KR101742346B1 - 리소솜 축적병 효소 - Google Patents

리소솜 축적병 효소 Download PDF

Info

Publication number
KR101742346B1
KR101742346B1 KR1020127030540A KR20127030540A KR101742346B1 KR 101742346 B1 KR101742346 B1 KR 101742346B1 KR 1020127030540 A KR1020127030540 A KR 1020127030540A KR 20127030540 A KR20127030540 A KR 20127030540A KR 101742346 B1 KR101742346 B1 KR 101742346B1
Authority
KR
South Korea
Prior art keywords
asn
lal
pharmaceutical composition
seq
leu
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
KR1020127030540A
Other languages
English (en)
Korean (ko)
Other versions
KR20130113312A (ko
Inventor
안토니 퀸
알렉스 제이. 하베이
Original Assignee
시나게바 바이오파르마, 코포레이션
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 시나게바 바이오파르마, 코포레이션 filed Critical 시나게바 바이오파르마, 코포레이션
Publication of KR20130113312A publication Critical patent/KR20130113312A/ko
Application granted granted Critical
Publication of KR101742346B1 publication Critical patent/KR101742346B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/18Carboxylic ester hydrolases (3.1.1)
    • C12N9/20Triglyceride splitting, e.g. by means of lipase
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278Knock-in vertebrates, e.g. humanised vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/185Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19Carboxylic acids, e.g. valproic acid
    • A61K31/194Carboxylic acids, e.g. valproic acid having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/38Albumins
    • A61K38/385Serum albumin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/04Antineoplastic agents specific for metastasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/01Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01013Sterol esterase (3.1.1.13)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/30Bird
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/01Animal expressing industrially exogenous proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/10041Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/11011Alpharetrovirus, e.g. avian leucosis virus
    • C12N2740/11041Use of virus, viral particle or viral elements as a vector
    • C12N2740/11043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/11011Alpharetrovirus, e.g. avian leucosis virus
    • C12N2740/11041Use of virus, viral particle or viral elements as a vector
    • C12N2740/11045Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2760/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
    • C12N2760/00011Details
    • C12N2760/20011Rhabdoviridae
    • C12N2760/20211Vesiculovirus, e.g. vesicular stomatitis Indiana virus
    • C12N2760/20222New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

Landscapes

  • 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)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (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)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Cardiology (AREA)
KR1020127030540A 2010-04-23 2011-04-23 리소솜 축적병 효소 Active KR101742346B1 (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US34317710P 2010-04-23 2010-04-23
US61/343,177 2010-04-23
US39637610P 2010-05-26 2010-05-26
US61/396,376 2010-05-26
US40301110P 2010-09-09 2010-09-09
US61/403,011 2010-09-09
US45601410P 2010-10-29 2010-10-29
US61/456,014 2010-10-29
US201161432372P 2011-01-13 2011-01-13
US61/432,372 2011-01-13
PCT/US2011/033699 WO2011133960A2 (en) 2010-04-23 2011-04-23 Lysosomal storage disease enzyme

Publications (2)

Publication Number Publication Date
KR20130113312A KR20130113312A (ko) 2013-10-15
KR101742346B1 true KR101742346B1 (ko) 2017-06-15

Family

ID=44834852

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020127030540A Active KR101742346B1 (ko) 2010-04-23 2011-04-23 리소솜 축적병 효소

Country Status (21)

