JP2024522106A - 炭酸脱水酵素２発現をサイレンシングするための組成物および方法 - Google Patents

炭酸脱水酵素２発現をサイレンシングするための組成物および方法 Download PDF

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JP2024522106A
JP2024522106A JP2023573112A JP2023573112A JP2024522106A JP 2024522106 A JP2024522106 A JP 2024522106A JP 2023573112 A JP2023573112 A JP 2023573112A JP 2023573112 A JP2023573112 A JP 2023573112A JP 2024522106 A JP2024522106 A JP 2024522106A
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nucleotides
strand
dsrna
antisense strand
nucleotide
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JP2024522106A5 (https=
Inventor
ズーバー，ジェフリー
ディ マキニンチ，ジェイムズ
ケイ シュリーゲル，マーク
カストレノ，アダム
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Alnylam Pharmaceuticals Inc
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    • 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
    • C12N15/1137—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 against enzymes
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
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    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
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    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12Y402/01—Hydro-lyases (4.2.1)
    • C12Y402/01001—Carbonate dehydratase (4.2.1.1), i.e. carbonic anhydrase
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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JP2023573112A 2021-05-27 2022-05-27 炭酸脱水酵素２発現をサイレンシングするための組成物および方法 Pending JP2024522106A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163194073P 2021-05-27 2021-05-27
US63/194,073 2021-05-27
US202163289319P 2021-12-14 2021-12-14
US63/289,319 2021-12-14
PCT/US2022/031337 WO2022251629A2 (en) 2021-05-27 2022-05-27 Compositions and methods for silencing carbonic anhydrase 2 expression

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JP2024522106A true JP2024522106A (ja) 2024-06-11
JP2024522106A5 JP2024522106A5 (https=) 2025-06-04

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JP2023573112A Pending JP2024522106A (ja) 2021-05-27 2022-05-27 炭酸脱水酵素２発現をサイレンシングするための組成物および方法

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US (2) US20240254493A1 (https=)
EP (1) EP4347836A4 (https=)
JP (1) JP2024522106A (https=)
KR (1) KR20240014109A (https=)
AU (1) AU2022280088A1 (https=)
BR (1) BR112023024269A2 (https=)
CA (1) CA3220604A1 (https=)
IL (1) IL308574A (https=)
MX (1) MX2023014008A (https=)
WO (1) WO2022251629A2 (https=)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070099862A1 (en) * 2002-11-14 2007-05-03 Dharmacon Inc. siRNA targeting carbonic anhydrase II
JP2008528632A (ja) * 2005-02-01 2008-07-31 アルコン，インコーポレイテッド 眼標的のＲＮＡｉ媒介型阻害
US20100331394A1 (en) * 2004-08-23 2010-12-30 Sylentis S.A.U. Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure
US20180216114A1 (en) * 2015-07-27 2018-08-02 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2487716C2 (ru) * 2007-10-03 2013-07-20 Кварк Фармасьютикалс, Инк. Новые структуры малых интерферирующих рнк (sirna)
ES2351005B1 (es) * 2009-07-10 2011-11-18 Consejo Superior De Investigaciones Científicas (Csic) Uso de anhidrasa carbónica ii para la elaboración de un medicamento.
MX2020011570A (es) * 2018-05-07 2020-11-24 Alnylam Pharmaceuticals Inc Administracion extrahepatica.
WO2022026531A1 (en) * 2020-07-29 2022-02-03 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070099862A1 (en) * 2002-11-14 2007-05-03 Dharmacon Inc. siRNA targeting carbonic anhydrase II
US20100331394A1 (en) * 2004-08-23 2010-12-30 Sylentis S.A.U. Methods and Compositions for the Treatment of Eye Disorders with Increased Intraocular Pressure
JP2008528632A (ja) * 2005-02-01 2008-07-31 アルコン，インコーポレイテッド 眼標的のＲＮＡｉ媒介型阻害
US20180216114A1 (en) * 2015-07-27 2018-08-02 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof

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EP4347836A4 (en) 2025-11-26
EP4347836A2 (en) 2024-04-10
CA3220604A1 (en) 2022-12-01
US20240254493A1 (en) 2024-08-01
KR20240014109A (ko) 2024-01-31
WO2022251629A2 (en) 2022-12-01
BR112023024269A2 (pt) 2024-01-30
WO2022251629A3 (en) 2023-01-12
MX2023014008A (es) 2023-12-11
US20250084418A1 (en) 2025-03-13
IL308574A (en) 2024-01-01
AU2022280088A1 (en) 2023-11-23

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