Patents Assigned to Genetic Therapy, Inc.
  • Peptide linkers for polypeptide compositions and methods for using same
    Patent number: 9206235
    Abstract: Disclosed herein are novel peptide linkers and polypeptide compositions comprising the linkers (e.g., chimeric polypeptides) and methods of using the polypeptide compositions. The compositions and methods are particularly useful for targeting/delivering a polypeptide or protein of interest (e.g., a therapeutic polypeptide) to a cell, tissue or organ of interest in order to treat various diseases or disorders (e.g., lysosomal storage disorders).
    Type: Grant
    Filed: November 12, 2013
    Date of Patent: December 8, 2015
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Paolo Martini, Michael Concino
  • TREATMENT OF COGNITIVE IMPAIRMENT OF HUNTER SYNDROME BY INTRATHECAL DELIVERY OF IDURONATE-2-SULFATASE
    Publication number: 20150328289
    Abstract: The present invention, provides a method of treating cognitive impairment of Hunter syndrome.
    Type: Application
    Filed: December 21, 2012
    Publication date: November 19, 2015
    Applicant: SHIRE HUMAN GENETIC THERAPIES, INC.
    Inventors: Ann BARBIER, Thomas MCCAULEY, Charles W. RICHARD, III
  • CFTR mRNA compositions and related methods and uses
    Patent number: 9181321
    Abstract: Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.
    Type: Grant
    Filed: June 17, 2014
    Date of Patent: November 10, 2015
    Assignees: Shire Human Genetic Therapies, Inc., Ethris GMBH
    Inventors: Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
  • STABILIZED COMPOSITIONS OF PROTEINS HAVING A FREE THIOL MOIETY
    Publication number: 20150283216
    Abstract: Compositions of proteins having free thiols, and methods of making and using such compositions, are described.
    Type: Application
    Filed: June 18, 2015
    Publication date: October 8, 2015
    Applicant: SHIRE HUMAN GENETIC THERAPIES, INC.
    Inventors: Gaozhong ZHU, Vinh NGUYEN, Kris LOWE, Zahra SHAHROKH
  • Stabilized compositions of proteins having a free thiol moiety
    Patent number: 9072785
    Abstract: Compositions of proteins having free thiols, and methods of making and using such compositions, are described.
    Type: Grant
    Filed: September 6, 2013
    Date of Patent: July 7, 2015
    Assignee: SHIRE HUMAN GENETIC THERAPIES, INC.
    Inventors: Gaozhong Zhu, Vinh Nguyen, Kris Lowe, Zahra Shahrokh
  • Competition-based detection assays
    Patent number: 9075057
    Abstract: Disclosed herein are methods and kits which are useful for detecting presence of an enzyme and the relative amount of glycan associated with the enzyme in a test sample based upon the enzyme's ability to competitively inhibit the binding of a ligand in such test sample. The present invention provides the ability to evaluate cell culture conditions and optimize the desired glycoform content of recombinantly prepared enzymes.
    Type: Grant
    Filed: June 8, 2012
    Date of Patent: July 7, 2015
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventor: Daniel S. Roseman
  • mRNA for use in treatment of human genetic diseases
    Patent number: 9061021
    Abstract: Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.
    Type: Grant
    Filed: July 8, 2014
    Date of Patent: June 23, 2015
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Braydon Charles Guild, Michael Heartlein, Frank DeRosa
  • Purification of iduronate-2-sulfatase
    Patent number: 9051556
    Abstract: 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 involving as few as four chromatography columns.
    Type: Grant
    Filed: March 14, 2013
    Date of Patent: June 9, 2015
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventor: Dave Nichols
  • Assays for agents affecting megakaryocyte development
    Patent number: 9017936
    Abstract: Methods and kits for identifying candidate anti-megakaryocyte agents are disclosed.
    Type: Grant
    Filed: October 5, 2010
    Date of Patent: April 28, 2015
    Assignees: Shire Human Genetic Therapies, Inc., University of Wales Institute, Cardiff
    Inventors: Jorge D. Erusalimsky, Maninder Ahluwalia
  • Assays for detection of glycosaminoglycans
    Patent number: 9012168
    Abstract: Disclosed herein are novel methods, assays and kits useful for the diagnosis and monitoring of subjects with mucopolysaccharidoses (MPS), The methods, assays and kits are particularly useful for detecting the presence of one or more glycosaminoglycans which correlate to MPS and its severity in a variety of biological samples.
    Type: Grant
    Filed: July 29, 2011
    Date of Patent: April 21, 2015
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Juan Ruiz, Marcia Sellos-Moura, Philip Shi
  • Crystal structure of human alpha-n-acetylglucosaminidase
    Publication number: 20150031112
    Abstract: The present invention provides the three-dimensional structure of human ?-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 ?-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)).
