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DK3204015T3 - Forbindelser og fremgangsmåder til inhibition af hao1-genekspression (hydroxysyre oxidase 1 (glycolat oxidase)) - Google Patents

Forbindelser og fremgangsmåder til inhibition af hao1-genekspression (hydroxysyre oxidase 1 (glycolat oxidase)) Download PDF

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Publication number
DK3204015T3
DK3204015T3 DK15848512.8T DK15848512T DK3204015T3 DK 3204015 T3 DK3204015 T3 DK 3204015T3 DK 15848512 T DK15848512 T DK 15848512T DK 3204015 T3 DK3204015 T3 DK 3204015T3
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Prior art keywords
oxidase
inhibitation
compounds
methods
gene expression
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DK15848512.8T
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English (en)
Inventor
William Querbes
Kevin Fitzgerald
Brian Bettencourt
Abigail Liebow
David V Erbe
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Alnylam Pharmaceuticals Inc
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    • 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
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    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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DK15848512.8T 2014-10-10 2015-10-09 Forbindelser og fremgangsmåder til inhibition af hao1-genekspression (hydroxysyre oxidase 1 (glycolat oxidase)) DK3204015T3 (da)

Applications Claiming Priority (4)

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

Publications (1)

Publication Number Publication Date
DK3204015T3 true DK3204015T3 (da) 2022-01-17

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DK15848512.8T DK3204015T3 (da) 2014-10-10 2015-10-09 Forbindelser og fremgangsmåder til inhibition af hao1-genekspression (hydroxysyre oxidase 1 (glycolat oxidase))

Country Status (40)

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US (4) US10478500B2 (da)
EP (2) EP4039809A1 (da)
JP (3) JP6836988B2 (da)
KR (2) KR20230113654A (da)
CN (2) CN108064154B (da)
AU (3) AU2015330726B9 (da)
BR (1) BR112017006469A2 (da)
CA (2) CA3187050A1 (da)
CO (1) CO2017004728A2 (da)
CR (1) CR20170190A (da)
CY (2) CY1125364T1 (da)
DK (1) DK3204015T3 (da)
DO (1) DOP2017000094A (da)
EA (1) EA037115B1 (da)
EC (1) ECSP17028310A (da)
ES (1) ES2905889T3 (da)
HR (1) HRP20211971T1 (da)
HU (2) HUE057327T2 (da)
IL (3) IL251276B (da)
JO (2) JOP20200115A1 (da)
LT (2) LT3204015T (da)
LU (1) LUC00252I2 (da)
MX (2) MX382225B (da)
MY (1) MY192926A (da)
NO (1) NO2022009I1 (da)
NZ (1) NZ730404A (da)
PE (1) PE20171763A1 (da)
PH (1) PH12017500669A1 (da)
PL (1) PL3204015T3 (da)
PT (1) PT3204015T (da)
RS (1) RS62854B1 (da)
SG (2) SG10201913756QA (da)
SI (1) SI3204015T1 (da)
SM (1) SMT202200026T1 (da)
TN (1) TN2017000125A1 (da)
TW (4) TW202342749A (da)
UA (1) UA126266C2 (da)
UY (1) UY36357A (da)
WO (1) WO2016057893A1 (da)
ZA (1) ZA201702071B (da)

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JP7277432B2 (ja) 2017-07-13 2023-05-19 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 乳酸脱水素酵素Ａ（ＬＤＨＡ）ｉＲＮＡ組成物及びその使用方法
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
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EP4065225A1 (en) * 2019-11-27 2022-10-05 DTX Pharma, Inc. Compounds and methods for the treatment of duchenne muscular dystrophy
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
EP4232581A1 (en) * 2020-10-21 2023-08-30 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
JP7642809B2 (ja) 2020-11-12 2025-03-10 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
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
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