AU2022214281B2 - Conjugated oligonucleotide compounds, methods of making and uses thereof - Google Patents
Conjugated oligonucleotide compounds, methods of making and uses thereofInfo
- Publication number
- AU2022214281B2 AU2022214281B2 AU2022214281A AU2022214281A AU2022214281B2 AU 2022214281 B2 AU2022214281 B2 AU 2022214281B2 AU 2022214281 A AU2022214281 A AU 2022214281A AU 2022214281 A AU2022214281 A AU 2022214281A AU 2022214281 B2 AU2022214281 B2 AU 2022214281B2
- Authority
- AU
- Australia
- Prior art keywords
- galnac
- invabasic
- day
- mfco
- linker
- Prior art date
- Legal status (The legal status 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 status listed.)
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Classifications
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
- C07H15/02—Acyclic radicals, not substituted by cyclic structures
- C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
- C07H15/08—Polyoxyalkylene derivatives
-
- 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
- A61K47/54—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 organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
- C07D207/02—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
- C07D207/30—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members
- C07D207/34—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having two double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
- C07D207/36—Oxygen or sulfur atoms
- C07D207/40—2,5-Pyrrolidine-diones
- C07D207/404—2,5-Pyrrolidine-diones with only hydrogen atoms or radicals containing only hydrogen and carbon atoms directly attached to other ring carbon atoms, e.g. succinimide
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
- C07H15/02—Acyclic radicals, not substituted by cyclic structures
- C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/313—Phosphorodithioates
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
Landscapes
- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Molecular Biology (AREA)
- Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Saccharide Compounds (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163143805P | 2021-01-30 | 2021-01-30 | |
| US63/143,805 | 2021-01-30 | ||
| US202163262311P | 2021-10-08 | 2021-10-08 | |
| US63/262,311 | 2021-10-08 | ||
| US202163271686P | 2021-10-25 | 2021-10-25 | |
| US63/271,686 | 2021-10-25 | ||
| PCT/EP2022/052067 WO2022162153A1 (en) | 2021-01-30 | 2022-01-28 | Conjugated oligonucleotide compounds, methods of making and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2022214281A1 AU2022214281A1 (en) | 2023-07-27 |
| AU2022214281B2 true AU2022214281B2 (en) | 2026-04-09 |
Family
ID=80446096
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2022214281A Active AU2022214281B2 (en) | 2021-01-30 | 2022-01-28 | Conjugated oligonucleotide compounds, methods of making and uses thereof |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US12338438B2 (https=) |
| EP (2) | EP4175965B1 (https=) |
| JP (1) | JP2024504505A (https=) |
| AU (1) | AU2022214281B2 (https=) |
| CA (1) | CA3204340A1 (https=) |
| ES (1) | ES2977721T3 (https=) |
| WO (1) | WO2022162153A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3204340A1 (en) | 2021-01-30 | 2022-08-04 | Ahmad Ali MORTAZAVI | Conjugated oligonucleotide compounds, methods of making and uses thereof |
| WO2023059948A1 (en) * | 2021-10-08 | 2023-04-13 | E-Therapeutics Plc | Nucleic acids containing abasic nucleosides |
| CN120615125A (zh) * | 2022-07-27 | 2025-09-09 | e-生物有限公司 | 核酸化合物 |
| CN120322552A (zh) * | 2022-07-27 | 2025-07-15 | e-生物有限公司 | 核酸化合物 |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014179620A1 (en) * | 2013-05-01 | 2014-11-06 | Isis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| WO2020097342A1 (en) * | 2018-11-08 | 2020-05-14 | Aligos Therapeutics, Inc. | S-antigen transport inhibiting oligonucleotide polymers and methods |
| WO2020172755A1 (en) * | 2019-02-28 | 2020-09-03 | Deep Genomics Incorporated | Ligand clusters and methods of their use and preparation |
| WO2021261992A1 (en) * | 2020-06-24 | 2021-12-30 | Sapreme Technologies B.V. | Conjugate of galnac and saponin, therapeutic composition comprising said conjugate and a galnac-oligonucleotide conjugate |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8273866B2 (en) | 2002-02-20 | 2012-09-25 | Merck Sharp & Dohme Corp. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA) |
| DE60310944T3 (de) | 2002-08-05 | 2017-08-03 | Silence Therapeutics Gmbh | Weitere neue formen von interferierende rns moleküle |
| US9150606B2 (en) | 2002-11-05 | 2015-10-06 | Isis Pharmaceuticals, Inc. | Compositions comprising alternating 2'-modified nucleosides for use in gene modulation |
| US9150605B2 (en) | 2002-11-05 | 2015-10-06 | Isis Pharmaceuticals, Inc. | Compositions comprising alternating 2′-modified nucleosides for use in gene modulation |
