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MX367024B - Proteinas de fusion de enzima lisosomal terapeutica objetivo y usos de las mismas. - Google Patents

Proteinas de fusion de enzima lisosomal terapeutica objetivo y usos de las mismas.

Info

Publication number
MX367024B
MX367024B MX2015006644A MX2015006644A MX367024B MX 367024 B MX367024 B MX 367024B MX 2015006644 A MX2015006644 A MX 2015006644A MX 2015006644 A MX2015006644 A MX 2015006644A MX 367024 B MX367024 B MX 367024B
Authority
MX
Mexico
Prior art keywords
fusion proteins
peptide
targeted therapeutic
lysosomal enzyme
lysosomal
Prior art date
Application number
MX2015006644A
Other languages
English (en)
Other versions
MX2015006644A (es
Inventor
Aoyagi-Scharber Mika
Margaret Christianson Teresa
Dvorak-Ewell Melita
J Wendt Daniel
Long Shinong
Lebowitz Jonathan
Solomon Gold Daniel
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 MX2015006644A publication Critical patent/MX2015006644A/es
Publication of MX367024B publication Critical patent/MX367024B/es

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)
  • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Toxicology (AREA)
  • Microbiology (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)

Abstract

La presente invención se refiere en general a proteínas de fusión terapéutica útiles para tratar enfermedades de almacenamiento lisosomal y métodos para tratar tales enfermedades. Proteínas de fusión terapéutica ejemplares comprenden una enzima lisosomal, una porción de objetivo lisosomal, por ejemplo, un péptido IGF-II, y un péptido espaciador. También se proporcionan composiciones y métodos para tratar Mucopolisacaridosis Tipo IIIB (Síndrome de Sanfilippo B), que comprenden una proteína de fusión terapéutica objetivo que comprende alfa-Nacetilglucosaminidasa (Naglu), una porción de objetivo lisosomal, por ejemplo, un péptido IGF-II, y un péptido espaciador.
MX2015006644A 2012-11-27 2013-11-27 Proteinas de fusion de enzima lisosomal terapeutica objetivo y usos de las mismas. MX367024B (es)

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
PCT/US2013/072287 WO2014085621A1 (en) 2012-11-27 2013-11-27 Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof

Publications (2)

Publication Number Publication Date
MX2015006644A MX2015006644A (es) 2015-08-10
MX367024B true MX367024B (es) 2019-08-02

Family

ID=49753532

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2015006644A MX367024B (es) 2012-11-27 2013-11-27 Proteinas de fusion de enzima lisosomal terapeutica objetivo y usos de las mismas.
MX2019009191A MX2019009191A (es) 2012-11-27 2015-05-26 Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2019009191A MX2019009191A (es) 2012-11-27 2015-05-26 Proteinas de fusion de enzima lisosomal terapeutica objetivo y uso de las mismas.

Country Status (27)

Country Link
US (8) US9376480B2 (es)
EP (2) EP3115372B1 (es)
JP (2) JP6831176B2 (es)
KR (4) KR102385392B1 (es)
CN (1) CN104822701B (es)
AR (1) AR093626A1 (es)
AU (1) AU2013352184B2 (es)
BR (1) BR112015012152B1 (es)
CA (1) CA2892146A1 (es)
CL (1) CL2015001371A1 (es)
CY (1) CY1122555T1 (es)
DK (2) DK3115372T3 (es)
ES (2) ES2729997T3 (es)
HK (1) HK1216026A1 (es)
HR (2) HRP20181351T1 (es)
HU (2) HUE043679T2 (es)
IL (3) IL238824B (es)
LT (1) LT3115372T (es)
MX (2) MX367024B (es)
PL (2) PL2925776T3 (es)
PT (2) PT2925776T (es)
RS (1) RS58916B1 (es)
RU (1) RU2680581C2 (es)
SI (1) SI3115372T1 (es)
TW (2) TWI711632B (es)
WO (1) WO2014085621A1 (es)
ZA (1) ZA201503509B (es)

