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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DK
Denmark
Prior art keywords
enzymfusion
proteins
targeted therapeutic
therapeutic lysosomal
lysosomal
Prior art date
Application number
DK16182420.6T
Other languages
English (en)
Inventor
Mika Aoyagi-Scharber
Teresa Margaret Christianson
Melita Dvorak-Ewell
Daniel Wendt
Shinong Long
Jonathan Lebowitz
Daniel Solomon Gold
Original Assignee
Biomarin Pharm 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 Biomarin Pharm Inc filed Critical 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 (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
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)

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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) EP3115372B1 (da)
JP (2) JP6831176B2 (da)
KR (4) KR102385392B1 (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) HUE043679T2 (da)
IL (3) IL238824B (da)
LT (1) LT3115372T (da)
MX (2) MX367024B (da)
PL (2) PL3115372T3 (da)
PT (2) PT3115372T (da)
RS (1) RS58916B1 (da)
RU (1) RU2680581C2 (da)
SI (1) SI3115372T1 (da)
TW (2) TWI711632B (da)
WO (1) WO2014085621A1 (da)
ZA (1) ZA201503509B (da)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2279210T3 (da) 2008-05-07 2017-07-24 Biomarin Pharm Inc Lysosomale targeting peptider og anvendelser deraf
CN104822701B (zh) 2012-11-27 2018-09-21 生物马林药物股份有限公司 靶向治疗性溶酶体酶融合蛋白及其用途
JP2016523835A (ja) * 2013-05-15 2016-08-12 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入
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
MA40791A (fr) * 2014-10-24 2017-09-27 Shire Human Genetic Therapies Ciblage lysosomal d'enzymes et utilisations associées
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
WO2017079729A1 (en) 2015-11-06 2017-05-11 Biomarin Pharmaceutical Inc. Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes
RU2751235C2 (ru) * 2016-02-24 2021-07-12 Байомарин Фармасьютикал Инк. Терапевтические слитые белки направленного действия на основе лизосомных ферментов, соответствующие составы и их применения
MA44874A (fr) 2016-04-15 2019-03-13 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type ii
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
SG11202002457RA (en) 2017-09-22 2020-04-29 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
SG11202002730XA (en) 2017-10-02 2020-04-29 Denali Therapeutics Inc Fusion proteins comprising enzyme replacement therapy enzymes
AU2019231889A1 (en) * 2018-03-09 2020-10-01 Avrobio, Inc. Compositions and methods for treating Parkinson's disease
BR112020021962A2 (pt) * 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
EP3802619A1 (en) 2018-06-08 2021-04-14 F. Hoffmann-La Roche AG Peptidic linker with reduced post-translational modification
BR112021006829A2 (pt) 2018-10-10 2021-07-20 Amicus Therapeutics, Inc. composições de polipeptídeos estabilizados com ligações dissulfeto e métodos de uso
CA3120087A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence
WO2021072372A1 (en) * 2019-10-10 2021-04-15 Amicus Therapeutics, Inc. Variant igf2 constructs
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
CN115975039A (zh) * 2021-10-15 2023-04-18 中山大学 重组融合抗体和抗体-药物偶联物及其用途

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5206161A (en) 1991-02-01 1993-04-27 Genentech, Inc. Human plasma carboxypeptidase B
AUPN674895A0 (en) 1995-11-23 1995-12-14 Women's And Children's Hospital Synthetic mammalian alpha-N-acetylglucosaminidase and genetic sequences encoding same
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
DE10035433C2 (de) 2000-07-20 2002-07-18 Tuma Wolfgang Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben
AU2002256423B2 (en) * 2001-04-30 2008-07-24 Zystor Therapeutics, Inc. Subcellular targeting of therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
JP2005506340A (ja) 2001-10-16 2005-03-03 シムバイオンティクス インコーポレイテッド 血液脳関門をわたる過小グリコシル化タンパク質の標的化のための方法および組成物
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
AU2003237314B2 (en) 2002-05-29 2010-03-04 Biomarin Pharmaceutical Inc. Targeted therapeutic proteins
MXPA05013565A (es) 2003-06-12 2006-03-09 Lilly Co Eli Proteinas de fusion analogas al glp-1.
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
ATE465250T1 (de) * 2004-02-10 2010-05-15 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
CN101171263A (zh) 2005-03-04 2008-04-30 比奥根艾迪克Ma公司 通过合理修饰互补决定区残基使免疫球蛋白可变区人源化的方法
DK2279210T3 (da) 2008-05-07 2017-07-24 Biomarin Pharm Inc Lysosomale targeting peptider og anvendelser deraf
US8697654B2 (en) * 2008-12-18 2014-04-15 E I Du Pont De Nemours And Company Peptide linkers for effective multivalent peptide binding
US9029530B2 (en) 2009-01-02 2015-05-12 Biomarin Pharmaceutical Inc. Detection of oligosaccharides
EP3679942A1 (en) * 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
MX343089B (es) 2010-06-25 2016-10-24 Amcor Ltd Sistema de depuración de oxígeno para un recipiente.
CN103269709A (zh) * 2010-06-25 2013-08-28 夏尔人类遗传性治疗公司 Sanfilippo综合症类型B的治疗
WO2012088461A2 (en) * 2010-12-23 2012-06-28 Biogen Idec Inc. Linker peptides and polypeptides comprising same
JP6175372B2 (ja) * 2011-03-04 2017-08-02 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ポリペプチド組成物用ペプチドリンカーおよびその使用方法
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
WO2012122477A1 (en) * 2011-03-10 2012-09-13 Novozymes A/S Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same
CN104822701B (zh) 2012-11-27 2018-09-21 生物马林药物股份有限公司 靶向治疗性溶酶体酶融合蛋白及其用途

Also Published As

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

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