SI3205351T1 - Encim za bolezen lizosomskega shranjevanja - Google Patents

Encim za bolezen lizosomskega shranjevanja

Info

Publication number
SI3205351T1
SI3205351T1 SI201132086T SI201132086T SI3205351T1 SI 3205351 T1 SI3205351 T1 SI 3205351T1 SI 201132086 T SI201132086 T SI 201132086T SI 201132086 T SI201132086 T SI 201132086T SI 3205351 T1 SI3205351 T1 SI 3205351T1
Authority
SI
Slovenia
Prior art keywords
lysosomal storage
storage disease
disease enzyme
enzyme
lysosomal
Prior art date
Application number
SI201132086T
Other languages
English (en)
Inventor
Anthony Quinn
Alex Harvey
Original Assignee
Alexion Pharmaceuticals, Inc.,
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Alexion Pharmaceuticals, Inc., filed Critical Alexion Pharmaceuticals, Inc.,
Publication of SI3205351T1 publication Critical patent/SI3205351T1/sl

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18—Carboxylic ester hydrolases (3.1.1)
    • C12N9/20—Triglyceride splitting, e.g. by means of lipase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/194—Carboxylic acids, e.g. valproic acid having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/38—Albumins
    • A61K38/385—Serum albumin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01013—Sterol esterase (3.1.1.13)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/30—Bird
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/01—Animal expressing industrially exogenous proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/11011—Alpharetrovirus, e.g. avian leucosis virus
    • C12N2740/11041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/11043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/11011—Alpharetrovirus, e.g. avian leucosis virus
    • C12N2740/11041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/11045—Special targeting system for viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011—Details
    • C12N2760/20011—Rhabdoviridae
    • C12N2760/20211—Vesiculovirus, e.g. vesicular stomatitis Indiana virus
    • C12N2760/20222—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Environmental Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Cardiology (AREA)
  • Biophysics (AREA)
SI201132086T 2010-04-23 2011-04-23 Encim za bolezen lizosomskega shranjevanja SI3205351T1 (sl)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US34317710P 2010-04-23 2010-04-23
US39637610P 2010-05-26 2010-05-26
US40301110P 2010-09-09 2010-09-09
US45601410P 2010-10-29 2010-10-29
US201161432372P 2011-01-13 2011-01-13
EP17158770.2A EP3205351B1 (en) 2010-04-23 2011-04-23 Lysosomal storage disease enzyme

Publications (1)

Publication Number Publication Date
SI3205351T1 true SI3205351T1 (sl) 2023-09-29

Family

ID=44834852

Family Applications (2)

Application Number Title Priority Date Filing Date
SI201132086T SI3205351T1 (sl) 2010-04-23 2011-04-23 Encim za bolezen lizosomskega shranjevanja
SI201131204A SI2561069T1 (sl) 2010-04-23 2011-04-23 Encim za bolezen lizosomskega shranjevanja

Family Applications After (1)

Application Number Title Priority Date Filing Date
SI201131204A SI2561069T1 (sl) 2010-04-23 2011-04-23 Encim za bolezen lizosomskega shranjevanja

Country Status (21)

Country Link
US (4) US20130209436A1 (sl)
EP (3) EP3205351B1 (sl)
JP (4) JP5925187B2 (sl)
KR (1) KR101742346B1 (sl)
CN (2) CN105214076B (sl)
AU (1) AU2011242461B2 (sl)
BR (1) BR112012027143B1 (sl)
CA (2) CA2995446C (sl)
DK (2) DK3205351T3 (sl)
ES (2) ES2627535T3 (sl)
FI (1) FI3205351T3 (sl)
HK (2) HK1183059A1 (sl)
HU (2) HUE033217T2 (sl)
IL (2) IL222513A0 (sl)
LT (1) LT3205351T (sl)
MX (2) MX362430B (sl)
PL (2) PL2561069T3 (sl)
PT (2) PT3205351T (sl)
RU (2) RU2015151189A (sl)
SI (2) SI3205351T1 (sl)
WO (1) WO2011133960A2 (sl)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2003523330A (ja) 2000-02-04 2003-08-05 チルドレンズ・ホスピタル・リサーチ・ファウンデイション アテローム性動脈硬化症および関連疾患のための脂質加水分解治療
US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases
KR101471445B1 (ko) * 2007-01-26 2014-12-15 시나게바 바이오파르마, 코포레이션 조류에서 이식유전자 발현
EP3205351B1 (en) 2010-04-23 2023-04-12 Alexion Pharmaceuticals, Inc. Lysosomal storage disease enzyme
NZ700824A (en) 2010-09-09 2016-03-31 Synageva Biopharma Corp Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
EP2675472A4 (en) * 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
EP2820032A4 (en) * 2012-03-02 2015-11-25 Synageva Biopharma Corp TRUNCATED LYSOSOMAL ACID LIPASE
US9938319B2 (en) * 2014-04-23 2018-04-10 Alexion Pharmaceuticals, Inc. Egg white processing
US11235036B2 (en) 2016-06-17 2022-02-01 Alexion Pharmaceuticals, Inc. Lysosomal acid lipase deficiency compositions and methods
EP3504222A1 (en) * 2016-08-23 2019-07-03 Alexion Pharmaceuticals, Inc. Method of purifying a heterologous protein from an egg white
WO2018064303A1 (en) 2016-09-30 2018-04-05 Alexion Pharmaceuticals, Inc. Methods for reducing liver fibrosis and treating lysosomal acid lipase deficiency in patients based on ishak fibrosis stage
US10633705B2 (en) 2016-10-14 2020-04-28 Alexion Pharmaceuticals, Inc. N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods
WO2019152343A1 (en) 2018-02-01 2019-08-08 Alexion Pharmaceuticals, Inc. Methods of desensitizing against infusion associated reactions in lysosomal acid lipase deficiency (lal-d) patients treated with exogenous lysosomal acid lipase (lal
CN114616000A (zh) * 2019-07-02 2022-06-10 M6P医疗(瑞士)有限责任公司 载体组合物及其用于治疗溶酶体贮积症的方法
CN114369620B (zh) * 2021-12-31 2024-05-24 广州博垚生物科技有限公司 一种可持续分泌lal的载体、脐带间充质干细胞及其构建方法和应用

