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)
Hungarian (hu)
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

    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
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
HUE13802834A HUE039334T2 (hu) 2012-11-27 2013-11-27 Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai
HUE16182420A HUE043679T2 (hu) 2012-11-27 2013-11-27 Célra irányított terápiás lizoszomális enzim fúziós fehérjék és alkalmazásaik

Family Applications After (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 (26)

Country Link
US (8) US9376480B2 (enExample)
EP (2) EP2925776B1 (enExample)
JP (2) JP6831176B2 (enExample)
KR (4) KR102262882B1 (enExample)
CN (1) CN104822701B (enExample)
AR (1) AR093626A1 (enExample)
AU (1) AU2013352184B2 (enExample)
BR (1) BR112015012152B1 (enExample)
CA (1) CA2892146A1 (enExample)
CL (1) CL2015001371A1 (enExample)
CY (1) CY1122555T1 (enExample)
DK (2) DK2925776T3 (enExample)
ES (2) ES2679374T3 (enExample)
HR (2) HRP20181351T1 (enExample)
HU (2) HUE039334T2 (enExample)
IL (3) IL238824B (enExample)
LT (1) LT3115372T (enExample)
MX (2) MX377150B (enExample)
PL (2) PL2925776T3 (enExample)
PT (2) PT3115372T (enExample)
RS (1) RS58916B1 (enExample)
RU (1) RU2680581C2 (enExample)
SI (1) SI3115372T1 (enExample)
TW (2) TWI711632B (enExample)
WO (1) WO2014085621A1 (enExample)
ZA (1) ZA201503509B (enExample)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
RU2680581C2 (ru) 2012-11-27 2019-02-22 Байомарин Фармасьютикал Инк. Направленные терапевтические химерные белки лизосомальных ферментов и их применение
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
AU2015301809A1 (en) * 2014-08-11 2017-02-02 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
US10556015B2 (en) 2014-10-24 2020-02-11 Criteo S.A. 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
KR20180025865A (ko) 2015-07-06 2018-03-09 리제너론 파마슈티칼스 인코포레이티드 다중특이적 항원 결합 분자 및 이의 용도
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
IL261246B2 (en) * 2016-02-24 2023-03-01 Biomarin Pharm Inc Therapeutic fusion proteins targeting lysosomal enzymes, their formulations and their uses
WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis 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
CN110809583A (zh) 2017-06-07 2020-02-18 瑞泽恩制药公司 用于内化酶的组合物和方法
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
BR112020005271A2 (pt) 2017-10-02 2020-09-15 Denali Therapeutics Inc. proteína, polipeptídeo, polinucleotídeo, vetor, célula hospedeira, método para produção de um polipeptídeo, métodos de tratamento de um distúrbio, de diminuição do acúmulo, de monitoramento, de transporte de um agente e de tratamento de um lsd e composição farmacêutica
KR20200118151A (ko) 2018-02-07 2020-10-14 리제너론 파마슈티칼스 인코포레이티드 치료 단백질 전달을 위한 방법 및 조성물
WO2019173756A1 (en) * 2018-03-09 2019-09-12 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
MX2020012350A (es) 2018-05-17 2021-01-29 Regeneron Pharma Anticuerpos anti-cd63, conjugados y usos de estos.
EP3802619A1 (en) 2018-06-08 2021-04-14 F. Hoffmann-La Roche AG Peptidic linker with reduced post-translational modification
EP3863663A2 (en) 2018-10-10 2021-08-18 Amicus Therapeutics, Inc. Disulfide bond stabilized polypeptide compositions and methods of use
AU2019381803A1 (en) * 2018-11-16 2021-06-10 AskBio Inc. Vectors comprising a nucleic acid encoding lysosomal enzymes fused to a lysosomal targeting sequence
BR112022006842A2 (pt) * 2019-10-10 2022-07-05 Amicus Therapeutics Inc Construtos de igf2 variante
WO2021133822A1 (en) 2019-12-24 2021-07-01 Juvena Therapeutics,Inc. Regenerative polypeptides and uses thereof
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
EP4359425A4 (en) 2021-06-21 2025-04-09 Juvena Therapeutics, Inc. REGENERATIVE POLYPEPTIDES AND THEIR USES
WO2022271981A2 (en) 2021-06-23 2022-12-29 Lycia Therapeutics, Inc. Bifunctional compounds containing igf-2 polypeptides
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
US20250339384A1 (en) * 2023-01-27 2025-11-06 Regeneron Pharmaceuticals, Inc. Modified rhabdovirus glycoproteins and uses thereof

