HUE039334T2 - Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai - Google Patents

Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai

Info

Publication number
HUE039334T2
HUE039334T2 HUE13802834A HUE13802834A HUE039334T2 HU E039334 T2 HUE039334 T2 HU E039334T2 HU E13802834 A HUE13802834 A HU E13802834A HU E13802834 A HUE13802834 A HU E13802834A HU E039334 T2 HUE039334 T2 HU E039334T2
Authority
HU
Hungary
Prior art keywords
fusion proteins
lysosomal enzyme
targeted therapeutic
enzyme fusion
therapeutic lysosomal
Prior art date
Application number
HUE13802834A
Other languages
English (en)
Inventor
Mika Aoyagi-Scharber
Teresa Christianson
Melita Dvorak-Ewell
Daniel Wendt
Shinong Long
Jonathan Lebowitz
Daniel 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
Publication of HUE039334T2 publication Critical patent/HUE039334T2/hu

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • 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)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
HUE13802834A 2012-11-27 2013-11-27 Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai HUE039334T2 (hu)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261730378P 2012-11-27 2012-11-27
US201361788968P 2013-03-15 2013-03-15

Publications (1)

Publication Number Publication Date
HUE039334T2 true HUE039334T2 (hu) 2018-12-28

Family

ID=49753532

Family Applications (2)

Application Number Title Priority Date Filing Date
HUE16182420A HUE043679T2 (hu) 2012-11-27 2013-11-27 Célra irányított terápiás lizoszomális enzim fúziós fehérjék és alkalmazásaik
HUE13802834A HUE039334T2 (hu) 2012-11-27 2013-11-27 Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai

Family Applications Before (1)

Application Number Title Priority Date Filing Date
HUE16182420A HUE043679T2 (hu) 2012-11-27 2013-11-27 Célra irányított terápiás lizoszomális enzim fúziós fehérjék és alkalmazásaik

Country Status (27)

Country Link
US (8) US9376480B2 (hu)
EP (2) EP3115372B1 (hu)
JP (2) JP6831176B2 (hu)
KR (4) KR102262882B1 (hu)
CN (1) CN104822701B (hu)
AR (1) AR093626A1 (hu)
AU (1) AU2013352184B2 (hu)
BR (1) BR112015012152B1 (hu)
CA (1) CA2892146A1 (hu)
CL (1) CL2015001371A1 (hu)
CY (1) CY1122555T1 (hu)
DK (2) DK2925776T3 (hu)
ES (2) ES2679374T3 (hu)
HK (1) HK1216026A1 (hu)
HR (2) HRP20181351T1 (hu)
HU (2) HUE043679T2 (hu)
IL (3) IL238824B (hu)
LT (1) LT3115372T (hu)
MX (2) MX367024B (hu)
PL (2) PL2925776T3 (hu)
PT (2) PT3115372T (hu)
RS (1) RS58916B1 (hu)
RU (1) RU2680581C2 (hu)
SI (1) SI3115372T1 (hu)
TW (2) TWI626250B (hu)
WO (1) WO2014085621A1 (hu)
ZA (1) ZA201503509B (hu)

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PL2279210T3 (pl) * 2008-05-07 2017-10-31 Biomarin Pharm Inc Lizosomalne peptydy kierujące i ich zastosowania
KR102262882B1 (ko) 2012-11-27 2021-06-10 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들
CN115120746A (zh) * 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
EP3185889B1 (en) * 2014-08-11 2020-07-15 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
WO2016049510A2 (en) * 2014-09-25 2016-03-31 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
CA3003750A1 (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
AU2017222620B2 (en) * 2016-02-24 2022-06-16 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
JP7171439B2 (ja) 2016-04-15 2022-11-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉ型を処置するための遺伝子療法
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
US11819539B2 (en) 2017-09-22 2023-11-21 The Trustees Of The University Of Pennsylvania Gene therapy for treating Mucopolysaccharidosis type II
MA50746A (fr) 2017-10-02 2020-08-12 Denali Therapeutics Inc Protéines de fusion comprenant des enzymes d'enzymothérapie substitutive
AU2019231889A1 (en) * 2018-03-09 2020-10-01 Avrobio, Inc. Compositions and methods for treating Parkinson's disease
JP2021521851A (ja) * 2018-04-30 2021-08-30 アミカス セラピューティックス インコーポレイテッド 遺伝子治療構築物及び使用方法
CN112243444A (zh) 2018-06-08 2021-01-19 豪夫迈·罗氏有限公司 具有减少的翻译后修饰的肽接头
US11097015B2 (en) 2018-10-10 2021-08-24 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
AU2019381803A1 (en) * 2018-11-16 2021-06-10 Asklepios Biopharmaceutical, Inc. Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence
CN115087458A (zh) * 2019-10-10 2022-09-20 阿米库斯治疗学公司 变体igf2构建体
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