Country Link
US (4) US20130209436A1 (https=)
EP (3) EP4241854A3 (https=)
JP (4) JP5925187B2 (https=)
KR (1) KR101742346B1 (https=)
CN (2) CN102985538B (https=)
AU (1) AU2011242461B2 (https=)
BR (1) BR112012027143B1 (https=)
CA (2) CA2796607C (https=)
DK (2) DK2561069T3 (https=)
ES (2) ES2627535T3 (https=)
FI (1) FI3205351T3 (https=)
HK (1) HK1219655A1 (https=)
HU (2) HUE062163T2 (https=)
IL (2) IL222513A0 (https=)
LT (1) LT3205351T (https=)
MX (2) MX338426B (https=)
PL (2) PL2561069T3 (https=)
PT (2) PT2561069T (https=)
RU (2) RU2015151189A (https=)
SI (2) SI2561069T1 (https=)
WO (1) WO2011133960A2 (https=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2328446T5 (es) 2000-02-04 2014-02-27 Children's Hospital Research Foundation Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas
WO2007030375A2 (en) * 2005-09-08 2007-03-15 Children's Hospital Medical Center Lysosomal acid lipase therapy for nafld and related diseases
AU2007345347B2 (en) * 2007-01-26 2013-11-07 Synageva Biopharma Corp Transgene expression in avians
PT2561069T (pt) 2010-04-23 2017-05-22 Alexion Pharma Inc Enzima de doença de armazenamento lisossomal
CA2810999C (en) 2010-09-09 2023-10-03 Anthony Quinn 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
WO2013131014A1 (en) * 2012-03-02 2013-09-06 Synageva Biopharma Corp. Truncated lysosomal acid lipase
BR112016024586B1 (pt) * 2014-04-23 2022-03-29 Alexion Pharmaceuticals, Inc Método de preparação de clara de ovo para o processamento cromatográfico em massa
US11235036B2 (en) 2016-06-17 2022-02-01 Alexion Pharmaceuticals, Inc. Lysosomal acid lipase deficiency compositions and methods
JP7027347B2 (ja) * 2016-08-23 2022-03-01 アレクシオン ファーマシューティカルズ インコーポレイテッド 卵白から異種タンパク質を精製する方法
ES2888126T3 (es) 2016-09-30 2021-12-30 Alexion Pharma Inc Sebelipasa alfa para su uso en fibrosis hepática y para tratar la deficiencia de lipasa ácida lisosomal en pacientes según el estadio de fibrosis de Ishak
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
US12492412B2 (en) * 2019-07-02 2025-12-09 M6P Terapeutics (Switzerland) Llc Vector compositions and methods of using same for treatment of lysosomal storage disorders
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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20090178147A1 (en) 2008-01-07 2009-07-09 Synageva Biopharma Corp. Glycosylation in avians
US20100062982A1 (en) 2005-06-24 2010-03-11 Harvey Alex J Avian derived fusion proteins

Family Cites Families (60)

* 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
AU3275595A (en) 1994-08-05 1996-03-04 Molecular/Structural Biotechnologies, Inc. Site-specific biomolecular complexes
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
DE69637875D1 (de) 1995-09-14 2009-04-30 Virginia Tech Intell Prop Produktion von lysosomalen enzymen in pflanzlichen expressionssystemen
JP4001925B2 (ja) 1996-09-13 2007-10-31 トランスカーヨティック セラピーズ インク. α―ガラクトシダーゼA欠損症の治療
US5897998A (en) 1997-08-04 1999-04-27 The University Of Georgia Research Foundation, Inc. Method for manipulating avian eggs
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
US20030166132A1 (en) 1998-08-26 2003-09-04 Genentech, Inc. Secreted and transmembrane polypeptides and nucleic acids encoding the same
DK1137762T3 (da) 1998-12-07 2009-02-02 Genzyme Corp Behandling af Pompes sygdom
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
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
ES2328446T5 (es) 2000-02-04 2014-02-27 Children's Hospital Research Foundation Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas
WO2001097829A2 (en) 2000-06-19 2001-12-27 Genzyme Corporation 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
AU2003207744A1 (en) 2002-01-30 2003-09-02 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
EP1546397A4 (en) 2002-09-27 2007-10-31 Cold Spring Harbor Lab CELL BASED RNA INTERFERENCE AND ASSOCIATED 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
WO2007030375A2 (en) 2005-09-08 2007-03-15 Children's 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
US7524626B2 (en) 2005-10-05 2009-04-28 Synageva Biopharma Corp. Rapid production of high titer virus
AU2006304856A1 (en) 2005-10-21 2007-04-26 Synageva Biopharma Corp. 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
WO2008054544A2 (en) 2006-05-22 2008-05-08 Immune Disease Institute, Inc. 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
AU2007345347B2 (en) 2007-01-26 2013-11-07 Synageva Biopharma Corp Transgene expression in avians
US8143265B2 (en) 2007-04-16 2012-03-27 Meharry Medical College Method of treating atherosclerosis
US8466118B2 (en) 2007-04-23 2013-06-18 Saint Louis University Modulation of blood brain barrier protein expression
EP3486653A1 (en) 2007-05-24 2019-05-22 The United States Government as represented by The Department of Veterans Affairs Treatment of skeletal muscle disorder using ent2
AU2008255565B2 (en) 2007-05-31 2013-10-24 Paradigm Biopharmaceuticals Ltd Sulphated xylans for treatment or prophylaxis of respiratory diseases
CA2693742A1 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
EP2185187A1 (en) 2007-08-29 2010-05-19 Shire Human Genetic Therapies, Inc. Subcutaneous administration of alpha-galatosidase a
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
ES2576853T3 (es) 2009-07-03 2016-07-11 Inserm - Institut National De La Santé Et De La Recherche Médicale Compuestos dirigidos al receptor del manosa 6-fosfato independiente de cationes
WO2011049737A1 (en) 2009-10-19 2011-04-28 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
PT2561069T (pt) * 2010-04-23 2017-05-22 Alexion Pharma Inc Enzima de doença de armazenamento lisossomal
US20150030582A1 (en) * 2010-04-23 2015-01-29 Synageva Biopharma Corp. Lysosomal Storage Disease Enzyme
CA2810999C (en) * 2010-09-09 2023-10-03 Anthony Quinn Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
WO2012112677A2 (en) 2011-02-15 2012-08-23 Children's Hospital Medical Center Methods for treating lysosomal acid lipase deficiency
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
US8865881B2 (en) 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
EP2709670A4 (en) 2011-05-18 2015-01-21 Childrens Hosp Medical Center TARGETED TRANSFER OF PROTEINS OVER THE BLOOD BRAIN BARRIER
WO2012177778A1 (en) 2011-06-20 2012-12-27 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
WO2013020064A1 (en) 2011-08-03 2013-02-07 Lotus Tissue Repair Inc. Collagen 7 and related methods

