EA201790178A1 - Пептиды, несущие маннозо-6-фосфат, слитые с лизосомальным ферментом - Google Patents

Пептиды, несущие маннозо-6-фосфат, слитые с лизосомальным ферментом

Info

Publication number
EA201790178A1
EA201790178A1 EA201790178A EA201790178A EA201790178A1 EA 201790178 A1 EA201790178 A1 EA 201790178A1 EA 201790178 A EA201790178 A EA 201790178A EA 201790178 A EA201790178 A EA 201790178A EA 201790178 A1 EA201790178 A1 EA 201790178A1
Authority
EA
Eurasian Patent Office
Prior art keywords
enzyme
lizosomal
fused
phosphate
therapeutic agent
Prior art date
Application number
EA201790178A
Other languages
English (en)
Russian (ru)
Inventor
Майкл Ф. Консино
Беттина Стрэк-Лог
Мутураман Мейяппан
Энджела В. Нортон
Бохун Чжан
Андреа Искендериан
Ле Юн Лоу
Дианна Лундберг
Алла Ромашко
Хишам Наими
Original Assignee
Шир Хьюман Дженетик Терапис, Инк.
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 Шир Хьюман Дженетик Терапис, Инк. filed Critical Шир Хьюман Дженетик Терапис, Инк.
Publication of EA201790178A1 publication Critical patent/EA201790178A1/ru

Links

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    • 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
    • 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
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/20Interleukins [IL]
    • A61K38/204IL-6
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22Hormones
    • A61K38/30Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4703Inhibitors; Suppressors
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4705Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/54Interleukins [IL]
    • C07K14/5412IL-6
    • 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
    • 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
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1288Transferases for other substituted phosphate groups (2.7.8)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/08Transferases for other substituted phosphate groups (2.7.8)
    • C12Y207/08017UDP-N-acetylglucosamine--lysosomal-enzyme N-acetylglucosaminephosphotransferase (2.7.8.17)
    • 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
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
EA201790178A 2014-08-11 2015-08-11 Пептиды, несущие маннозо-6-фосфат, слитые с лизосомальным ферментом EA201790178A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462036082P 2014-08-11 2014-08-11
PCT/US2015/044713 WO2016025519A1 (en) 2014-08-11 2015-08-11 Mannose-6-phosphate bearing peptides fused to lysosomal enzymes

Publications (1)

Publication Number Publication Date
EA201790178A1 true EA201790178A1 (ru) 2017-07-31

Family

ID=54035297

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201790178A EA201790178A1 (ru) 2014-08-11 2015-08-11 Пептиды, несущие маннозо-6-фосфат, слитые с лизосомальным ферментом

Country Status (10)

Country Link
US (2) US10722559B2 (cg-RX-API-DMAC7.html)
EP (1) EP3185889B1 (cg-RX-API-DMAC7.html)
JP (1) JP6623213B2 (cg-RX-API-DMAC7.html)
CN (1) CN106573039A (cg-RX-API-DMAC7.html)
AU (1) AU2015301809A1 (cg-RX-API-DMAC7.html)
BR (1) BR112017002741A2 (cg-RX-API-DMAC7.html)
CA (1) CA2956469A1 (cg-RX-API-DMAC7.html)
EA (1) EA201790178A1 (cg-RX-API-DMAC7.html)
MX (1) MX2017001898A (cg-RX-API-DMAC7.html)
WO (1) WO2016025519A1 (cg-RX-API-DMAC7.html)

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UA129740C2 (uk) 2010-06-25 2025-07-23 Такеда Фармасьютікал Компані Лімітед Застосування композиції, що включає білок арилсульфатази а (аса)
US20220133863A1 (en) * 2010-06-25 2022-05-05 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
US20200277584A1 (en) 2017-10-02 2020-09-03 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
EA202190416A1 (ru) 2018-08-02 2021-06-23 Дайн Терапьютикс, Инк. Мышечно-специфические комлексы и их применение для лечения плече-лопаточно-лицевой мышечной дистрофии
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2020132100A1 (en) * 2018-12-19 2020-06-25 The Board Of Trustees Of The Leland Stanford Junior University Bifunctional molecules for lysosomal targeting and related compositions and methods
KR20220069917A (ko) * 2019-07-02 2022-05-27 엠6피 테라퓨틱스 (스위처랜드) 엘엘씨 리소좀 축적 장애의 치료를 위한 벡터 조성물 및 이의 사용 방법
CN111455004B (zh) * 2020-02-29 2023-05-05 浙江农林大学 一种脂肪酶催化在线合成香豆素-3-羧酸-6’-o-d-甘露糖酯的方法
WO2022026906A2 (en) * 2020-07-30 2022-02-03 The Board Of Trustees Of The Leland Stanford Junior University Lysosomal targeting molecules comprising knottin peptides and related compositions and methods
WO2023288033A1 (en) 2021-07-14 2023-01-19 Lycia Therapeutics, Inc. Asgpr cell surface receptor binding compounds and conjugates
WO2023150387A1 (en) * 2022-02-07 2023-08-10 M6P Therapeutics, Inc. Compositions comprising acid alpha glucosidase and methods of use thereof
CN119183457A (zh) 2022-04-15 2024-12-24 达因疗法公司 用于治疗强直性肌营养不良的肌肉靶向复合物和制剂
WO2025238106A1 (en) * 2024-05-15 2025-11-20 M6P Therapeutics (Switzerland) GmbH Lysosome-targeting degradation fusion design

Family Cites Families (12)

* Cited by examiner, † Cited by third party
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US20040142474A1 (en) * 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
AU2003222568B2 (en) * 2002-01-11 2009-05-07 Bioasis Technologies, Inc. Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
US20110027267A1 (en) * 2007-11-09 2011-02-03 Anaphore, Inc. Fusion Proteins of Mannose Binding Lectins for Treatment of Disease
US20110223147A1 (en) * 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
WO2011163648A1 (en) * 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
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
EP2975134A3 (en) 2010-07-22 2016-02-24 Shire Human Genetic Therapies, Inc. Crystal structure of human alpha-n-acetylglucosaminidase
JP5959114B2 (ja) 2011-05-19 2016-08-02 学校法人 明治薬科大学 リン酸化糖鎖を含有する組換えヒトサポシンbタンパク質及びその用途
WO2012166653A2 (en) * 2011-05-27 2012-12-06 Callidus Biopharma, Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
RU2014117291A (ru) * 2011-10-12 2015-11-20 Синаджева Биофарма Корп. Рекомбинантный человеческий белок naglu и его применение
DK3115372T3 (da) 2012-11-27 2019-06-11 Biomarin Pharm Inc Målrettede terapeutiske lysosomale enzymfusionsproteiner og anvendelser deraf

Also Published As

Publication number Publication date
JP6623213B2 (ja) 2019-12-18
JP2017529326A (ja) 2017-10-05
WO2016025519A1 (en) 2016-02-18
EP3185889B1 (en) 2020-07-15
EP3185889A1 (en) 2017-07-05
CN106573039A (zh) 2017-04-19
BR112017002741A2 (pt) 2018-02-27
US20170232076A1 (en) 2017-08-17
CA2956469A1 (en) 2016-02-18
MX2017001898A (es) 2017-04-11
AU2015301809A1 (en) 2017-02-02
US20200376095A1 (en) 2020-12-03
US10722559B2 (en) 2020-07-28

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