RS56913B1 - Postupci za kuplovanje peptida za ciljanje na rekombinantne lizosomalne encime za poboljšanje tretmana lizosomalnih bolesti taloženja - Google Patents

Postupci za kuplovanje peptida za ciljanje na rekombinantne lizosomalne encime za poboljšanje tretmana lizosomalnih bolesti taloženja

Info

Publication number
RS56913B1
RS56913B1 RS20180188A RSP20180188A RS56913B1 RS 56913 B1 RS56913 B1 RS 56913B1 RS 20180188 A RS20180188 A RS 20180188A RS P20180188 A RSP20180188 A RS P20180188A RS 56913 B1 RS56913 B1 RS 56913B1
Authority
RS
Serbia
Prior art keywords
recombinant human
modified
peptide
lysosomal enzyme
igf
Prior art date
Application number
RS20180188A
Other languages
English (en)
Serbian (sr)
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 RS56913B1 publication Critical patent/RS56913B1/sr

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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)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (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)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
RS20180188A 2011-05-27 2012-05-25 Postupci za kuplovanje peptida za ciljanje na rekombinantne lizosomalne encime za poboljšanje tretmana lizosomalnih bolesti taloženja RS56913B1 (sr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161490957P 2011-05-27 2011-05-27
PCT/US2012/039705 WO2012166653A2 (en) 2011-05-27 2012-05-25 Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
EP12793015.4A EP2714752B1 (en) 2011-05-27 2012-05-25 Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases

Publications (1)

Publication Number Publication Date
RS56913B1 true RS56913B1 (sr) 2018-05-31

Family

ID=47260229

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20180188A RS56913B1 (sr) 2011-05-27 2012-05-25 Postupci za kuplovanje peptida za ciljanje na rekombinantne lizosomalne encime za poboljšanje tretmana lizosomalnih bolesti taloženja

Country Status (21)

Country Link
US (2) US9545450B2 (enExample)
EP (1) EP2714752B1 (enExample)
JP (2) JP6300720B2 (enExample)
KR (2) KR20190022943A (enExample)
CN (2) CN104160033B (enExample)
BR (1) BR112013030432A2 (enExample)
CA (1) CA2836318C (enExample)
CY (1) CY1120485T1 (enExample)
DK (1) DK2714752T3 (enExample)
ES (1) ES2660185T3 (enExample)
HR (1) HRP20180291T1 (enExample)
HU (1) HUE038172T2 (enExample)
LT (1) LT2714752T (enExample)
NO (1) NO2820016T3 (enExample)
PL (1) PL2714752T3 (enExample)
PT (1) PT2714752T (enExample)
RS (1) RS56913B1 (enExample)
SI (1) SI2714752T1 (enExample)
SM (1) SMT201800101T1 (enExample)
TR (1) TR201802230T4 (enExample)
WO (1) WO2012166653A2 (enExample)

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AU2015301809A1 (en) * 2014-08-11 2017-02-02 Shire Human Genetic Therapies, Inc. Mannose-6-phosphate bearing peptides fused to lysosomal enzymes
SI4273241T1 (sl) * 2014-09-30 2025-03-31 Amicus Therapeutics, Inc. Zelo močna kislinska alfa-glukozidaza z okrepljenimi ogljikovimi hidrati
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
US10525495B2 (en) * 2015-03-11 2020-01-07 Ocv Intellectual Capital, Llc Methods and systems for filling mufflers with fibrous material
CN108474788A (zh) * 2015-11-06 2018-08-31 生物马林药物股份有限公司 用于检测中和溶酶体酶的摄取的抗体或其它因子的基于细胞的测定
DK3782639T3 (da) * 2015-12-08 2022-08-29 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
MA50546A (fr) 2017-06-07 2020-09-16 Regeneron Pharma Compositions et méthodes pour l'internalisation d'enzymes
KR20200018483A (ko) 2017-06-14 2020-02-19 비로메틱스 아게 호흡기 합포체 바이러스에 대한 방어를 위한 고리형 펩티드
WO2019070577A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. FUSION PROTEINS COMPRISING SUBSTITUTE ENZYM THERAPY ENZYMES
AU2019218892B2 (en) 2018-02-07 2025-08-14 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
TWI808169B (zh) 2018-04-30 2023-07-11 美商阿米庫斯醫療股份有限公司 基因治療構築體及使用方法
AU2019269685B2 (en) 2018-05-17 2025-12-04 Regeneron Pharmaceuticals, Inc. Anti-CD63 antibodies, conjugates, and uses thereof
EA202190999A1 (ru) 2018-10-10 2021-10-15 Амикус Терапьютикс, Инк. Композиции на основе полипептидов, стабилизированных с помощью дисульфидной связи, и способы применения
US11175228B2 (en) * 2018-11-28 2021-11-16 Promega Corporation Reactive peptide labeling
CA3123086A1 (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
AU2019408048A1 (en) 2018-12-20 2021-06-24 Universität Zürich Lipopeptide building blocks and synthetic virus-like particles
US20220193207A1 (en) * 2019-04-30 2022-06-23 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of pompe disease
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
EP4041284A4 (en) * 2019-10-10 2023-10-18 Amicus Therapeutics, Inc. VARIANT OF IGF2 CONSTRUCTS
WO2022063990A1 (en) 2020-09-28 2022-03-31 Dbv Technologies Particle comprising an rsv-f protein for use in rsv vaccination
JP2023551903A (ja) 2020-12-01 2023-12-13 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 組織特異的標的化モチーフを有する新規組成物及びそれを含有する組成物
MX2023012513A (es) 2021-04-23 2023-12-15 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen.
EP4359430A4 (en) 2021-06-23 2025-07-16 Lycia Therapeutics Inc BIFUNCTIONAL COMPOUNDS CONTAINING IGF-2 POLYPEPTIDES
WO2023288033A1 (en) 2021-07-14 2023-01-19 Lycia Therapeutics, Inc. Asgpr cell surface receptor binding compounds and conjugates
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
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
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Publication number Publication date
BR112013030432A2 (pt) 2016-12-13
KR101955054B1 (ko) 2019-03-07
CA2836318C (en) 2018-11-27
PL2714752T3 (pl) 2018-04-30
US20140302001A1 (en) 2014-10-09
JP6300720B2 (ja) 2018-03-28
HRP20180291T1 (hr) 2018-03-23
US20170319710A1 (en) 2017-11-09
JP2017225444A (ja) 2017-12-28
CY1120485T1 (el) 2019-07-10
EP2714752B1 (en) 2017-11-22
CN104160033B (zh) 2018-06-01
ES2660185T3 (es) 2018-03-21
WO2012166653A2 (en) 2012-12-06
PT2714752T (pt) 2018-02-26
HUE038172T2 (hu) 2018-09-28
KR20140033155A (ko) 2014-03-17
WO2012166653A3 (en) 2014-05-01
JP2014518063A (ja) 2014-07-28
CA2836318A1 (en) 2012-12-06
NO2820016T3 (enExample) 2017-12-30
EP2714752A4 (en) 2015-08-26
DK2714752T3 (en) 2018-02-26
CN108586621A (zh) 2018-09-28
EP2714752A2 (en) 2014-04-09
SMT201800101T1 (it) 2018-03-08
TR201802230T4 (tr) 2018-03-21
US10660972B2 (en) 2020-05-26
CN104160033A (zh) 2014-11-19
SI2714752T1 (en) 2018-03-30
KR20190022943A (ko) 2019-03-06
LT2714752T (lt) 2018-03-12
US9545450B2 (en) 2017-01-17

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