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LT4273241T - Labai stipriai veikianti rūgštinė alfa-gliukosidazė, pasižyminti didesniu angliavandenių kiekiu - Google Patents

Labai stipriai veikianti rūgštinė alfa-gliukosidazė, pasižyminti didesniu angliavandenių kiekiu

Info

Publication number
LT4273241T
LT4273241T LTEP23191307.0T LT23191307T LT4273241T LT 4273241 T LT4273241 T LT 4273241T LT 23191307 T LT23191307 T LT 23191307T LT 4273241 T LT4273241 T LT 4273241T
Authority
LT
Lithuania
Prior art keywords
glucosidase
acid alpha
higher carbohydrates
powerful acid
powerful
Prior art date
Application number
LTEP23191307.0T
Other languages
English (en)
Inventor
Russell GOTSCHALL
Hung Do
Original Assignee
Amicus Therapeutics, 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 Amicus Therapeutics, Inc. filed Critical Amicus Therapeutics, Inc.
Publication of LT4273241T publication Critical patent/LT4273241T/lt

Links

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    • 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)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102Alpha-glucosidase (3.2.1.20)
    • 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
    • C12N2510/00Genetically modified cells
    • C12N2510/02Cells for production

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
LTEP23191307.0T 2014-09-30 2015-09-30 Labai stipriai veikianti rūgštinė alfa-gliukosidazė, pasižyminti didesniu angliavandenių kiekiu LT4273241T (lt)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201462057847P 2014-09-30 2014-09-30
US201462057842P 2014-09-30 2014-09-30
US201562112463P 2015-02-05 2015-02-05
US201562135345P 2015-03-19 2015-03-19

Publications (1)

Publication Number Publication Date
LT4273241T true LT4273241T (lt) 2025-02-10

Family

ID=55631441

Family Applications (4)

Application Number Title Priority Date Filing Date
LTEP23191307.0T LT4273241T (lt) 2014-09-30 2015-09-30 Labai stipriai veikianti rūgštinė alfa-gliukosidazė, pasižyminti didesniu angliavandenių kiekiu
LTEPPCT/US2015/053252T LT3201320T (lt) 2014-09-30 2015-09-30 Didelio aktyvumo rūgštinė alfa-gliukozidazė su sustiprintais karbohidratais
LTPA2024509C LTPA2024509I1 (lt) 2014-09-30 2024-04-02
LTPA2025521C LTPA2025521I1 (lt) 2014-09-30 2025-06-02

Family Applications After (3)

Application Number Title Priority Date Filing Date
LTEPPCT/US2015/053252T LT3201320T (lt) 2014-09-30 2015-09-30 Didelio aktyvumo rūgštinė alfa-gliukozidazė su sustiprintais karbohidratais
LTPA2024509C LTPA2024509I1 (lt) 2014-09-30 2024-04-02
LTPA2025521C LTPA2025521I1 (lt) 2014-09-30 2025-06-02

Country Status (39)

