HUE038172T2 - Eljárások célzó peptidek rekombináns lizoszómális enzimekre történõ kapcsolására lizoszómális tárolási betegségek javított kezelésében - Google Patents

Eljárások célzó peptidek rekombináns lizoszómális enzimekre történõ kapcsolására lizoszómális tárolási betegségek javított kezelésében

Info

Publication number
HUE038172T2
HUE038172T2 HUE12793015A HUE12793015A HUE038172T2 HU E038172 T2 HUE038172 T2 HU E038172T2 HU E12793015 A HUE12793015 A HU E12793015A HU E12793015 A HUE12793015 A HU E12793015A HU E038172 T2 HUE038172 T2 HU E038172T2
Authority
HU
Hungary
Prior art keywords
methods
storage diseases
targeting peptides
lysosomal
improved treatments
Prior art date
Application number
HUE12793015A
Other languages
English (en)
Hungarian (hu)
Inventor
Hung Do
Original Assignee
Amicus Therapeutics 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 Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of HUE038172T2 publication Critical patent/HUE038172T2/hu

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/107—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length by chemical modification of precursor peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/68—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6847—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a hormone or a hormone-releasing or -inhibiting factor
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/642—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent the peptide or protein in the drug conjugate being a cytokine, e.g. IL2, chemokine, growth factors or interferons being the inactive part of the conjugate
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/16—Extraction; Separation; Purification by chromatography
    • C07K1/18—Ion-exchange chromatography
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/16—Extraction; Separation; Purification by chromatography
    • C07K1/22—Affinity chromatography or related techniques based upon selective absorption processes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/30—Extraction; Separation; Purification by precipitation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/30—Extraction; Separation; Purification by precipitation
    • C07K1/306—Extraction; Separation; Purification by precipitation by crystallization
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/36—Extraction; Separation; Purification by a combination of two or more processes of different types
    • 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
    • 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
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Analytical Chemistry (AREA)
  • Epidemiology (AREA)
  • Endocrinology (AREA)
  • Diabetes (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Emergency Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
HUE12793015A 2011-05-27 2012-05-25 Eljárások célzó peptidek rekombináns lizoszómális enzimekre történõ kapcsolására lizoszómális tárolási betegségek javított kezelésében HUE038172T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201161490957P 2011-05-27 2011-05-27

Publications (1)

Publication Number Publication Date
HUE038172T2 true HUE038172T2 (hu) 2018-09-28

Family

ID=47260229

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE12793015A HUE038172T2 (hu) 2011-05-27 2012-05-25 Eljárások célzó peptidek rekombináns lizoszómális enzimekre történõ kapcsolására lizoszómális tárolási betegségek javított kezelésében

Country Status (21)

Country Link
US (2) US9545450B2 (https=)
EP (1) EP2714752B1 (https=)
JP (2) JP6300720B2 (https=)
KR (2) KR20190022943A (https=)
CN (2) CN108586621A (https=)
BR (1) BR112013030432A2 (https=)
CA (1) CA2836318C (https=)
CY (1) CY1120485T1 (https=)
DK (1) DK2714752T3 (https=)
ES (1) ES2660185T3 (https=)
HR (1) HRP20180291T1 (https=)
HU (1) HUE038172T2 (https=)
LT (1) LT2714752T (https=)
NO (1) NO2820016T3 (https=)
PL (1) PL2714752T3 (https=)
PT (1) PT2714752T (https=)
RS (1) RS56913B1 (https=)
SI (1) SI2714752T1 (https=)
SM (1) SMT201800101T1 (https=)
TR (1) TR201802230T4 (https=)
WO (1) WO2012166653A2 (https=)

