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

Info

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
Authority
CN
China
Prior art keywords
polypeptide
gaa
kda
amino acid
modified
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.)
Pending
Application number
CN201280030662.2A
Other languages
English (en)
Chinese (zh)
Inventor
W.M.坎菲尔德
R.J.莫雷兰
工藤麻里子
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
Original Assignee
Genzyme Corp
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=46000406&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CN103797115(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CN103797115A publication Critical patent/CN103797115A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Diabetes (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (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 (enExample)
EP (1) EP2699676A1 (enExample)
JP (2) JP2014513952A (enExample)
KR (1) KR20140037082A (enExample)
CN (1) CN103797115A (enExample)
AU (1) AU2012245280A1 (enExample)
BR (1) BR112013026976A2 (enExample)
CA (1) CA2833371A1 (enExample)
CL (1) CL2013003010A1 (enExample)
CO (1) CO6811810A2 (enExample)
CR (1) CR20130555A (enExample)
EC (1) ECSP13013036A (enExample)
GT (1) GT201300252A (enExample)
IL (1) IL228871A0 (enExample)
MA (1) MA35125B1 (enExample)
MX (1) MX2013012345A (enExample)
NI (1) NI201300110A (enExample)
PE (1) PE20140617A1 (enExample)
PH (1) PH12013502149A1 (enExample)
RU (1) RU2013151875A (enExample)
SG (2) SG194486A1 (enExample)
TN (1) TN2013000427A1 (enExample)
WO (1) WO2012145644A1 (enExample)
ZA (1) ZA201307696B (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
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

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10017581B2 (en) 2013-02-20 2018-07-10 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
FI3201320T3 (fi) 2014-09-30 2024-01-08 Amicus Therapeutics Inc Erittäin tehokas hapan alfa-glukosidaasi parannetuilla hiilihydraateilla
CA3010205A1 (en) * 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
KR20250050123A (ko) * 2016-03-30 2025-04-14 아미쿠스 세라퓨틱스, 인코포레이티드 고 m6p 재조합 단백질의 선택 방법
AU2017239641A1 (en) 2016-03-30 2018-10-18 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
IL299325B2 (en) 2016-09-12 2025-08-01 Inst Nat Sante Rech Med Acid-alpha glucosidase variants and uses thereof
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293203A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293260A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
PT3624831T (pt) 2017-05-15 2023-07-04 Amicus Therapeutics Inc Alfa-glucosidase ácida humana recombinante
EP3635009B1 (en) 2017-06-07 2026-02-25 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
KR101942093B1 (ko) * 2018-01-05 2019-01-24 인하대학교 산학협력단 만노시다제 억제제를 포함하는 고만노즈 타입 n-당쇄를 갖는 인간 리소좀 효소 생산용 조성물
IL276464B2 (en) 2018-02-07 2026-01-01 Regeneron Pharma Methods and compositions for administering therapeutic protein
MA52626A (fr) 2018-05-17 2021-03-24 Regeneron Pharma Anticorps anti-cd63, conjugués et leurs utilisations
WO2021005176A1 (en) 2019-07-09 2021-01-14 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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1408117A1 (en) * 2001-06-14 2004-04-14 National Institute of Advanced Industrial Science and Technology Glycoprotein and process for producing the same
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

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6071890A (en) 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
US5952516A (en) 1997-05-08 1999-09-14 Genzyme Corporation Cationic amphiphiles containing multiplesteroid lipophilic groups
WO2000009153A1 (en) 1997-10-29 2000-02-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
US6287857B1 (en) 1998-02-09 2001-09-11 Genzyme Corporation Nucleic acid delivery vehicles
GB9807464D0 (en) * 1998-04-07 1998-06-10 Pharming Bv Purification of human acid µ-glucosidase
KR20010101131A (ko) * 1998-12-07 2001-11-14 추후기재 폼페병의 치료 방법
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
US7001994B2 (en) 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
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
EP2266968B1 (en) 2001-07-16 2013-01-09 Genzyme Corporation Synthesis of UDP-glucose: N-acylsphingosine glucosyltransferase inhibitors
AU2002348261A1 (en) 2001-11-26 2003-06-10 Genzyme Corporation Diastereoselective synthesis of udp-glucose : n-acylsphingosine glucosyltransferase inhibitors
US6916802B2 (en) 2002-04-29 2005-07-12 Genzyme Corporation Amino ceramide-like compounds and therapeutic methods of use
EP1587923B1 (en) * 2003-01-22 2011-08-24 Duke University Improved constructs for expressing lysosomal polypeptides
US20100196345A1 (en) 2003-04-27 2010-08-05 Protalix Production of high mannose proteins in plant culture
EP3346010A1 (en) 2003-06-24 2018-07-11 Genzyme Corporation Novel beta-actin and rps21 promoters and uses thereof
EP2099523A2 (en) * 2006-11-13 2009-09-16 ZyStor Therapeutics , Inc. Methods for treating pompe disease
SI2889043T1 (sl) 2008-12-16 2019-08-30 Genzyme Corporation Sintetične vmesne spojine za pripravo konjugatov oligosaharid-protein

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1408117A1 (en) * 2001-06-14 2004-04-14 National Institute of Advanced Industrial Science and Technology Glycoprotein and process for producing the same
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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
RABEN N ET AL.: "Acid alpha-glucosidase deficiency(Glycogenosis Type II, Pompe disease)", 《CURRENT MOLECULAR MEDICINE》 *

Cited By (2)

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

Similar Documents

Publication Publication Date Title
CN103797115A (zh) 具有快速加工性能的经过修饰的酸性α葡糖苷酶
US11116823B2 (en) Treatment of α-galactosidase a deficiency
US20240066104A1 (en) Formulations comprising recombinant acid alpha-glucosidase
US11441138B2 (en) Method for selection of high M6P recombinant proteins
CN104822701A (zh) 靶向治疗性溶酶体酶融合蛋白及其用途
US20230058973A1 (en) Mutated arylsulfatase a
OA16639A (en) Modified acid alpha glucosidase with accelerated processing.
US20240167007A1 (en) Mutated arylsulfatase a with increased stability
EA043224B1 (ru) Составы, содержащие рекомбинантную кислую альфа-глюкозидазу
HK1161548A (en) Treatment of alpha-galactosidase a deficiency

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20140514