Country Link
US (6) US10208299B2 (lt)
EP (3) EP4273241B1 (lt)
JP (3) JP6851964B2 (lt)
KR (4) KR102741048B1 (lt)
CN (2) CN107075468B (lt)
AU (3) AU2015325028B2 (lt)
BR (1) BR112017005810A2 (lt)
CA (2) CA2961762C (lt)
CL (1) CL2017000722A1 (lt)
CO (1) CO2017002776A2 (lt)
CR (1) CR20170107A (lt)
CY (2) CY1126544T1 (lt)
DK (2) DK3201320T5 (lt)
DO (1) DOP2017000079A (lt)
EA (1) EA038986B1 (lt)
EC (1) ECSP17019103A (lt)
ES (2) ES3017134T3 (lt)
FI (4) FI4273241T3 (lt)
FR (2) FR24C1012I1 (lt)
HR (2) HRP20250058T1 (lt)
HU (4) HUE064629T2 (lt)
IL (4) IL307587A (lt)
LT (4) LT4273241T (lt)
MX (2) MX388241B (lt)
MY (1) MY186336A (lt)
NI (1) NI201700039A (lt)
NO (1) NO2025023I1 (lt)
PE (1) PE20170906A1 (lt)
PH (1) PH12017500455A1 (lt)
PL (2) PL4273241T3 (lt)
PT (2) PT3201320T (lt)
RS (2) RS66510B1 (lt)
SG (2) SG11201702114TA (lt)
SI (2) SI4273241T1 (lt)
SM (2) SMT202400010T1 (lt)
SV (1) SV2017005416A (lt)
TN (1) TN2017000082A1 (lt)
WO (1) WO2016054231A1 (lt)
ZA (2) ZA201701945B (lt)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102741048B1 (ko) 2014-09-30 2024-12-11 아미쿠스 세라퓨틱스, 인코포레이티드 개선된 탄수화물을 가지는 고도로 강력한 산성 알파-글루코시다제
CA3010205A1 (en) * 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
HUE054733T2 (hu) * 2015-12-30 2021-09-28 Amicus Therapeutics Inc Megnövelt savas alfa-glükozidáz Pompe-kór kezelésére
TWI823272B (zh) * 2016-03-30 2023-11-21 美商阿米庫斯醫療股份有限公司 用於選擇高m6p重組蛋白之方法
MX2018011951A (es) * 2016-03-30 2019-02-13 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p.
KR20250017756A (ko) 2016-03-30 2025-02-04 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
TW201829770A (zh) * 2017-01-10 2018-08-16 美商阿米庫斯醫療股份有限公司 用於治療fabry氏病之重組α-半乳糖苷酶A
EA202290114A1 (ru) * 2017-03-30 2022-03-29 Амикус Терапьютикс, Инк. Способ отбора рекомбинантных белков с высоким содержанием m6p
HRP20230576T1 (hr) 2017-05-15 2023-09-01 Amicus Therapeutics, Inc. Rekombinantna ljudska kisela alfa-glukozidaza
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2021046443A1 (en) 2019-09-06 2021-03-11 Amicus Therapeutics, Inc. Method for capturing and purification of biologics
BR112023016212A2 (pt) * 2021-02-11 2023-11-28 Amicus Therapeutics Inc Alfa-glucosidase ácida humana recombinante e usos da mesma
US20250134967A1 (en) * 2022-02-07 2025-05-01 M6P Therapeutics, Inc. Compositions comprising acid alpha glucosidase and methods of use thereof
AU2023265155A1 (en) * 2022-05-05 2024-12-12 Amicus Therapeutics, Inc. Methods for treating pompe disease
AU2023406510A1 (en) * 2022-12-02 2025-07-10 Amicus Therapeutics, Inc. Fexamethods for treating infantile-onset pompe disease in pediatric patients
EP4626465A1 (en) * 2022-12-02 2025-10-08 Amicus Therapeutics, Inc. Methods for treating late onset pompe disease in pediatric patients
WO2026006625A1 (en) 2024-06-26 2026-01-02 Amicus Therapeutics, Inc. Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4837237A (en) 1985-07-09 1989-06-06 Fred Hutchinson Cancer Research Center Therapy using glucosidase processing inhibitors
US5879680A (en) 1987-12-23 1999-03-09 The United States Of America As Represented By The Department Of Health And Human Services Cloned DNA for synthesizing unique glucocerebrosidase
US4985445A (en) 1988-02-12 1991-01-15 Meiji Seika Kaisha, Ltd. Cancer cell metastasis inhibitors and novel compounds
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5236838A (en) 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5179023A (en) 1989-03-24 1993-01-12 Research Corporation Technologies, Inc. Recombinant α-galactosidase a therapy for Fabry disease
US5011829A (en) 1989-06-02 1991-04-30 G. D. Searle & Co. Pharmaceutical composition and method of inhibiting virus
US5103008A (en) 1989-08-17 1992-04-07 Monsanto Company Compound, N-butyl-deoxynojirimycin-6-phosphate
US5401650A (en) 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
US5399567A (en) 1993-05-13 1995-03-21 Monsanto Company Method of treating cholera
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
US6458574B1 (en) 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US6210666B1 (en) 1997-10-21 2001-04-03 Orphan Medical, Inc. Truncated α-galactosidase A to treat fabry disease
CA2309321A1 (en) 1997-11-10 1999-05-20 G.D. Searle & Co. Use of alkylated iminosugars to treat multidrug resistance
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
JP4990434B2 (ja) 1998-12-07 2012-08-01 ジェンザイム・コーポレーション ポンペ病の処置
WO2000047198A2 (en) 1999-02-12 2000-08-17 G.D. Searle & Co. Use of substituted-1,5-dideoxy-1,5-imino-d-glucitol compounds for treating hepatitis virus infections
ATE234626T1 (de) 1999-07-26 2003-04-15 Searle & Co Verwendung von langkettigen n-alkyl derivaten von deoxynojirimycin mit glucocerebrosidase enzyme zur herstellung eines medikaments zur behandlung von mit glykolipiden akkumulation zusammenhängenden krankheiten
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
ES2523024T3 (es) 2000-07-18 2014-11-20 Duke University Tratamiento de glucogenosis de tipo II
CN1638739A (zh) 2000-08-18 2005-07-13 法玛西雅厄普约翰美国公司 治疗成瘾性障碍的化合物
