KR20230152154A - 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법 - Google Patents

케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법 Download PDF

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KR20230152154A
KR20230152154A KR1020237035500A KR20237035500A KR20230152154A KR 20230152154 A KR20230152154 A KR 20230152154A KR 1020237035500 A KR1020237035500 A KR 1020237035500A KR 20237035500 A KR20237035500 A KR 20237035500A KR 20230152154 A KR20230152154 A KR 20230152154A
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double
nucleotides
rnai agent
stranded rnai
nucleotide
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Korean (ko)
Inventor
케빈 피츠제럴드
브라이언 베텐코트
그레고리 힝클
제니퍼 윌러비
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알닐람 파마슈티칼스 인코포레이티드
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Application filed by 알닐람 파마슈티칼스 인코포레이티드 filed Critical 알닐람 파마슈티칼스 인코포레이티드
Publication of KR20230152154A publication Critical patent/KR20230152154A/ko
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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
    • A—HUMAN NECESSITIES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
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    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
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    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • 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
    • A61P3/06—Antihyperlipidemics
    • 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
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01003—Ketohexokinase (2.7.1.3)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Biotechnology (AREA)
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  • Hematology (AREA)
  • Obesity (AREA)
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  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Emergency Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Pain & Pain Management (AREA)
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KR1020237035500A 2014-02-11 2015-02-11 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법 Pending KR20230152154A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201461938567P 2014-02-11 2014-02-11
US61/938,567 2014-02-11
PCT/US2015/015367 WO2015123264A1 (en) 2014-02-11 2015-02-11 Ketohexokinase (khk) irna compositions and methods of use thereof
KR1020227013057A KR102592370B1 (ko) 2014-02-11 2015-02-11 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법

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KR1020227013057A Division KR102592370B1 (ko) 2014-02-11 2015-02-11 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법

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KR20230152154A true KR20230152154A (ko) 2023-11-02

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KR1020237035500A Pending KR20230152154A (ko) 2014-02-11 2015-02-11 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법
KR1020227013057A Active KR102592370B1 (ko) 2014-02-11 2015-02-11 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법
KR1020167024701A Active KR102389968B1 (ko) 2014-02-11 2015-02-11 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법

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KR1020167024701A Active KR102389968B1 (ko) 2014-02-11 2015-02-11 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법

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US (5) US10370666B2 (cg-RX-API-DMAC7.html)
EP (2) EP3105331B1 (cg-RX-API-DMAC7.html)
JP (4) JP6594902B2 (cg-RX-API-DMAC7.html)
KR (3) KR20230152154A (cg-RX-API-DMAC7.html)
CN (3) CN118845818A (cg-RX-API-DMAC7.html)
AU (3) AU2015217301A1 (cg-RX-API-DMAC7.html)
CA (1) CA2938857A1 (cg-RX-API-DMAC7.html)
EA (1) EA201691587A1 (cg-RX-API-DMAC7.html)
WO (1) WO2015123264A1 (cg-RX-API-DMAC7.html)

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JP2020534285A (ja) 2017-09-15 2020-11-26 アンパサンド バイオファーマシューティカルズ インコーポレイテッドＡｍｐｅｒｓａｎｄ Ｂｉｏｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃ． 投与および処置の方法
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CA3105385A1 (en) * 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
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TW202526019A (zh) * 2019-11-13 2025-07-01 美商阿尼拉製藥公司 用於治療血管收縮素原相關病症之方法及組成物
JP2023516095A (ja) * 2020-03-06 2023-04-17 アルナイラム ファーマシューティカルズ， インコーポレイテッド ケトヘキソキナーゼ（ＫＨＫ）ｉＲＮＡ組成物およびその使用方法
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TW202430191A (zh) 2022-10-11 2024-08-01 德商百靈佳殷格翰國際股份有限公司 用於治療伴有晚期纖維化及/或硬化之nash的方法
WO2024175062A1 (zh) * 2023-02-23 2024-08-29 大睿生物医药科技(上海)有限公司 调控KHK基因活性的dsRNA分子
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