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MX2017001898A - Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales. - Google Patents

Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales.

Info

Publication number
MX2017001898A
MX2017001898A MX2017001898A MX2017001898A MX2017001898A MX 2017001898 A MX2017001898 A MX 2017001898A MX 2017001898 A MX2017001898 A MX 2017001898A MX 2017001898 A MX2017001898 A MX 2017001898A MX 2017001898 A MX2017001898 A MX 2017001898A
Authority
MX
Mexico
Prior art keywords
mannose
lysosomal enzymes
bearing peptides
peptides fused
lysosomal
Prior art date
Application number
MX2017001898A
Other languages
English (en)
Inventor
Michael F Concino
Alla Romashko
Muthuraman Meiyappan
Bohong Zhang
Dianna Lundberg
W Norton Angela
Yoon LOW Lieh
NAIMY Hicham
Iskenderian Andrea
Original Assignee
Shire Human Genetic Therapies
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 Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of MX2017001898A publication Critical patent/MX2017001898A/es

Links

Classifications

    • 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
    • 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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/20—Interleukins [IL]
    • A61K38/204—IL-6
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/22—Hormones
    • A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/5412—IL-6
    • 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, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1288—Transferases for other substituted phosphate groups (2.7.8)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/08—Transferases for other substituted phosphate groups (2.7.8)
    • C12Y207/08017—UDP-N-acetylglucosamine--lysosomal-enzyme N-acetylglucosaminephosphotransferase (2.7.8.17)
    • 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
    • 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)

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)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • General Engineering & Computer Science (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Obesity (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)

Abstract

Un agente terapéutico dirigido que incluye una enzima lisosomal y una porción de direccionamiento lisosomal que es un péptido que contiene al menos un sitio de glicosilación unido a N; los métodos para producir el agente terapéutico dirigido pueden incluir ácidos nucleótidos que lo codifican y células hospedadoras que expresan GNPT de manera conjunta; composiciones farmacéuticas que comprenden el agente terapéutico dirigido y métodos para utilizarlo para tratar una enfermedad por depósito lisosomal.
MX2017001898A 2014-08-11 2015-08-11 Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales. MX2017001898A (es)

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
MX2017001898A true MX2017001898A (es) 2017-04-11

Family

ID=54035297

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2017001898A MX2017001898A (es) 2014-08-11 2015-08-11 Peptidos que contienen manosa-6-fosfato fusionados a enzimas lisosomales.

Country Status (10)

Country Link
US (2) US10722559B2 (es)
EP (1) EP3185889B1 (es)
JP (1) JP6623213B2 (es)
CN (1) CN106573039A (es)
AU (1) AU2015301809A1 (es)
BR (1) BR112017002741A2 (es)
CA (1) CA2956469A1 (es)
EA (1) EA201790178A1 (es)
MX (1) MX2017001898A (es)
WO (1) WO2016025519A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20211520T1 (hr) 2010-06-25 2021-12-24 Shire Human Genetic Therapies, Inc. Postupci i pripravci za dostavu iduronat-2-sulfataze u središnji živčani sustav
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
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
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
SG11202106209RA (en) * 2018-12-19 2021-07-29 Univ Leland Stanford Junior Bifunctional molecules for lysosomal targeting and related compositions and methods
CN114616000A (zh) * 2019-07-02 2022-06-10 M6P医疗(瑞士)有限责任公司 载体组合物及其用于治疗溶酶体贮积症的方法
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
US20230211001A1 (en) * 2020-07-30 2023-07-06 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
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
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
Publication number Priority date Publication date Assignee Title
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
JP2006506317A (ja) * 2002-01-11 2006-02-23 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 治療用リソソーム酵素の送達のための酵素送達系としてのｐ９７の使用
US20110027267A1 (en) * 2007-11-09 2011-02-03 Anaphore, Inc. Fusion Proteins of Mannose Binding Lectins for Treatment of Disease
PT2279210T (pt) * 2008-05-07 2017-07-10 Biomarin Pharm Inc Péptidos de direcionamento lisossomal e suas utilizações
NZ605871A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
DK2588130T3 (en) * 2010-06-25 2016-10-24 Shire Human Genetic Therapies Cns delivery of therapeutic agents
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
BR112013030432A2 (pt) * 2011-05-27 2016-12-13 Callidus Biopharma Inc método para preparar um peptídeo conjugado alvo para uma enzima lisossomal recombinante, método para preparar uma molécula para terapia de reposição de enzima, conjugado, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um paciente sofrendo da doença de pompe, doença de fabry, e doença de gaucher, mps i, mps ii, mps vii, tay sachs, sandhoff, alfa-manosidose, e wohlman, sequência de dna e sequência de aminoácidos
CA2852027A1 (en) 2011-10-12 2013-04-18 Synageva Biopharma Corp. Recombinant human naglu protein and uses thereof
DK2925776T3 (en) * 2012-11-27 2018-09-03 Biomarin Pharm Inc TARGETED THERAPEUTIC LYSOSOMAL ENZYMUS FUSION PROTEINS AND APPLICATIONS THEREOF

Also Published As

Publication number Publication date
JP2017529326A (ja) 2017-10-05
EP3185889A1 (en) 2017-07-05
BR112017002741A2 (pt) 2018-02-27
CN106573039A (zh) 2017-04-19
US20200376095A1 (en) 2020-12-03
AU2015301809A1 (en) 2017-02-02
US20170232076A1 (en) 2017-08-17
JP6623213B2 (ja) 2019-12-18
EA201790178A1 (ru) 2017-07-31
US10722559B2 (en) 2020-07-28
EP3185889B1 (en) 2020-07-15
CA2956469A1 (en) 2016-02-18
WO2016025519A1 (en) 2016-02-18

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