US7723296B2 (en) 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
IL158623A0 (en) 2001-04-30 2004-05-12 Symbiontics Inc Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
JP2005523882A (ja) 2001-10-16 2005-08-11 アールエックスキネティックス,インコーポレイテッド 高濃度タンパク質製剤および製造の方法
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US7658916B2 (en) 2002-04-05 2010-02-09 Genzyme Corporation Methods of enhancing lysosomal storage disease therapy by modulation of cell surface receptor density
ES2545619T3 (es) 2003-01-31 2015-09-14 The Mount Sinai School Of Medicine Of New York University Terapia de combinación para tratar trastornos de deficiencia de proteínas
FR2861991B1 (fr) 2003-11-07 2008-01-18 Centre Nat Rech Scient Utilisation d'inhibiteurs de glucosidase pour une therapie de la mucoviscidose
AU2005211775B2 (en) * 2004-02-06 2009-10-08 Biomarin Pharmaceutical Inc. Manufacture of highly phosphorylated lysosomal enzymes and uses thereof
EP1716232B9 (en) 2004-02-10 2010-10-13 ZyStor Therapeutics , Inc. Acid alpha-glucosidase and fragments thereof
US20060142234A1 (en) 2004-12-23 2006-06-29 Guohua Chen Injectable non-aqueous suspension
MX2007014470A (es) 2005-05-17 2008-02-06 Amicus Therapeutics Inc Metodo para el tratamiento de enfermedad de pompe usando derivados de 1-desoxinojirimicina.
AU2007322123A1 (en) 2006-11-13 2008-05-29 Biomarin Pharmaceutical Inc. Methods for treating Pompe disease
CN101636200A (zh) * 2006-11-13 2010-01-27 齐斯特治疗公司 用于治疗庞贝氏症的方法
WO2008112525A2 (en) 2007-03-09 2008-09-18 Link Medicine Corporation Treatment of lysosomal storage diseases
CA2685332A1 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2252288A1 (en) 2007-11-21 2010-11-24 Summit Corporation Plc Treatment of protein folding disorders
WO2009075815A1 (en) 2007-12-07 2009-06-18 Duke University Immunomodulating gene therapy
DK2252313T3 (en) 2008-02-12 2015-07-13 Amicus Therapeutics Inc METHOD TO PREDICT response to Pharmacological chaperone TREATMENT OF DISEASES
CA2718182A1 (en) 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Treatment of pompe disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
JP2011517556A (ja) 2008-03-12 2011-06-16 アミカス セラピューティックス インコーポレイテッド ポンペ病のための治療選択肢を診断し評価するための検定
CA2723412A1 (en) * 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
EP2323652A2 (en) 2008-08-06 2011-05-25 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
US20100119502A1 (en) 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
HUE060974T2 (hu) 2008-12-16 2023-04-28 Genzyme Corp Szintetikus intermedierek oligoszacharid-fehérje konjugátumok elõállítására
US8466340B2 (en) * 2009-02-18 2013-06-18 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
ES2564093T3 (es) 2009-04-09 2016-03-17 Amicus Therapeutics, Inc. Métodos para prevenir y/o tratar trastornos del almacenamiento lisosomal
EP2475376B1 (en) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
JP5415170B2 (ja) 2009-07-21 2014-02-12 富士フイルム株式会社 複眼撮像装置
CN104651428A (zh) * 2009-09-29 2015-05-27 根特大学 水解甘露糖-1-磷酸-6-甘露糖连接为磷酸-6-甘露糖
WO2011109600A1 (en) 2010-03-05 2011-09-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for modifying the glycosylation pattern of a polypeptide
JP6151183B2 (ja) * 2010-09-29 2017-06-21 オキシレイン ユーケー リミテッド マンノース−1−ホスホ−6−マンノース結合からキャップを外すことおよびリン酸化n−グリカンを脱マンノシル化することができるマンノシダーゼならびに哺乳動物細胞による糖タンパク質の取り込みを促進する方法
SG194486A1 (en) 2011-04-22 2013-12-30 Genzyme Corp Modified acid alpha glucosidase with accelerated processing
BR112013030432A2 (pt) * 2011-05-27 2016-12-13 Callidus Biopharma Inc método para preparar um peptídeo conjugado alvo para uma enzima lisossomal recombinante, método para preparar uma molécula para terapia de reposição de enzima, conjugado, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um paciente sofrendo da doença de pompe, doença de fabry, e doença de gaucher, mps i, mps ii, mps vii, tay sachs, sandhoff, alfa-manosidose, e wohlman, sequência de dna e sequência de aminoácidos
WO2013013017A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Compositions and methods for modifying the glycosylation of lysosomal storage disorder therapeutics
JP2014522105A (ja) * 2011-07-22 2014-08-28 インサイト フォトニック ソリューションズ,インコーポレイテッド 波長連続及び規定された時間に対する波長掃引をレーザーから動的及び適応的に生成するシステム及び方法
CA2860085A1 (en) 2011-12-22 2013-06-27 Centogene Ip Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
WO2013134530A1 (en) 2012-03-07 2013-09-12 Amicus Therapeutics, Inc. High concentration alpha-glucosidase compositions for the treatment of pompe disease
US20150086530A1 (en) 2012-05-03 2015-03-26 Amicus Therapeutics, Inc. Dosing regimens for the treatment of pompe disease
US20150147309A1 (en) 2012-06-06 2015-05-28 Fondazione Telethon Allosteric chaperones and uses thereof
TWI529396B (zh) 2014-07-18 2016-04-11 Mpi Corp Probe card and its transfer circuit board and signal feed structure
KR102741048B1 (ko) 2014-09-30 2024-12-11 아미쿠스 세라퓨틱스, 인코포레이티드 개선된 탄수화물을 가지는 고도로 강력한 산성 알파-글루코시다제

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