| KR100576357B1 (ko) | 2003-10-21 | 2006-05-03 | 삼성전자주식회사 | 레졸을 함유하는 수지용액, 이를 사용하여 형성된열경화수지막 및 이를 사용하여 열경화수지막을 형성하는방법 |
| WO2009073809A2 (en) * | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| AU2009298802A1 (en) * | 2008-09-23 | 2010-04-08 | Alnylam Pharmaceuticals, Inc. | Chemical modifications of monomers and oligonucleotides with cycloaddition |
| EP3564374A1 (en) | 2013-06-21 | 2019-11-06 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulation of target nucleic acids |
| WO2015042447A1 (en) * | 2013-09-20 | 2015-03-26 | Isis Pharmaceuticals, Inc. | Targeted therapeutic nucleosides and their use |
| US10119136B2 (en) | 2014-01-09 | 2018-11-06 | Alnylam Pharmaceuticals, Inc. | RNAi agents modified at the 4′-C position |
| WO2018013525A1 (en) * | 2016-07-11 | 2018-01-18 | Translate Bio Ma, Inc. | Nucleic acid conjugates and uses thereof |
| US11504391B1 (en) | 2016-11-23 | 2022-11-22 | Alnylam Pharmaceuticals, Inc. | Modified RNA agents with reduced off-target effect |
| TWI788312B (zh) | 2016-11-23 | 2023-01-01 | 美商阿尼拉製藥公司 | 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法 |
| WO2018112320A1 (en) | 2016-12-16 | 2018-06-21 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions |
| CN110268060B (zh) | 2017-01-10 | 2024-07-26 | 箭头药业股份有限公司 | α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法 |
| AU2018213379A1 (en) | 2017-01-30 | 2019-07-18 | Arrowhead Pharmaceuticals Inc. | Compositions and methods for inhibition of factor XII gene expression |
| EA202090720A1 (ru) | 2017-09-14 | 2020-07-07 | Янссен Байофарма, Инк. | ПРОИЗВОДНЫЕ GalNAc |
| EP3976788A1 (en) | 2019-05-30 | 2022-04-06 | Deep Genomics Incorporated | Oligonucleotide therapy for wolman disease and cholesteryl ester storage disease |
| AU2022212580A1 (en) | 2021-01-30 | 2023-07-27 | E-Therapeutics Plc | Nucleic acids containing abasic nucleotides |
| JP2024504504A (ja) | 2021-01-30 | 2024-01-31 | イー セラピューティクス パブリック リミテッド カンパニー | コンジュゲートオリゴヌクレオチド化合物、その作製方法及び使用 |
| EP4176061A1 (en) * | 2021-01-30 | 2023-05-10 | E-Therapeutics plc | Nucleic acids containing abasic nucleotides |
| CA3204340A1 (en) | 2021-01-30 | 2022-08-04 | Ahmad Ali MORTAZAVI | Conjugated oligonucleotide compounds, methods of making and uses thereof |
| CN120225673A (zh) | 2022-06-01 | 2025-06-27 | e-生物有限公司 | 表达和/或功能抑制剂 |
-
2022
- 2022-01-28 CA CA3204340A patent/CA3204340A1/en active Pending
- 2022-01-28 JP JP2023546405A patent/JP2024504505A/ja active Pending
- 2022-01-28 WO PCT/EP2022/052067 patent/WO2022162153A1/en not_active Ceased
- 2022-01-28 EP EP22704881.6A patent/EP4175965B1/en active Active
- 2022-01-28 ES ES22704881T patent/ES2977721T3/es active Active
- 2022-01-28 EP EP24160680.5A patent/EP4357334A3/en active Pending
- 2022-01-28 AU AU2022214281A patent/AU2022214281B2/en active Active
-
2023
- 2023-02-06 US US18/106,149 patent/US12338438B2/en active Active
-
2025
- 2025-05-16 US US19/211,026 patent/US20260085314A1/en active Pending
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014179620A1 (en) * | 2013-05-01 | 2014-11-06 | Isis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| WO2014179626A2 (en) * | 2013-05-01 | 2014-11-06 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating apolipoprotein c-iii expression |
| WO2020097342A1 (en) * | 2018-11-08 | 2020-05-14 | Aligos Therapeutics, Inc. | S-antigen transport inhibiting oligonucleotide polymers and methods |
| WO2020172755A1 (en) * | 2019-02-28 | 2020-09-03 | Deep Genomics Incorporated | Ligand clusters and methods of their use and preparation |
| WO2021261992A1 (en) * | 2020-06-24 | 2021-12-30 | Sapreme Technologies B.V. | Conjugate of galnac and saponin, therapeutic composition comprising said conjugate and a galnac-oligonucleotide conjugate |
Non-Patent Citations (2)
| Title |
|---|
| LI MING-HSIN ET AL: "Ligand Characteristics Important to Avidity Interactions of Multivalent Nanoparticles", BIOCONJUGATE CHEMISTRY, vol. 28, no. 6, 2017, pages 1649 - 1657 * |
| WENSKA MALGORZATA ET AL: "An activated triple bond linker enables 'click' attachment of peptides to oligonucleotides on solid support", NUCLEIC ACIDS RESEARCH, vol. 39, no. 20, 2011, pages 9047 - 9059 * |
Also Published As
| Publication number | Publication date |
|---|---|
| US20240102008A1 (en) | 2024-03-28 |
| EP4357334A3 (en) | 2024-08-07 |
| EP4175965B1 (en) | 2024-03-13 |
| US20260085314A1 (en) | 2026-03-26 |
| ES2977721T3 (es) | 2024-08-29 |
| US12338438B2 (en) | 2025-06-24 |
| AU2022214281A1 (en) | 2023-07-27 |
| EP4175965A1 (en) | 2023-05-10 |
| CA3204340A1 (en) | 2022-08-04 |
| WO2022162153A1 (en) | 2022-08-04 |
| JP2024504505A (ja) | 2024-01-31 |
| EP4357334A2 (en) | 2024-04-24 |
| EP4357334A9 (en) | 2024-06-19 |
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