Family Cites Families (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE9001985D0 (sv) 1990-06-01 1990-06-01 Astra Ab New chemical products
AU1580292A (en) 1991-03-13 1992-10-21 United States of America, as represented by the Secretary, U.S. Department of Commerce, The Increasing the therapeutic efficiency of macrophage-targeted therapeutic agents by up-regulating the mannose lectin on macrophages
US5631365A (en) 1993-09-21 1997-05-20 Schering Corporation Hydroxy-substituted azetidinone compounds useful as hypocholesterolemic agents
EP0952841A4 (en) 1994-08-05 2000-11-02 Molecular Structural Biotechno DIRECTED BIOMOLECULAR COMPLEXES
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
EP0865499B1 (en) 1995-09-14 2009-03-18 Virginia Tech Intellectual Properties, Inc. Production of lysosomal enzymes in plant-based expression systems
EP0935651B1 (en) 1996-09-13 2004-12-29 Transkaryotic Therapies, Inc. THERAPY FOR alpha-GALACTOSIDASE A DEFICIENCY
US5897998A (en) 1997-08-04 1999-04-27 The University Of Georgia Research Foundation, Inc. Method for manipulating avian eggs
DE69835367T2 (de) 1997-10-29 2007-08-02 Genzyme Corp., Framingham Gentherapie für gaucher-krankheit
US20030166132A1 (en) 1998-08-26 2003-09-04 Genentech, Inc. Secreted and transmembrane polypeptides and nucleic acids encoding the same
ES2677343T3 (es) 1998-12-07 2018-08-01 Genzyme Corporation Tratamiento de la enfermedad de Pompe
US7033780B1 (en) 1999-06-14 2006-04-25 Millennium Pharmaceuticals, Inc. Nucleic acids corresponding to TANGO 294 a gene encoding a lipase—like protein
US7927587B2 (en) 1999-08-05 2011-04-19 Regents Of The University Of Minnesota MAPC administration for the treatment of lysosomal storage disorders
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
JP2003523330A (ja) 2000-02-04 2003-08-05 チルドレンズ・ホスピタル・リサーチ・ファウンデイション アテローム性動脈硬化症および関連疾患のための脂質加水分解治療
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20040038365A1 (en) 2000-10-31 2004-02-26 Yonghong Xiao Regulation of human lysosomal acid lipase
US20030064437A1 (en) 2000-11-15 2003-04-03 Nick Wan Expression system for recombinant proteins
US20020193303A1 (en) 2001-01-25 2002-12-19 Millennium Pharmaceuticals, Inc. 58860, a human cholesteryl ester hydrolase and uses therefor
US6387680B1 (en) * 2001-03-29 2002-05-14 Pe Corporation (Ny) Isolated human lipase proteins, nucleic acid molecules encoding human lipase proteins, and uses thereof
US6800472B2 (en) 2001-12-21 2004-10-05 Genzyme Glycobiology Research Institute, Inc. Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase
WO2003064614A2 (en) 2002-01-30 2003-08-07 Yale University Transport peptides and uses therefor
ES2498369T3 (es) 2002-04-16 2014-09-24 Vlaams Interuniversitair Instituut Voor Biotechnologie Vzw. Un marcador para medir cirrosis hepática
AU2003283976B2 (en) 2002-09-27 2009-12-10 Cold Spring Harbor Laboratory Cell-based RNA interference and related methods and compositions
US20050208090A1 (en) 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
WO2006002053A2 (en) 2004-06-15 2006-01-05 Mayo Foundation For Medical Education And Research Helicases
NZ553420A (en) 2004-08-19 2010-02-26 Biogen Idec Inc Refolding transforming growth factor beta family proteins
US20070270367A1 (en) 2005-05-18 2007-11-22 University Of Kentucky Research Foundation Rybozyme-catalyzed insertion of targeted sequences into RNA
US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases
PL377180A1 (pl) 2005-09-21 2007-04-02 Instytut Farmaceutyczny Zastosowanie izoflawonów i ich pochodnych w leczeniu mukopolisacharydoz
CA2622074C (en) 2005-10-05 2013-01-22 Avigenics, Inc. Rapid production of high titer virus
