abstract: 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.

Abstract: 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.