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Shire Human Genetic Therapies Inc
Shire Human Genetic Therapies Inc A Delaware Corporation

Shire Human Genetic Therapies Inc patents


Recent patent applications related to Shire Human Genetic Therapies Inc. Shire Human Genetic Therapies Inc is listed as an Agent/Assignee. Note: Shire Human Genetic Therapies Inc may have other listings under different names/spellings. We're not affiliated with Shire Human Genetic Therapies Inc, we're just tracking patents.

ARCHIVE: New 2018 2017 2016 2015 2014 2013 2012 2011 2010 2009 | Company Directory "S" | Shire Human Genetic Therapies Inc-related inventors


Stabilized compositions of proteins having a free thiol moiety

Compositions of proteins having free thiols, and methods of making and using such compositions, are described.... Shire Human Genetic Therapies Inc

Anti-flt-1 antibodies in treating bronchopulmonary dysplasia

The present invention provides, among other things, methods and compositions for treating chronic lung disorders, in particular, bronchopulmonary dysplasia (BPD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to BPD an effective amount of an anti-Flt-1 antibody,... Shire Human Genetic Therapies Inc

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... Shire Human Genetic Therapies Inc

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... Shire Human Genetic Therapies Inc

Treatment of cognitive impairment of hunter syndrome by intrathecal delivery of iduronate-2-sulfatase

The present invention, provides a method of treating cognitive impairment of Hunter syndrome. Among other things, the present invention provides a method comprising a step of administering intrathecally to a subject in need of treatment a recombinant iduronate-2-sulfatase (I2S) enzyme at a therapeutically effective dose and an administration interval for... Shire Human Genetic Therapies Inc

Anti-flt-1 antibodies in treating bronchopulmonary dysplasia

The present invention provides, among other things, methods and compositions for treating chronic lung disorders, in particular, bronchopulmonary dysplasia (BPD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to BPD an effective amount of an anti-Flt-1 antibody,... Shire Human Genetic Therapies Inc

Anti-flt-1 antibodies for treating duchenne muscular dystrophy

The present invention provides, among other things, anti-Flt-1 antibodies and methods for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of an anti-Flt-1... Shire Human Genetic Therapies Inc

Enzymatic activity assays for glucocerebrosidase

The present invention provides, among other things, methods and compositions for determining enzyme kinetic parameters (e.g., Vmax, Km, and specific activity, etc.) indicative of clinically relevant properties of glucocerebrosidase using a physiologically relevant substrate, in particular, a substrate that is representative of substrates that typically accumulate in patients suffering from... Shire Human Genetic Therapies Inc

Methods and compositions for intrathecally administered treatment of mucupolysaccharidosis type iiia

The present invention provides, among other things, effective treatment for Sanfilippo Syndrome Type A (MPS IDA) based on intrathecal delivery of recombinant heparin N-Sulfatase (HNS) enzyme. In some embodiments, the present invention includes methods of treating Sanfilippo Syndrome Type A (MPS MA) Syndrome by intrathecal administration of a recombinant HNS... Shire Human Genetic Therapies Inc

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... Shire Human Genetic Therapies Inc

Cells for producing recombinant iduronate-2-sulfatase

The present invention provides, among other things, methods and compositions for production of recombinant I2S protein with improved potency and activity using cells co-express I2S and FGE protein. In some embodiments, cells according to the present invention are engineered to simultaneously over-express recombinant I2S and FGE proteins. Cells according to... Shire Human Genetic Therapies Inc

Conjugated c1 esterase inhibitor and uses thereof

The present invention provides, among other things, a conjugated C1-INH for improved treatment of complement-mediated disorders, including hereditary angioedema (HAE). In some embodiments, a conjugated C1-INH provided by the present invention is a PEGylated C1-INH. In some embodiments, a conjugated C1-INH provided by the present invention is a polysialic acid... Shire Human Genetic Therapies Inc

Enzymatic activity assays for i2s

In certain embodiments of the present invention, kinetic parameters of I2S enzyme are determined. In some instances, a sample including I2S enzyme is incubated under defined conditions, with a series of determined amounts of I2S substrate including a detectable label. Following incubation, the reaction mixture can be analyzed, e.g., by... Shire Human Genetic Therapies Inc

Crystal structure of human four-phosphate adaptor protein 2 glycolipid transfer protein like domain