US20070092486A1 (en) 2005-10-21 2007-04-26 Avigenics, Inc. Glycolated and glycosylated poultry derived therapeutic proteins
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
US8748567B2 (en) 2006-05-22 2014-06-10 Children's Medical Center Corporation Method for delivery across the blood brain barrier
WO2007137303A2 (en) 2006-05-24 2007-11-29 Myelin Repair Foundation, Inc. Permeability of blood-brain barrier
KR101471445B1 (ko) 2007-01-26 2014-12-15 시나게바 바이오파르마, 코포레이션 조류에서 이식유전자 발현
US8143265B2 (en) 2007-04-16 2012-03-27 Meharry Medical College Method of treating atherosclerosis
WO2008131431A2 (en) 2007-04-23 2008-10-30 St. Louis University Modulation of blood brain barrier protein expression
US8956825B2 (en) 2007-05-24 2015-02-17 The United States Of America As Represented By The Department Of Veterans Affairs Intranuclear protein transduction through a nucleoside salvage pathway
AU2008255565B2 (en) 2007-05-31 2013-10-24 Paradigm Biopharmaceuticals Ltd Sulphated xylans for treatment or prophylaxis of respiratory diseases
CN101790385A (zh) 2007-07-12 2010-07-28 普罗森那技术公司 用于使化合物靶向多种选定器官、组织或肿瘤细胞的分子
WO2009032171A1 (en) 2007-08-29 2009-03-12 Shire Human Genetic Therapies, Inc. Subcutaneous administration of alpha-galatosidase a
NZ585555A (en) * 2008-01-07 2012-08-31 Synageva Biopharma Corp Transgenic chicken containing a transgene comprising a glycosyltransferase coding sequence
WO2010033854A2 (en) 2008-09-19 2010-03-25 Synageva Biopharma Corp. Avian derived fusion proteins
FR2937322B1 (fr) 2008-10-22 2013-02-22 Vect Horus Derives peptidiques et leur utilisation comme vecteurs de molecules sous forme de conjugues
US8232073B2 (en) 2009-01-02 2012-07-31 Zacharon Pharmaceuticals, Inc. Quantification of non-reducing end glycan residual compounds
WO2010078515A2 (en) 2009-01-02 2010-07-08 Zacharon Pharmaceuticals, Inc. Polymer end group detection
US8257694B2 (en) 2009-05-14 2012-09-04 Product Partners, Llc Nutritional compositions for reducing oxidative damage
EP2448600B1 (en) 2009-07-03 2016-03-30 INSERM - Institut National de la Santé et de la Recherche Médicale Compounds targeting the cation-independent mannose 6-phosphate receptor
CA2778349C (en) 2009-10-19 2018-12-04 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
KR101077618B1 (ko) 2009-11-13 2011-10-27 경북대학교 산학협력단 뇌혈관 장벽을 통과하여 퇴행성 뇌신경 질환 부위의 세포사멸을 표적하는 펩타이드 및 이의 용도
US9095541B2 (en) 2009-11-24 2015-08-04 Arch Cancer Therapeutics, Inc. Brain tumor targeting peptides and methods
EP3205351B1 (en) * 2010-04-23 2023-04-12 Alexion Pharmaceuticals, Inc. Lysosomal storage disease enzyme
US20150030582A1 (en) * 2010-04-23 2015-01-29 Synageva Biopharma Corp. Lysosomal Storage Disease Enzyme
NZ700824A (en) 2010-09-09 2016-03-31 Synageva Biopharma Corp Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
EP2675472A4 (en) * 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
WO2012112677A2 (en) 2011-02-15 2012-08-23 Children's Hospital Medical Center Methods for treating lysosomal acid lipase deficiency
CN103492574B (zh) 2011-02-22 2015-12-09 加州理工学院 使用腺相关病毒（aav）载体递送蛋白
EP2709670A4 (en) 2011-05-18 2015-01-21 Childrens Hosp Medical Center TARGETED ADMINISTRATION OF PROTEINS ACROSS THE HEMATO-ENCEPHALIC BARRIER
EP2721151B1 (en) 2011-06-20 2017-12-06 Mount Sinai School Of Medicine Anti-tnf-therapy for the mucopolysaccharidoses and other lysosomal disorders
WO2012177639A2 (en) 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
AU2012289916B2 (en) 2011-08-03 2017-08-03 Phoenix Tissue Repair, Inc. Collagen 7 and related methods

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