DK3115372T3 - Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf - Google Patents

Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf Download PDF

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Publication number
DK3115372T3
DK3115372T3 DK16182420.6T DK16182420T DK3115372T3 DK 3115372 T3 DK3115372 T3 DK 3115372T3 DK 16182420 T DK16182420 T DK 16182420T DK 3115372 T3 DK3115372 T3 DK 3115372T3
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Denmark
Prior art keywords
enzymfusion
proteins
targeted therapeutic
therapeutic lysosomal
lysosomal
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DK16182420.6T
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English (en)
Inventor
Mika Aoyagi-Scharber
Teresa Margaret Christianson
Melita Dvorak-Ewell
Daniel Wendt
Shinong Long
Jonathan Lebowitz
Daniel Solomon Gold
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Biomarin Pharm Inc
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Publication of DK3115372T3 publication Critical patent/DK3115372T3/da

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575Hormones
    • C07K14/65Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • 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
    • 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
    • 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)
    • 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)
    • C12Y302/0105Alpha-N-acetylglucosaminidase (3.2.1.50)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
DK16182420.6T 2012-11-27 2013-11-27 Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf DK3115372T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261730378P 2012-11-27 2012-11-27
US201361788968P 2013-03-15 2013-03-15
EP13802834.5A EP2925776B1 (en) 2012-11-27 2013-11-27 Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof

Publications (1)

Publication Number Publication Date
DK3115372T3 true DK3115372T3 (da) 2019-06-11

Family

ID=49753532

Family Applications (2)

Application Number Title Priority Date Filing Date
DK16182420.6T DK3115372T3 (da) 2012-11-27 2013-11-27 Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf
DK13802834.5T DK2925776T3 (da) 2012-11-27 2013-11-27 Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK13802834.5T DK2925776T3 (da) 2012-11-27 2013-11-27 Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf

Country Status (27)

Country Link
US (8) US9376480B2 (da)
EP (2) EP2925776B1 (da)
JP (2) JP6831176B2 (da)
KR (4) KR102262882B1 (da)
CN (1) CN104822701B (da)
AR (1) AR093626A1 (da)
AU (1) AU2013352184B2 (da)
BR (1) BR112015012152B1 (da)
CA (1) CA2892146A1 (da)
CL (1) CL2015001371A1 (da)
CY (1) CY1122555T1 (da)
DK (2) DK3115372T3 (da)
ES (2) ES2729997T3 (da)
HK (1) HK1216026A1 (da)
HR (2) HRP20181351T1 (da)
HU (2) HUE039334T2 (da)
IL (3) IL238824B (da)
LT (1) LT3115372T (da)
MX (2) MX367024B (da)
PL (2) PL3115372T3 (da)
PT (2) PT2925776T (da)
RS (1) RS58916B1 (da)
RU (1) RU2680581C2 (da)
SI (1) SI3115372T1 (da)
TW (2) TWI626250B (da)
WO (1) WO2014085621A1 (da)
ZA (1) ZA201503509B (da)

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EP3622821A1 (en) * 2013-05-15 2020-03-18 Regents of the University of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
WO2016025519A1 (en) * 2014-08-11 2016-02-18 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
EP3197503A4 (en) * 2014-09-25 2018-05-16 The General Hospital Corporation Cell-based targeted delivery of pseudonomas exotoxin
EP3220958A1 (en) 2014-10-24 2017-09-27 Shire Human Genetic Therapies, Inc. Lysosomal targeting of enzymes, and uses thereof
TWI752907B (zh) 2015-05-08 2022-01-21 美商拜奧馬林製藥公司 用於治療cln2疾病之tpp1調配物及方法
JP2018538008A (ja) 2015-11-06 2018-12-27 バイオマリン ファーマシューティカル インコーポレイテッド リソソーム酵素の取り込みを中和する抗体または他の因子の検出のための細胞ベースアッセイ
CA3015358A1 (en) 2016-02-24 2017-08-31 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
KR20230125339A (ko) 2016-04-15 2023-08-29 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
CL2016003282A1 (es) 2016-12-21 2017-08-18 Univ Chile Virus aav/igf2, método de tratamiento genético y su uso en enfermedades relacionadas con mal plegamiento de proteínas tal como la enfermedad de huntington
AU2018335752A1 (en) 2017-09-22 2020-03-12 Christian HINDERER Gene therapy for treating Mucopolysaccharidosis type ii
CN111278859A (zh) 2017-10-02 2020-06-12 戴纳立制药公司 包含酶替代疗法酶的融合蛋白
WO2019173756A1 (en) * 2018-03-09 2019-09-12 Avrobio, Inc. Compositions and methods for treating parkinson's disease
TWI808169B (zh) * 2018-04-30 2023-07-11 美商阿米庫斯醫療股份有限公司 基因治療構築體及使用方法
EP3802619A1 (en) 2018-06-08 2021-04-14 F. Hoffmann-La Roche AG Peptidic linker with reduced post-translational modification
WO2020077114A2 (en) 2018-10-10 2020-04-16 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
WO2020102667A2 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence
TW202128740A (zh) * 2019-10-10 2021-08-01 美商阿米庫斯醫療股份有限公司 變體igf2構築體
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
CN117343197A (zh) * 2021-10-15 2024-01-05 中山大学 重组融合抗体