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100062982A1 (en) 2005-06-24 2010-03-11 Harvey Alex J Avian derived fusion proteins
US20090178147A1 (en) 2008-01-07 2009-07-09 Synageva Biopharma Corp. Glycosylation in avians

Also Published As

Publication number Publication date
IL252115A0 (en) 2017-07-31
US11560554B2 (en) 2023-01-24
PL2561069T3 (pl) 2017-08-31
EP3205351A1 (en) 2017-08-16
JP2018100289A (ja) 2018-06-28
ES2950358T3 (es) 2023-10-09
US20200063113A1 (en) 2020-02-27
SI3205351T1 (sl) 2023-09-29
WO2011133960A9 (en) 2013-06-13
JP2015204842A (ja) 2015-11-19
LT3205351T (lt) 2023-07-25
WO2011133960A3 (en) 2012-07-12
MX362430B (es) 2019-01-17
EP3205351B1 (en) 2023-04-12
IL222513A0 (en) 2012-12-31
AU2011242461B2 (en) 2015-10-01
EP2561069A4 (en) 2014-03-26
US10407671B2 (en) 2019-09-10
PL3205351T3 (pl) 2023-08-28
BR112012027143B1 (pt) 2020-02-11
CA2995446A1 (en) 2011-10-27
RU2017105260A (ru) 2018-08-17
HUE033217T2 (hu) 2017-11-28
CN102985538B (zh) 2015-09-09
IL252115B (en) 2019-11-28
JP6212081B2 (ja) 2017-10-11
HK1219655A1 (zh) 2017-04-13
JP5925187B2 (ja) 2016-05-25
PT3205351T (pt) 2023-06-28
EP2561069B1 (en) 2017-03-08
RU2741116C2 (ru) 2021-01-22
HK1183059A1 (en) 2013-12-13
CN105214076B (zh) 2020-03-31
RU2012149936A (ru) 2014-05-27
US20170314000A1 (en) 2017-11-02
SI2561069T1 (sl) 2017-07-31
KR20130113312A (ko) 2013-10-15
HUE062163T2 (hu) 2023-09-28
JP2013526856A (ja) 2013-06-27
MX338426B (es) 2016-04-15
EP4241854A3 (en) 2023-11-08
AU2011242461A1 (en) 2012-11-15
RU2017105260A3 (https=) 2020-06-15
RU2015151189A (ru) 2019-01-14
US20210171922A1 (en) 2021-06-10
US20130209436A1 (en) 2013-08-15
BR112012027143A2 (pt) 2016-11-16
MX2012012356A (es) 2013-04-11
FI3205351T3 (fi) 2023-07-06
WO2011133960A2 (en) 2011-10-27
CN102985538A (zh) 2013-03-20
CA2995446C (en) 2020-05-12
US10858638B2 (en) 2020-12-08
CA2796607A1 (en) 2011-10-27
CN105214076A (zh) 2016-01-06
PT2561069T (pt) 2017-05-22
ES2627535T3 (es) 2017-07-28
JP6496690B2 (ja) 2019-04-03
JP2016190867A (ja) 2016-11-10
EP4241854A2 (en) 2023-09-13
EP2561069A2 (en) 2013-02-27
DK2561069T3 (en) 2017-05-01
DK3205351T3 (da) 2023-06-19
CA2796607C (en) 2019-09-10
BR112012027143A8 (pt) 2017-09-19