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EP3185889B1 (en) * 2014-08-11 2020-07-15 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
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CN104356238B (zh) * 2014-10-15 2018-01-09 大连理工大学 一种重组蛋白a亲和配基的定点固定化方法
HUE045254T2 (hu) * 2015-03-11 2019-12-30 Ocv Intellectual Capital Llc Eljárások kipufogódobok szálasanyaggal történõ megtöltésére
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
DK3386534T3 (da) * 2015-12-08 2020-11-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
DK3635009T3 (da) 2017-06-07 2026-03-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
CA3066026A1 (en) 2017-06-14 2018-12-20 Virometix Ag Cyclic peptides for protection against respiratory syncytial virus
CR20200129A (es) 2017-10-02 2020-08-22 Denali Therapeutics Inc Proteínas de fusión que comprenden enzimas de terapia de reemplazo de enzimas
US12258597B2 (en) 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
US11492610B2 (en) 2018-03-08 2022-11-08 California Institute Of Technology Sample multiplexing for single-cell RNA sequencing
BR112020021962A2 (pt) 2018-04-30 2021-01-26 Amicus Therapeutics, Inc. construtos para terapia gênica e métodos de uso
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
CN113631182B (zh) 2018-10-10 2025-03-21 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
WO2020113036A2 (en) * 2018-11-28 2020-06-04 Promega Corporation Reactive peptide labeling
AU2019404061A1 (en) 2018-12-19 2021-07-22 The Board Of Trustees Of The Leland Stanford Junior University Bifunctional molecules for lysosomal targeting and related compositions and methods
JP7550762B2 (ja) 2018-12-20 2024-09-13 ヴァイロメティックス アーゲー リポペプチドビルディングブロックおよび合成ウイルス様粒子
JP7585233B2 (ja) * 2019-04-30 2024-11-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ポンペ病の治療のために有用な組成物
CN110373405B (zh) * 2019-07-24 2023-12-15 杭州恩和生物科技有限公司 一种接枝聚合物的生物酶及其制备方法和固定方法
KR20220077921A (ko) * 2019-10-10 2022-06-09 아미쿠스 세라퓨틱스, 인코포레이티드 변이체 igf2 작제물
EP4216992A1 (en) 2020-09-28 2023-08-02 DBV Technologies Particle comprising an rsv-f protein for use in rsv vaccination
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
CA3216004A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2022271981A2 (en) 2021-06-23 2022-12-29 Lycia Therapeutics, Inc. Bifunctional compounds containing igf-2 polypeptides
EP4370157A1 (en) 2021-07-14 2024-05-22 Lycia Therapeutics, Inc. Asgpr cell surface receptor binding compounds and conjugates
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
CN121693342A (zh) 2023-07-12 2026-03-17 维罗迈科斯股份公司 肺炎球菌抗原的组合物
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JP2014518063A (ja) 2014-07-28
CY1120485T1 (el) 2019-07-10
TR201802230T4 (tr) 2018-03-21
US10660972B2 (en) 2020-05-26
EP2714752A4 (en) 2015-08-26
KR20190022943A (ko) 2019-03-06
DK2714752T3 (en) 2018-02-26
HRP20180291T1 (hr) 2018-03-23
PL2714752T3 (pl) 2018-04-30
WO2012166653A2 (en) 2012-12-06
KR101955054B1 (ko) 2019-03-07
SMT201800101T1 (it) 2018-03-08
WO2012166653A3 (en) 2014-05-01
EP2714752B1 (en) 2017-11-22
CN104160033B (zh) 2018-06-01
JP6300720B2 (ja) 2018-03-28
KR20140033155A (ko) 2014-03-17
CA2836318C (en) 2018-11-27
CA2836318A1 (en) 2012-12-06
RS56913B1 (sr) 2018-05-31
US20170319710A1 (en) 2017-11-09
EP2714752A2 (en) 2014-04-09
US20140302001A1 (en) 2014-10-09
BR112013030432A2 (pt) 2016-12-13
CN108586621A (zh) 2018-09-28
ES2660185T3 (es) 2018-03-21
CN104160033A (zh) 2014-11-19
US9545450B2 (en) 2017-01-17
JP2017225444A (ja) 2017-12-28
PT2714752T (pt) 2018-02-26
NO2820016T3 (https=) 2017-12-30
LT2714752T (lt) 2018-03-12
SI2714752T1 (en) 2018-03-30

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