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RU2692251C2 (ru) * 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
BR112017002741A2 (pt) 2014-08-11 2018-02-27 Shire Human Genetic Therapies, Inc peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas
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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
US11262348B2 (en) 2015-11-06 2022-03-01 Biomarin Pharmaceutical Inc. Cell-based assays for detection of antibodies or other factors that neutralize uptake of lysosomal enzymes
EP4275751A3 (en) * 2016-02-24 2023-12-27 BioMarin Pharmaceutical Inc. Improved naglu fusion protein formulation
US11253612B2 (en) 2016-04-15 2022-02-22 The Trustees Of The University Of Pennsylvania 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
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
CR20200129A (es) 2017-10-02 2020-08-22 Denali Therapeutics Inc Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas
MA54792A (fr) * 2018-03-09 2021-12-01 Avrobio Inc Compositions et méthodes pour le traitement de la maladie de parkinson
CA3098674A1 (en) * 2018-04-30 2019-11-07 Amicus Therapeutics, Inc. Gene therapy constructs and methods of use
JP2021526026A (ja) 2018-06-08 2021-09-30 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft 翻訳後修飾が低減したペプチドリンカー
MX2021004188A (es) 2018-10-10 2021-09-08 Amicus Therapeutics Inc Composiciones polipeptídicas estabilizadas con enlace disulfuro y métodos de uso.
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
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
WO2022271981A2 (en) 2021-06-23 2022-12-29 Lycia Therapeutics, Inc. Bifunctional compounds containing igf-2 polypeptides
CN117343197A (zh) * 2021-10-15 2024-01-05 中山大学 重组融合抗体
WO2024159071A1 (en) * 2023-01-27 2024-08-02 Regeneron Pharmaceuticals, Inc. Modified rhabdovirus glycoproteins and uses thereof

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Also Published As

Publication number Publication date
US20170355744A1 (en) 2017-12-14
JP2016505539A (ja) 2016-02-25
US9771408B2 (en) 2017-09-26
BR112015012152A2 (pt) 2017-08-15
US9376480B2 (en) 2016-06-28
US20160031964A1 (en) 2016-02-04
TWI711632B (zh) 2020-12-01
JP6913719B2 (ja) 2021-08-04
IL262976A (en) 2018-12-31
US20140161788A1 (en) 2014-06-12
TW201431884A (zh) 2014-08-16
CY1122555T1 (el) 2021-01-27
US9834588B2 (en) 2017-12-05
BR112015012152B1 (pt) 2023-04-25
HRP20181351T1 (hr) 2018-11-02
WO2014085621A1 (en) 2014-06-05
EP2925776B1 (en) 2018-05-30
CN104822701A (zh) 2015-08-05
KR102521039B1 (ko) 2023-04-12
AU2013352184B2 (en) 2018-05-31
CN104822701B (zh) 2018-09-21
ZA201503509B (en) 2016-11-30
AR093626A1 (es) 2015-06-17
HUE043679T2 (hu) 2019-09-30
PT2925776T (pt) 2018-07-30
EP3115372A1 (en) 2017-01-11
JP6831176B2 (ja) 2021-02-17
DK3115372T3 (da) 2019-06-11
CL2015001371A1 (es) 2015-10-09
MX2015006644A (es) 2015-08-10
EP3115372B1 (en) 2019-03-06
CA2892146A1 (en) 2014-06-05
DK2925776T3 (en) 2018-09-03
LT3115372T (lt) 2019-06-25
US20220127326A1 (en) 2022-04-28
US9845346B2 (en) 2017-12-19
RU2680581C2 (ru) 2019-02-22
IL272854A (en) 2020-04-30
ES2679374T3 (es) 2018-08-24
MX2019009191A (es) 2019-10-09
IL262976B (en) 2020-02-27
US20160039900A1 (en) 2016-02-11
PT3115372T (pt) 2019-06-12
KR102262882B1 (ko) 2021-06-10
IL238824B (en) 2018-11-29
US20160031963A1 (en) 2016-02-04
US11254725B2 (en) 2022-02-22
ES2729997T3 (es) 2019-11-07
KR20220047892A (ko) 2022-04-19
AU2013352184A1 (en) 2015-06-04
PL2925776T3 (pl) 2018-11-30
US10301369B2 (en) 2019-05-28
US20160031965A1 (en) 2016-02-04
EP2925776A1 (en) 2015-10-07
IL272854B (en) 2021-07-29
TW201827468A (zh) 2018-08-01
KR20210070389A (ko) 2021-06-14
KR20230054482A (ko) 2023-04-24
HRP20190918T1 (hr) 2019-09-20
HUE039334T2 (hu) 2018-12-28
SI3115372T1 (sl) 2019-08-30
KR20150088317A (ko) 2015-07-31
PL3115372T3 (pl) 2019-09-30
US9834587B2 (en) 2017-12-05
KR102385392B1 (ko) 2022-04-11
RS58916B1 (sr) 2019-08-30
IL238824A0 (en) 2015-06-30
RU2015125491A (ru) 2017-01-10
HK1216026A1 (zh) 2016-10-07
TWI626250B (zh) 2018-06-11
JP2019206556A (ja) 2019-12-05
US20190225666A1 (en) 2019-07-25

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