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Publication number Publication date
AU2013352184A1 (en) 2015-06-04
IL272854B (en) 2021-07-29
JP6913719B2 (ja) 2021-08-04
KR102521039B1 (ko) 2023-04-12
HUE043679T2 (hu) 2019-09-30
KR20210070389A (ko) 2021-06-14
US9376480B2 (en) 2016-06-28
IL262976B (en) 2020-02-27
JP6831176B2 (ja) 2021-02-17
PL3115372T3 (pl) 2019-09-30
KR102262882B1 (ko) 2021-06-10
LT3115372T (lt) 2019-06-25
US9845346B2 (en) 2017-12-19
TWI711632B (zh) 2020-12-01
HRP20181351T1 (hr) 2018-11-02
US9771408B2 (en) 2017-09-26
HK1216026A1 (zh) 2016-10-07
US20190225666A1 (en) 2019-07-25
US9834587B2 (en) 2017-12-05
US10301369B2 (en) 2019-05-28
BR112015012152A2 (pt) 2017-08-15
US20160031965A1 (en) 2016-02-04
ES2729997T3 (es) 2019-11-07
CL2015001371A1 (es) 2015-10-09
IL272854A (en) 2020-04-30
HUE039334T2 (hu) 2018-12-28
AU2013352184B2 (en) 2018-05-31
PT3115372T (pt) 2019-06-12
KR102385392B1 (ko) 2022-04-11
RS58916B1 (sr) 2019-08-30
TW201827468A (zh) 2018-08-01
MX2019009191A (es) 2019-10-09
BR112015012152B1 (pt) 2023-04-25
JP2019206556A (ja) 2019-12-05
EP3115372A1 (en) 2017-01-11
TW201431884A (zh) 2014-08-16
KR20150088317A (ko) 2015-07-31
EP3115372B1 (en) 2019-03-06
WO2014085621A1 (en) 2014-06-05
IL238824B (en) 2018-11-29
RU2680581C2 (ru) 2019-02-22
US20220127326A1 (en) 2022-04-28
RU2015125491A (ru) 2017-01-10
CN104822701B (zh) 2018-09-21
AR093626A1 (es) 2015-06-17
EP2925776B1 (en) 2018-05-30
TWI626250B (zh) 2018-06-11
US20170355744A1 (en) 2017-12-14
US20140161788A1 (en) 2014-06-12
CY1122555T1 (el) 2021-01-27
US20160031964A1 (en) 2016-02-04
US11254725B2 (en) 2022-02-22
KR20230054482A (ko) 2023-04-24
KR20220047892A (ko) 2022-04-19
IL262976A (en) 2018-12-31
DK2925776T3 (da) 2018-09-03
CN104822701A (zh) 2015-08-05
MX2015006644A (es) 2015-08-10
SI3115372T1 (sl) 2019-08-30
HRP20190918T1 (hr) 2019-09-20
CA2892146A1 (en) 2014-06-05
IL238824A0 (en) 2015-06-30
US9834588B2 (en) 2017-12-05
US20160031963A1 (en) 2016-02-04
PT2925776T (pt) 2018-07-30
MX367024B (es) 2019-08-02
ZA201503509B (en) 2016-11-30
PL2925776T3 (pl) 2018-11-30
US20160039900A1 (en) 2016-02-11
ES2679374T3 (es) 2018-08-24
JP2016505539A (ja) 2016-02-25
EP2925776A1 (en) 2015-10-07

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