LT3115372T - Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas - Google Patents

Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas

Info

Publication number
LT3115372T
LT3115372T LTEP16182420.6T LT16182420T LT3115372T LT 3115372 T LT3115372 T LT 3115372T LT 16182420 T LT16182420 T LT 16182420T LT 3115372 T LT3115372 T LT 3115372T
Authority
LT
Lithuania
Prior art keywords
fusion proteins
lysosomal enzyme
targeted therapeutic
enzyme fusion
therapeutic lysosomal
Prior art date
Application number
LTEP16182420.6T
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 Pharmaceutical 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 Pharmaceutical Inc. filed Critical Biomarin Pharmaceutical Inc.
Publication of LT3115372T publication Critical patent/LT3115372T/lt

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)
LTEP16182420.6T 2012-11-27 2013-11-27 Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas LT3115372T (lt)

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
LT3115372T true LT3115372T (lt) 2019-06-25

Family

ID=49753532

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP16182420.6T LT3115372T (lt) 2012-11-27 2013-11-27 Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas

Country Status (27)

Country Link
US (8) US9376480B2 (lt)
EP (2) EP2925776B1 (lt)
JP (2) JP6831176B2 (lt)
KR (4) KR20230054482A (lt)
CN (1) CN104822701B (lt)
AR (1) AR093626A1 (lt)
AU (1) AU2013352184B2 (lt)
BR (1) BR112015012152B1 (lt)
CA (1) CA2892146A1 (lt)
CL (1) CL2015001371A1 (lt)
CY (1) CY1122555T1 (lt)
DK (2) DK2925776T3 (lt)
ES (2) ES2729997T3 (lt)
HK (1) HK1216026A1 (lt)
HR (2) HRP20181351T1 (lt)
HU (2) HUE039334T2 (lt)
IL (3) IL238824B (lt)
LT (1) LT3115372T (lt)
MX (2) MX367024B (lt)
PL (2) PL2925776T3 (lt)
PT (2) PT3115372T (lt)
RS (1) RS58916B1 (lt)
RU (1) RU2680581C2 (lt)
SI (1) SI3115372T1 (lt)
TW (2) TWI626250B (lt)
WO (1) WO2014085621A1 (lt)
ZA (1) ZA201503509B (lt)

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HUE034850T2 (en) 2008-05-07 2018-03-28 Biomarin Pharm Inc Lysosomal targeting peptides and their use
LT3115372T (lt) 2012-11-27 2019-06-25 Biomarin Pharmaceutical Inc. Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas
KR20240093939A (ko) * 2013-05-15 2024-06-24 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
JP6623213B2 (ja) * 2014-08-11 2019-12-18 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 リソソーム酵素に融合したマンノース−6−リン酸含有ペプチド
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
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
BR112018071156A2 (pt) 2016-04-15 2019-03-12 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo 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
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
JP2021500857A (ja) 2017-10-02 2021-01-14 デナリ セラピューティクス インコーポレイテッドDenali Therapeutics Inc. 酵素補充療法用酵素を含む融合タンパク質
WO2019173756A1 (en) * 2018-03-09 2019-09-12 Avrobio, Inc. Compositions and methods for treating parkinson's disease
SG11202010533WA (en) * 2018-04-30 2020-11-27 Amicus Therapeutics Inc Gene therapy constructs and methods of use
WO2019233842A1 (en) 2018-06-08 2019-12-12 F. Hoffmann-La Roche Ag Peptidic linker with reduced post-translational modification
CN113631182A (zh) 2018-10-10 2021-11-09 阿米库斯治疗学公司 二硫键稳定的多肽组合物和使用方法
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
CA3154189A1 (en) * 2019-10-10 2021-04-15 Hung Do Variant igf2 constructs
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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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
IL158623A0 (en) * 2001-04-30 2004-05-12 Symbiontics Inc Subcellular targeting of therapeutic proteins
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Also Published As

Publication number Publication date
JP6831176B2 (ja) 2021-02-17
BR112015012152B1 (pt) 2023-04-25
IL238824A0 (en) 2015-06-30
TW201827468A (zh) 2018-08-01
RS58916B1 (sr) 2019-08-30
CL2015001371A1 (es) 2015-10-09
KR20150088317A (ko) 2015-07-31
PL3115372T3 (pl) 2019-09-30
KR102262882B1 (ko) 2021-06-10
US11254725B2 (en) 2022-02-22
AR093626A1 (es) 2015-06-17
US9845346B2 (en) 2017-12-19
RU2680581C2 (ru) 2019-02-22
CN104822701A (zh) 2015-08-05
US9834588B2 (en) 2017-12-05
IL272854A (en) 2020-04-30
CY1122555T1 (el) 2021-01-27
JP6913719B2 (ja) 2021-08-04
KR102385392B1 (ko) 2022-04-11
TWI711632B (zh) 2020-12-01
PT2925776T (pt) 2018-07-30
WO2014085621A1 (en) 2014-06-05
BR112015012152A2 (pt) 2017-08-15
US9834587B2 (en) 2017-12-05
DK2925776T3 (en) 2018-09-03
JP2019206556A (ja) 2019-12-05
US20170355744A1 (en) 2017-12-14
IL262976B (en) 2020-02-27
US20140161788A1 (en) 2014-06-12
IL262976A (en) 2018-12-31
HUE043679T2 (hu) 2019-09-30
HRP20190918T1 (hr) 2019-09-20
HK1216026A1 (zh) 2016-10-07
DK3115372T3 (da) 2019-06-11
CA2892146A1 (en) 2014-06-05
TWI626250B (zh) 2018-06-11
EP3115372A1 (en) 2017-01-11
KR102521039B1 (ko) 2023-04-12
KR20230054482A (ko) 2023-04-24
IL238824B (en) 2018-11-29
RU2015125491A (ru) 2017-01-10
US10301369B2 (en) 2019-05-28
AU2013352184A1 (en) 2015-06-04
ES2729997T3 (es) 2019-11-07
EP2925776B1 (en) 2018-05-30
CN104822701B (zh) 2018-09-21
US20190225666A1 (en) 2019-07-25
US20160039900A1 (en) 2016-02-11
MX367024B (es) 2019-08-02
KR20210070389A (ko) 2021-06-14
SI3115372T1 (sl) 2019-08-30
IL272854B (en) 2021-07-29
US9376480B2 (en) 2016-06-28
TW201431884A (zh) 2014-08-16
US9771408B2 (en) 2017-09-26
JP2016505539A (ja) 2016-02-25
PL2925776T3 (pl) 2018-11-30
MX2019009191A (es) 2019-10-09
ES2679374T3 (es) 2018-08-24
EP2925776A1 (en) 2015-10-07
US20160031963A1 (en) 2016-02-04
US20160031965A1 (en) 2016-02-04
EP3115372B1 (en) 2019-03-06
ZA201503509B (en) 2016-11-30
US20220127326A1 (en) 2022-04-28
US20160031964A1 (en) 2016-02-04
MX2015006644A (es) 2015-08-10
HUE039334T2 (hu) 2018-12-28
AU2013352184B2 (en) 2018-05-31
PT3115372T (pt) 2019-06-12
HRP20181351T1 (hr) 2018-11-02
KR20220047892A (ko) 2022-04-19

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