In some embodiments, the present invention provides method of identifying compounds that bind to phosphoinositol 4-phosphate adaptor protein-2 (FAPP2), including the steps of computationally identifying a compound that binds to FAPP2 using the atomic coordinates of at least the amino acids which make up the substrate binding pocket of FAPP2.... Shire Human Genetic Therapies Inc

Determination of glycosaminoglycan levels by mass spectrometry

Detecting glycosaminoglycans (GAGs) and/or determining the level of one or more glycosaminoglycans can be useful, e.g., in identifying or monitoring various medical conditions, the status of patients having various medical conditions, and/or the response to treatment of individuals having various medical conditions. The present invention provides methods for detecting glycosaminoglycans... Shire Human Genetic Therapies Inc

Lysosomal targeting of enzymes, 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... Shire Human Genetic Therapies Inc

Antisense oligonucleotide modulators of serotonin receptor 2c and uses thereof

The present invention provides, among other things, oligonucleotide modulators of human 5′-HT2C receptor (HTR2C) and improved methods and composition for treating HTR2C-related diseases, disorders or conditions based on such modulators. In particular, oligonucleotides modulators according to the invention target specific regions in the Exon V/Intron V junction of the human... Shire Human Genetic Therapies Inc

Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase

A method of producing mannose-6-phosphate (M6P)-containing recombinant alpha-N-acetyl-glucosaminidase (Naglu), including the steps of providing a high mannose containing recombinant Naglu protein; and contacting the high mannose containing recombinant Naglu protein with N-acetyl-glucosamine-1-phosphotransferase (GNPT) under conditions that permit phosphorylation of one or more mannose residues on the recombinant Naglu protein, thereby... Shire Human Genetic Therapies Inc

Lysosomal targeting and uses thereof

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.... Shire Human Genetic Therapies Inc

Biodegradable lipids for delivery of nucleic acids

The present invention provides, in part, a biodegradable compound of formula I, and sub-formulas thereof: Formula (I) or a pharmaceutically acceptable salt thereof, where each X independently is O or S, each Y independently is O or S, and each R1 independently is defined herein; and a liposome composition comprising... Shire Human Genetic Therapies Inc

Ionizable cationic lipids

Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized... Shire Human Genetic Therapies Inc

Mannose-6-phosphate bearing peptides fused to lysosomal enzymes

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... Shire Human Genetic Therapies Inc

Treatment of alpha-galactosidase a deficiency

The invention provides methods of treating α-galactosidase A deficiency. Dosage forms, methods of administration, and methods of analyzing human α-galactosidase A are also included.... Shire Human Genetic Therapies Inc

Substrate clearance assays for lysosomal enzymes

The present invention provides, among other things, improved substrate clearance assays for lysosomal enzyme that are particularly useful for measuring potency of lysosomal enzymes or other therapeutics for treatment of lysosomal storage diseases. In particular, the present invention combines a physiologically relevant substrate cell assay and an efficient scintillation based... Shire Human Genetic Therapies Inc

Lipoprotein lipase for treatment of hypertriglyceridemic-related conditions including acute pancreatitis

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... Shire Human Genetic Therapies Inc

Modification of rna-related enzymes for enhanced production

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

Mrna therapy for the treatment of ocular diseases

The present invention provides, among other things, a method of ocular delivery of messenger RNA (mRNA), comprising administering into an eye of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administration of the composition results in expression of the protein encoded... Shire Human Genetic Therapies Inc

Biomarkers for sanfilippo syndrome and uses thereof

The present invention provides biomarkers for efficient and accurate characterization of Sanfilippo syndrome. In particular, the present invention provides biomarkers differentially expressed in Sanfilippo syndrome. Those biomarkers, used alone or in combination, may permit more accurate robust characterization of Sanfilippo syndrome, resulting in more precise determination of the types and/or... Shire Human Genetic Therapies Inc

Mrna therapy for phenylketonuria

The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced... Shire Human Genetic Therapies Inc

Purification of iduronate-2-sulfatase

The present invention provides, among other things, improved methods for purifying I2S protein produced recombinantly for enzyme replacement therapy. The present invention is, in part, based on the surprising discovery that recombinant I2S protein can be purified from unprocessed biological materials, such as, I2S-containing cell culture medium, using a process... Shire Human Genetic Therapies Inc

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... Shire Human Genetic Therapies Inc

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... Shire Human Genetic Therapies Inc








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