Similar Documents

Publication Publication Date Title
KR101742346B1 (ko) 리소솜 축적병 효소
CN108588071A (zh) CRISPR-Cas9靶向敲除人肠癌细胞CNR1基因及其特异性的sgRNA
CN111372943B (zh) 腺病毒及其用途
CN108642053A (zh) CRISPR-Cas9靶向敲除人肠癌细胞PPP1R1C基因及其特异性的sgRNA
CN107893075A (zh) CRISPR‑Cas9靶向敲除人肠癌细胞RITA基因及其特异性的sgRNA
KR100994610B1 (ko) 변형된 성장 호르몬
AU2023226754A1 (en) Compositions and methods for modifying genomes
DK2443248T3 (en) IMPROVEMENT OF LONG-CHAIN POLYUM Saturated OMEGA-3 AND OMEGA-6 FATTY ACID BIOS SYNTHESIS BY EXPRESSION OF ACYL-CoA LYSOPHOSPHOLIPID ACYL TRANSFERASES
KR101828828B1 (ko) 항-히알루로난 제제 치료를 위한 동반 진단 및 이의 사용 방법
CN110914439B (zh) 自灭活病毒载体
CN107090441A (zh) 用于获得高产量重组蛋白表达的基于mgmt的方法
CN108779480A (zh) 生产鞘氨醇碱和鞘脂类的方法
CN101595220A (zh) 减毒重组体新城疫病毒及包含该病毒的疫苗
CN114645066B (zh) 一种用于艾滋病基因治疗的核酸构建体
CN113637672A (zh) 一种碱基编辑工具及其构建方法
KR100862049B1 (ko) 약독화된 재조합 뉴캐슬병 바이러스
DK2475679T3 (da) Forbedrede, optimerede stammer af yarrowia lipolytica til fremstilling af højkoncentreret eicosapentaensyre
CN116135974B (zh) 一种重组糖基化酶碱基编辑系统及其应用
RU2831176C2 (ru) Фермент лизосомальной болезни накопления
US20130090374A1 (en) Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis
CN113846124A (zh) 一种用于糖代谢相关疾病基因治疗的核酸构建体
CN112029798B (zh) 一种在胰腺腺泡细胞特异表达pik3c3 s282a的小鼠模型的构建方法
KR102065917B1 (ko) Glb1 유전자를 발현하는 형질전환된 줄기세포 및 이를 포함하는 gm1 강글리오시드증 치료용 약학 조성물
CN114015722B (zh) 一种构建重症免疫缺陷和肝损伤双重猪模型的方法及应用
KR102315601B1 (ko) 재조합 벡터 및 이를 이용한 재조합 섬유아세포 성장인자 19의 제조 방법

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20121122

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20150720

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20160718

Patent event code: PE09021S01D

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20170227

GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20170525

Patent event code: PR07011E01D

PR1002 Payment of registration fee

Payment date: 20170525

End annual number: 3

Start annual number: 1

PG1601 Publication of registration
FPAY Annual fee payment

Payment date: 20200515

Year of fee payment: 4

PR1001 Payment of annual fee

Payment date: 20200515

Start annual number: 4

End annual number: 4

FPAY Annual fee payment

Payment date: 20210514

Year of fee payment: 5

PR1001 Payment of annual fee

Payment date: 20210514

Start annual number: 5

End annual number: 5

FPAY Annual fee payment

Payment date: 20220513

Year of fee payment: 6

PR1001 Payment of annual fee

Payment date: 20220513

Start annual number: 6

End annual number: 6

PR1001 Payment of annual fee

Payment date: 20250507

Start annual number: 9

End annual number: 9