SI3204015T1 - Sestavki in postopki za zaviranje genskega izražanja HAO1 (hidroksiacid oksidaze 1 (glikolat oksidaze)) - Google Patents

Sestavki in postopki za zaviranje genskega izražanja HAO1 (hidroksiacid oksidaze 1 (glikolat oksidaze))

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Publication number
SI3204015T1
SI3204015T1 SI201531766T SI201531766T SI3204015T1 SI 3204015 T1 SI3204015 T1 SI 3204015T1 SI 201531766 T SI201531766 T SI 201531766T SI 201531766 T SI201531766 T SI 201531766T SI 3204015 T1 SI3204015 T1 SI 3204015T1
Authority
SI
Slovenia
Prior art keywords
oxidase
hao1
inhibition
compositions
methods
Prior art date
Application number
SI201531766T
Other languages
English (en)
Slovenian (sl)
Inventor
William Querbes
Kevin Fitzgerald
Brian Bettencourt
Abigail Liebow
David V. Erbe
Original Assignee
Alnylam Pharmaceuticals, 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 Alnylam Pharmaceuticals, Inc., filed Critical Alnylam Pharmaceuticals, Inc.,
Publication of SI3204015T1 publication Critical patent/SI3204015T1/sl

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
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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
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    • 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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    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C12Y101/03—Oxidoreductases acting on the CH-OH group of donors (1.1) with a oxygen as acceptor (1.1.3)
    • C12Y101/03015—(S)-2-Hydroxy-acid oxidase (1.1.3.15)
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Physics & Mathematics (AREA)
  • Urology & Nephrology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
SI201531766T 2014-10-10 2015-10-09 Sestavki in postopki za zaviranje genskega izražanja HAO1 (hidroksiacid oksidaze 1 (glikolat oksidaze)) SI3204015T1 (sl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201462062751P 2014-10-10 2014-10-10
US201562147976P 2015-04-15 2015-04-15
US201562214602P 2015-09-04 2015-09-04
PCT/US2015/054881 WO2016057893A1 (en) 2014-10-10 2015-10-09 Compositions and methods for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
EP15848512.8A EP3204015B1 (en) 2014-10-10 2015-10-09 Compositions and methods for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression

Publications (1)

Publication Number Publication Date
SI3204015T1 true SI3204015T1 (sl) 2022-04-29

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SI201531766T SI3204015T1 (sl) 2014-10-10 2015-10-09 Sestavki in postopki za zaviranje genskega izražanja HAO1 (hidroksiacid oksidaze 1 (glikolat oksidaze))

Country Status (42)

Country Link
US (4) US10478500B2 (enExample)
EP (2) EP3204015B1 (enExample)
JP (3) JP6836988B2 (enExample)
KR (2) KR20230113654A (enExample)
CN (2) CN113599389B (enExample)
AU (3) AU2015330726B9 (enExample)
BR (1) BR112017006469A2 (enExample)
CA (2) CA3187050A1 (enExample)
CO (1) CO2017004728A2 (enExample)
CR (1) CR20170190A (enExample)
CY (2) CY1125364T1 (enExample)
DK (1) DK3204015T3 (enExample)
DO (1) DOP2017000094A (enExample)
EA (1) EA037115B1 (enExample)
EC (1) ECSP17028310A (enExample)
ES (1) ES2905889T3 (enExample)
FI (1) FIC20220011I1 (enExample)
HR (1) HRP20211971T1 (enExample)
HU (2) HUE057327T2 (enExample)
IL (3) IL293229B2 (enExample)
JO (2) JOP20200115A1 (enExample)
LT (2) LT3204015T (enExample)
LU (1) LUC00252I2 (enExample)
MX (2) MX382225B (enExample)
MY (1) MY192926A (enExample)
NL (1) NL301167I2 (enExample)
NO (1) NO2022009I1 (enExample)
NZ (1) NZ730404A (enExample)
PE (1) PE20171763A1 (enExample)
PH (1) PH12017500669A1 (enExample)
PL (1) PL3204015T3 (enExample)
PT (1) PT3204015T (enExample)
RS (1) RS62854B1 (enExample)
SG (2) SG11201702836PA (enExample)
SI (1) SI3204015T1 (enExample)
SM (1) SMT202200026T1 (enExample)
TN (1) TN2017000125A1 (enExample)
TW (4) TW202342749A (enExample)
UA (1) UA126266C2 (enExample)
UY (1) UY36357A (enExample)
WO (1) WO2016057893A1 (enExample)
ZA (1) ZA201702071B (enExample)

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TWI851574B (zh) * 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
CN113454222B (zh) * 2018-12-19 2025-01-14 阿尼拉制药公司 淀粉样前体蛋白(APP)RNAi剂组成物及其使用方法
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TW202237150A (zh) 2020-12-01 2022-10-01 美商艾拉倫製藥股份有限公司 用於抑制ｈａｏ１（羥基酸氧化酶１（乙醇酸氧化酶））基因表現的方法及組成物
EP4314293A1 (en) * 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
JP2024522272A (ja) 2021-06-04 2024-06-13 ニューミルナ セラピューティクス エーピーエス アデノシンキナーゼを標的とするアンチセンスオリゴヌクレオチド
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