CN103797115A - 具有快速加工性能的经过修饰的酸性α葡糖苷酶 - Google Patents

具有快速加工性能的经过修饰的酸性α葡糖苷酶 Download PDF

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Publication number
CN103797115A
CN103797115A CN201280030662.2A CN201280030662A CN103797115A CN 103797115 A CN103797115 A CN 103797115A CN 201280030662 A CN201280030662 A CN 201280030662A CN 103797115 A CN103797115 A CN 103797115A
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China
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polypeptide
gaa
kda
amino acid
modified
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Pending
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CN201280030662.2A
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English (en)
Chinese (zh)
Inventor
W.M.坎菲尔德
R.J.莫雷兰
工藤麻里子
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Genzyme Corp
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Genzyme Corp
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Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CN103797115A publication Critical patent/CN103797115A/zh
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    • 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)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Diabetes (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Endocrinology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Emergency Medicine (AREA)
  • Epidemiology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
CN201280030662.2A 2011-04-22 2012-04-20 具有快速加工性能的经过修饰的酸性α葡糖苷酶 Pending CN103797115A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161478336P 2011-04-22 2011-04-22
US61/478,336 2011-04-22
PCT/US2012/034479 WO2012145644A1 (en) 2011-04-22 2012-04-20 Modified acid alpha glucosidase with accelerated processing

Publications (1)

Publication Number Publication Date
CN103797115A true CN103797115A (zh) 2014-05-14

Family

ID=46000406

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201280030662.2A Pending CN103797115A (zh) 2011-04-22 2012-04-20 具有快速加工性能的经过修饰的酸性α葡糖苷酶

Country Status (24)

Country Link
US (1) US20140186326A1 (https=)
EP (1) EP2699676A1 (https=)
JP (2) JP2014513952A (https=)
KR (1) KR20140037082A (https=)
CN (1) CN103797115A (https=)
AU (1) AU2012245280A1 (https=)
BR (1) BR112013026976A2 (https=)
CA (1) CA2833371A1 (https=)
CL (1) CL2013003010A1 (https=)
CO (1) CO6811810A2 (https=)
CR (1) CR20130555A (https=)
EC (1) ECSP13013036A (https=)
GT (1) GT201300252A (https=)
IL (1) IL228871A0 (https=)
MA (1) MA35125B1 (https=)
MX (1) MX2013012345A (https=)
NI (1) NI201300110A (https=)
PE (1) PE20140617A1 (https=)
PH (1) PH12013502149A1 (https=)
RU (1) RU2013151875A (https=)
SG (2) SG10201605874TA (https=)
TN (1) TN2013000427A1 (https=)
WO (1) WO2012145644A1 (https=)
ZA (1) ZA201307696B (https=)

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CN108474788A (zh) * 2015-11-06 2018-08-31 生物马林药物股份有限公司 用于检测中和溶酶体酶的摄取的抗体或其它因子的基于细胞的测定

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AU2014218854B2 (en) 2013-02-20 2019-01-24 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
CN114540327B (zh) 2014-09-30 2026-03-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
KR20250007693A (ko) * 2015-12-30 2025-01-14 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
CA3019354A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Method for selection of high m6p recombinant proteins
EP3293260A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2018046774A1 (en) 2016-09-12 2018-03-15 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
SMT202300187T1 (it) 2017-05-15 2023-07-20 Amicus Therapeutics Inc Alfa-glucosidasi acida umana ricombinante
DK3635009T3 (da) 2017-06-07 2026-03-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
KR101942093B1 (ko) * 2018-01-05 2019-01-24 인하대학교 산학협력단 만노시다제 억제제를 포함하는 고만노즈 타입 n-당쇄를 갖는 인간 리소좀 효소 생산용 조성물
US12258597B2 (en) 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
EP3996696A1 (en) 2019-07-09 2022-05-18 Genethon Treatment of glycogen storage disease (gsd)
KR20220098384A (ko) * 2019-11-19 2022-07-12 아스클레피오스 바이오파마슈티컬, 인크. 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스

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WO2005078077A2 (en) * 2004-02-10 2005-08-25 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
WO2010096369A1 (en) * 2009-02-18 2010-08-26 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof

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WO2005078077A2 (en) * 2004-02-10 2005-08-25 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
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Cited By (2)

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Publication number Priority date Publication date Assignee Title
CN108474788A (zh) * 2015-11-06 2018-08-31 生物马林药物股份有限公司 用于检测中和溶酶体酶的摄取的抗体或其它因子的基于细胞的测定
US11262348B2 (en) 2015-11-06 2022-03-01 Biomarin Pharmaceutical Inc. Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes

Also Published As

Publication number Publication date
PE20140617A1 (es) 2014-05-28
ZA201307696B (en) 2014-07-30
JP2014513952A (ja) 2014-06-19
JP2017035091A (ja) 2017-02-16
BR112013026976A2 (pt) 2019-09-24
US20140186326A1 (en) 2014-07-03
IL228871A0 (en) 2013-12-31
CA2833371A1 (en) 2012-10-26
EP2699676A1 (en) 2014-02-26
GT201300252A (es) 2015-02-09
NI201300110A (es) 2014-02-28
KR20140037082A (ko) 2014-03-26
MX2013012345A (es) 2015-05-07
WO2012145644A1 (en) 2012-10-26
CO6811810A2 (es) 2013-12-16
AU2012245280A1 (en) 2013-11-07
RU2013151875A (ru) 2015-05-27
ECSP13013036A (es) 2015-04-30
SG10201605874TA (en) 2016-09-29
MA35125B1 (fr) 2014-05-02
SG194486A1 (en) 2013-12-30
PH12013502149A1 (en) 2019-09-02
CR20130555A (es) 2013-12-09
TN2013000427A1 (en) 2015-03-30
CL2013003010A1 (es) 2014-03-07

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Application publication date: 20140514