    Type: Application
    Filed: July 7, 2014
    Publication date: January 29, 2015
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventors: Muthuraman Meiyappan, Michael F. Concino, Angela W. Norton
  • METHODS FOR PREPARATION OF CULTURE MEDIA
    Publication number: 20150010898
    Abstract: Methods for preparation of culture media and production of recombinant proteins described herein.
    Type: Application
    Filed: October 26, 2012
    Publication date: January 8, 2015
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventor: Robin Ng
  • OPTIMIZED MESSENGER RNA
    Publication number: 20140350089
    Abstract: The present invention is directed to a synthetic nucleic acid sequence which encodes a protein wherein at least one non-common codon or less-common codon is replaced by a common codon. The synthetic nucleic acid sequence can include a continuous stretch of at least 90 codons all of which are common codons.
    Type: Application
    Filed: March 3, 2014
    Publication date: November 27, 2014
    Applicant: SHIRE HUMAN GENETIC THERAPIES, INC.
    Inventors: Richard F. Selden, Allan M. Miller, Douglas A. Treco
  • ENZYMATIC ACTIVITY BASED-DETECTION
    Publication number: 20140302531
    Abstract: Disclosed herein are methods and kits which are useful for detecting presence of an enzyme in a test sample based upon the intrinsic enzymatic activity of such test sample. The present invention provides the ability to evaluate cell culture conditions and optimize the desired glycoform content of recombinantly prepared enzymes.
    Type: Application
    Filed: June 9, 2014
    Publication date: October 9, 2014
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventor: Daniel S. Roseman
  • mRNA for use in treatment of human genetic diseases
    Patent number: 8853377
    Abstract: Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification conferring stability to the RNA, as well as related methods, are disclosed.
    Type: Grant
    Filed: November 29, 2011
    Date of Patent: October 7, 2014
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Braydon Charles Guild, Michael Heartlein
  • METHOD OF CHARACTERIZING LYSOSOMAL ENZYMES
    Publication number: 20140294800
    Abstract: The present invention provides, among other things, methods for the characterization of recombinant Heparan N-Sulfatase (HNS) during manufacture. The present invention uses capillary zone electrophoresis to determine the charge profile, isoform distribution, and/or glycan profile of recombinant HNS; and represents a quality feature for the batch consistency, storage stability, biological half-life, pharmacokinetic, pharmacodynamic and biological activity of the enzyme. In particular, such characterization methods may be beneficial to optimize conditions and ensure consistency for the manufacture of HNS for the treatment of a patient diagnosed with Sanfilippo syndrome using enzyme replacement therapy.
    Type: Application
    Filed: March 13, 2014
    Publication date: October 2, 2014
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventor: Daniel Roseman
  • CLEAVABLE LIPIDS
    Publication number: 20140288160
    Abstract: 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 as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.
    Type: Application
    Filed: June 8, 2012
    Publication date: September 25, 2014
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventors: Braydon Charles Guild, Michael Heartlein, Frank DeRosa, Jerry Chi Zhang
  • METHODS AND COMPOSITIONS FOR CNS DELIVERY OF IDURONATE-2-SULFATASE
    Publication number: 20140271598
    Abstract: 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.
    Type: Application
    Filed: August 23, 2013
    Publication date: September 18, 2014
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventors: Gaozhong Zhu, Kris Lowe, Zahra Shahrokh, James Christian, Rick Fahrner, Jing Pan, Teresa Leah Wright, Pericles Calias
  • ANTISENSE OLIGONUCLEOTIDE MODULATORS OF SEROTONIN RECEPTOR 2C AND USES THEREOF
    Publication number: 20140275222
    Abstract: 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 HTR2C pre-mRNA and drive expression of HTR2C Vb splice isoform, leading to increased generation of non-edited strong HTR2C receptor and enhanced serotonin receptor activity.
    Type: Application
    Filed: November 9, 2012
    Publication date: September 18, 2014
    Applicants: Shire Human Genetic Therapies, Inc., University of Kentucky
    Inventors: Stefan Stamm, Manli Shen, Serene Josiah
  • COMPOSITIONS AND METHODS FOR TREATING GAUCHER DISEASE
    Publication number: 20140242061
    Abstract: Methods and compositions for treating Gaucher disease are described.
    Type: Application
    Filed: January 31, 2014
    Publication date: August 28, 2014
    Applicant: SHIRE HUMAN GENETIC THERAPIES, INC.
    Inventors: Peter Francis Daniel, Michael W. Heartlein