| Shire Human Genetic Therapies, Inc Patent applications |
| Patent application number | Title | Published |
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| 20120021436 | Crystal structure of human alpha-N-acetylglucosaminidase - The present invention provides the three-dimensional structure of human α-N-acetylglucosaminidase (NAGLU) protein. This crystallographic information is useful in the identification and development of novel binding compounds of NAGLU, NAGLU mutants, for example, those associated with Sanfilippo syndrome type B (mucopolysaccharidosis III B (MPS III-B)), and other NAGLU family members (family 89 α-N-acetylglucosaminidase) which may modulate the activity and/or stability of mutated NAGLU. Such compounds may be useful for the treatment of Sanfilippo syndrome type B (mucopolysaccharidosis III B (MPS III-B)). | 01-26-2012 |
| 20120014936 | METHODS AND COMPOSITIONS FOR CNS DELIVERY OF HEPARAN N-SULFATASE - The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A. | 01-19-2012 |
| 20120009171 | METHODS AND COMPOSITIONS FOR CNS DELIVERY OF ARYLSULFATASE A - The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an arylsulfatase A (ASA) protein, salt, and a polysorbate surfactant for the treatment of Metachromatic Leukodystrophy Disease. | 01-12-2012 |
| 20120003202 | CNS DELIVERY OF THERAPEUTIC AGENTS - The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme. | 01-05-2012 |
| 20110318327 | TREATMENT OF SANFILIPPO SYNDROME TYPE B - 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. | 12-29-2011 |
| 20110318324 | METHODS AND COMPOSITIONS FOR CNS DELIVERY OF B-GALACTOCEREBROSIDASE - The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an B-Galactocerebrosidase protein, salt, and a polysorbate surfactant for the treatment of GLD Disease. | 12-29-2011 |
| 20110318323 | METHODS AND COMPOSITIONS FOR CNS DELIVERY OF IDURONATE-2-SULFATASE - The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an iduronate-2-sulfatase (I2S) protein, salt, and a polysorbate surfactant for the treatment of Hunters Syndrome. | 12-29-2011 |
| 20100291060 | SUBCUTANEOUS ADMINISTRATION OF ALPHA-GALACTOSIDASE A - The invention relates, in part, to improved methods of administering α-galactosidase A for the treatment of α-galactosidase A deficiencies including Fabry disease. | 11-18-2010 |
| 20100143314 | In Vivo Production and Delivery of Erythropoietin or Insulinotropin for Gene Therapy - The present invention relates to transfected primary and secondary somatic cells of vertebrate origin, particularly mammalian origin, transfected with exogenous genetic material (DNA) which encodes erythropoietin or an insulinotropin [e.g., derivatives of glucagon-like peptide 1 (GLP-1)], methods by which primary and secondary cells are transfected to include exogenous genetic material encoding erythropoietin or an insulinotropin, methods of producing clonal cell strains or heterogenous cell strains which express erythropoietin or an insulinotropin, methods of gene therapy in which the transfected primary or secondary cells are used, and methods of producing antibodies using the transfected primary or secondary cells. The present invention also includes primary and secondary somatic cells, such as fibroblasts, keratinocytes, epithelial cells, endothelial cells, glial cells, neural cells, formed elements of the blood, muscle cells, other somatic cells, which can be cultured and somatic cell precursors, which have been transfected with exogenous DNA encoding EPO or an insulinotropin, which is stably integrated into their genomes or is expressed in the cells episomally. | 06-10-2010 |