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AUPN674895A0 (en) 1995-11-23 1995-12-14 Women's And Children's Hospital Synthetic mammalian alpha-N-acetylglucosaminidase and genetic sequences encoding same
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DE10035433C2 (de) 2000-07-20 2002-07-18 Tuma Wolfgang Schonende Hochanreicherung von fetalen Zellen aus pripherem Blut und Verwendung derselben
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JP4641705B2 (ja) 2001-04-30 2011-03-02 ザイストール セラピューティクス， インコーポレイテッド 治療的タンパク質の亜細胞標的化
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PL2279210T3 (pl) * 2008-05-07 2017-10-31 Biomarin Pharm Inc Lizosomalne peptydy kierujące i ich zastosowania
US8697654B2 (en) * 2008-12-18 2014-04-15 E I Du Pont De Nemours And Company Peptide linkers for effective multivalent peptide binding
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JP6063380B2 (ja) * 2010-06-25 2017-01-18 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 サンフィリポ症候群ｂ型の処置
US9409950B2 (en) * 2010-12-23 2016-08-09 Biogen Ma Inc. Linker peptides and polypeptides comprising same
EP2680879B1 (en) * 2011-03-04 2021-01-06 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
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
KR102262882B1 (ko) 2012-11-27 2021-06-10 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들

Also Published As

Publication number Publication date
KR20150088317A (ko) 2015-07-31
PL3115372T3 (pl) 2019-09-30
US20160039900A1 (en) 2016-02-11
HRP20181351T1 (hr) 2018-11-02
SI3115372T1 (sl) 2019-08-30
AU2013352184B2 (en) 2018-05-31
ZA201503509B (en) 2016-11-30
IL238824B (en) 2018-11-29
US20190225666A1 (en) 2019-07-25
MX2015006644A (es) 2015-08-10
KR102262882B1 (ko) 2021-06-10
JP6831176B2 (ja) 2021-02-17
HUE043679T2 (hu) 2019-09-30
CY1122555T1 (el) 2021-01-27
RU2680581C2 (ru) 2019-02-22
KR102385392B1 (ko) 2022-04-11
US10301369B2 (en) 2019-05-28
TW201431884A (zh) 2014-08-16
EP2925776A1 (en) 2015-10-07
IL272854A (en) 2020-04-30
ES2679374T3 (es) 2018-08-24
IL238824A0 (en) 2015-06-30
US9834588B2 (en) 2017-12-05
CL2015001371A1 (es) 2015-10-09
EP3115372B1 (en) 2019-03-06
TW201827468A (zh) 2018-08-01
US9834587B2 (en) 2017-12-05
AU2013352184A1 (en) 2015-06-04
JP6913719B2 (ja) 2021-08-04
CN104822701A (zh) 2015-08-05
TWI711632B (zh) 2020-12-01
JP2016505539A (ja) 2016-02-25
TWI626250B (zh) 2018-06-11
DK3115372T3 (da) 2019-06-11
HRP20190918T1 (hr) 2019-09-20
DK2925776T3 (en) 2018-09-03
KR102521039B1 (ko) 2023-04-12
ES2729997T3 (es) 2019-11-07
IL262976A (en) 2018-12-31
AR093626A1 (es) 2015-06-17
HK1216026A1 (zh) 2016-10-07
KR20210070389A (ko) 2021-06-14
US11254725B2 (en) 2022-02-22
US9376480B2 (en) 2016-06-28
LT3115372T (lt) 2019-06-25
JP2019206556A (ja) 2019-12-05
KR20230054482A (ko) 2023-04-24
US9845346B2 (en) 2017-12-19
RU2015125491A (ru) 2017-01-10
US20220127326A1 (en) 2022-04-28
US20160031963A1 (en) 2016-02-04
WO2014085621A1 (en) 2014-06-05
PT3115372T (pt) 2019-06-12
CA2892146A1 (en) 2014-06-05
PT2925776T (pt) 2018-07-30
US20160031965A1 (en) 2016-02-04
EP3115372A1 (en) 2017-01-11
US20160031964A1 (en) 2016-02-04
CN104822701B (zh) 2018-09-21
PL2925776T3 (pl) 2018-11-30
KR20220047892A (ko) 2022-04-19
EP2925776B1 (en) 2018-05-30
US20170355744A1 (en) 2017-12-14
US20140161788A1 (en) 2014-06-12
RS58916B1 (sr) 2019-08-30
US9771408B2 (en) 2017-09-26
MX2019009191A (es) 2019-10-09
MX367024B (es) 2019-08-02
IL262976B (en) 2020-02-27
IL272854B (en) 2021-07-29
BR112015012152B1 (pt) 2023-04-25
BR112015012152A2 (pt) 2017-08-15

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