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
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
ATE384736T1 (de) 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
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
WO2003032913A2 (en) 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting proteins across the blood brain barrier
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
EP1514106A4 (en) 2002-05-29 2007-05-09 Zystor Therapeutics Inc TARGETED THERAPEUTIC PROTEINS
CA2528591C (en) 2003-06-12 2013-01-08 Eli Lilly And Company Glp-1 analog fusion proteins
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
ES2344302T3 (es) * 2004-02-10 2010-08-24 Zystor Therapeutics , Inc. Alfa glucosidasa acida y fragmentos de la misma.
EP1869084A2 (en) 2005-03-04 2007-12-26 Biogen Idec MA Inc. Methods of humanizing immunoglobulin variable regions through rational modification of complementarity determining residues
US20110223147A1 (en) * 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
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
US20110318327A1 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
EP2585385B1 (en) 2010-06-25 2019-12-18 Amcor Rigid Plastics USA, LLC Oxygen scavenging system for a container
US9409950B2 (en) * 2010-12-23 2016-08-09 Biogen Ma Inc. Linker peptides and polypeptides comprising same
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
CA3080181A1 (en) * 2011-03-04 2012-09-13 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
US9409958B2 (en) * 2011-03-10 2016-08-09 Novozymes, Inc. Polypeptides having cellulolytic enhancing activity and polynucleotides encoding same
RU2680581C2 (ru) 2012-11-27 2019-02-22 Байомарин Фармасьютикал Инк. Направленные терапевтические химерные белки лизосомальных ферментов и их применение

Also Published As

Publication number Publication date
KR20210070389A (ko) 2021-06-14
CL2015001371A1 (es) 2015-10-09
KR102521039B1 (ko) 2023-04-12
BR112015012152B1 (pt) 2023-04-25
US9845346B2 (en) 2017-12-19
US20220127326A1 (en) 2022-04-28
RU2015125491A (ru) 2017-01-10
IL272854B (en) 2021-07-29
AU2013352184A1 (en) 2015-06-04
US20140161788A1 (en) 2014-06-12
US9771408B2 (en) 2017-09-26
JP2016505539A (ja) 2016-02-25
IL262976A (en) 2018-12-31
SI3115372T1 (sl) 2019-08-30
IL262976B (en) 2020-02-27
WO2014085621A1 (en) 2014-06-05
TW201827468A (zh) 2018-08-01
AU2013352184B2 (en) 2018-05-31
US20190225666A1 (en) 2019-07-25
US20170355744A1 (en) 2017-12-14
KR20220047892A (ko) 2022-04-19
TWI711632B (zh) 2020-12-01
EP3115372A1 (en) 2017-01-11
US11254725B2 (en) 2022-02-22
PT2925776T (pt) 2018-07-30
MX2015006644A (es) 2015-08-10
KR102385392B1 (ko) 2022-04-11
CY1122555T1 (el) 2021-01-27
HK1216026A1 (en) 2016-10-07
US20160031963A1 (en) 2016-02-04
IL272854A (en) 2020-04-30
JP6831176B2 (ja) 2021-02-17
ZA201503509B (en) 2016-11-30
IL238824B (en) 2018-11-29
EP2925776A1 (en) 2015-10-07
DK2925776T3 (en) 2018-09-03
US10301369B2 (en) 2019-05-28
DK3115372T3 (da) 2019-06-11
HUE043679T2 (hu) 2019-09-30
CN104822701A (zh) 2015-08-05
RU2680581C2 (ru) 2019-02-22
RS58916B1 (sr) 2019-08-30
HRP20190918T1 (hr) 2019-09-20
US9834587B2 (en) 2017-12-05
MX367024B (es) 2019-08-02
BR112015012152A2 (pt) 2017-08-15
EP2925776B1 (en) 2018-05-30
CN104822701B (zh) 2018-09-21
AR093626A1 (es) 2015-06-17
MX2019009191A (es) 2019-10-09
CA2892146A1 (en) 2014-06-05
HRP20181351T1 (hr) 2018-11-02
US20160039900A1 (en) 2016-02-11
US20160031964A1 (en) 2016-02-04
ES2679374T3 (es) 2018-08-24
US20160031965A1 (en) 2016-02-04
ES2729997T3 (es) 2019-11-07
TW201431884A (zh) 2014-08-16
JP6913719B2 (ja) 2021-08-04
TWI626250B (zh) 2018-06-11
MX377150B (es) 2025-03-07
KR20150088317A (ko) 2015-07-31
PL3115372T3 (pl) 2019-09-30
KR102262882B1 (ko) 2021-06-10
EP3115372B1 (en) 2019-03-06
LT3115372T (lt) 2019-06-25
PT3115372T (pt) 2019-06-12
PL2925776T3 (pl) 2018-11-30
US9376480B2 (en) 2016-06-28
US9834588B2 (en) 2017-12-05
IL238824A0 (en) 2015-06-30
JP2019206556A (ja) 2019-12-05
KR20230054482A (ko) 2023-04-24

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