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UY37565A - Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso - Google Patents

Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso

Info

Publication number
UY37565A
UY37565A UY0001037565A UY37565A UY37565A UY 37565 A UY37565 A UY 37565A UY 0001037565 A UY0001037565 A UY 0001037565A UY 37565 A UY37565 A UY 37565A UY 37565 A UY37565 A UY 37565A
Authority
UY
Uruguay
Prior art keywords
aat
agents
iarn
methods
compositions including
Prior art date
Application number
UY0001037565A
Other languages
English (en)
Inventor
Tao Pei
Zhen Li
Rui Zhu
I Wooddell Christine
Original Assignee
Arrowhead 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 Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of UY37565A publication Critical patent/UY37565A/es

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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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
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    • G01N2333/811—Serine protease (E.C. 3.4.21) inhibitors
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    • G01N2800/08—Hepato-biliairy disorders other than hepatitis
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  • Health & Medical Sciences (AREA)
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  • Veterinary Medicine (AREA)
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  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Cell Biology (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Se describen agentes de iARN para inhibir la expresión del gen alfa-1 antitripsina (AAT), composiciones que incluyen agente de iARN AAT y métodos para su uso, También se describen composiciones farmacéuticas que comprenden uno o más agentes de iARN AAT junto con uno o más excipientes capaces de suministrar uno o más agentes de iARN AAT a una célula hepática in vivo. El suministro de uno o más agentes de iARN AAT a células hepáticas in vivo prevé la inhibición de la expresión del gen AAT y el tratamiento de enfermedades asociadas con la deficiencia de AAT, como hepatitis crónica.
UY0001037565A 2017-01-10 2018-01-10 Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso UY37565A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762444452P 2017-01-10 2017-01-10
US201762486720P 2017-04-18 2017-04-18
US201762596232P 2017-12-08 2017-12-08

Publications (1)

Publication Number Publication Date
UY37565A true UY37565A (es) 2018-07-31

Family

ID=62782328

Family Applications (1)

Application Number Title Priority Date Filing Date
UY0001037565A UY37565A (es) 2017-01-10 2018-01-10 Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso

Country Status (15)

Country Link
US (4) US10450565B2 (es)
EP (1) EP3568482A4 (es)
JP (2) JP7258773B2 (es)
KR (2) KR20240096846A (es)
CN (1) CN110268060B (es)
AU (2) AU2018208505B2 (es)
BR (1) BR112019014282A2 (es)
CA (1) CA3045045A1 (es)
IL (2) IL310548A (es)
JO (1) JOP20180003B1 (es)
MX (2) MX2019008252A (es)
TW (1) TWI826365B (es)
UY (1) UY37565A (es)
WO (1) WO2018132432A1 (es)
ZA (3) ZA201903348B (es)

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IL290633B2 (en) * 2016-06-06 2024-11-01 Arrowhead Pharmaceuticals Inc 5'-cyclophosphonate modified nucleotides, preparations containing them and their uses
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TW202313978A (zh) 2016-11-23 2023-04-01 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
BR112019014282A2 (pt) 2017-01-10 2020-03-03 Arrowhead Pharmaceuticals, Inc. Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso
EP4219713A3 (en) 2017-04-05 2023-08-16 Silence Therapeutics GmbH Products and compositions
CN108931653A (zh) * 2018-08-31 2018-12-04 广州华澳生物科技有限公司 一种α1- 抗胰蛋白酶免疫层析检测试剂卡及其制备方法
CN113164509A (zh) * 2018-09-19 2021-07-23 箭头药业股份有限公司 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
CA3125289A1 (en) * 2018-12-28 2020-07-02 Sirnaomics, Inc. Targeted delivery of therapeutic molecules
AU2020286462A1 (en) * 2019-06-06 2022-01-27 Arrowhead Pharmaceuticals, Inc. Methods for the treatment of alpha-1 antitrypsin deficiency (AATD)
JPWO2021153687A1 (es) * 2020-01-30 2021-08-05
KR20230067648A (ko) 2020-09-15 2023-05-16 애로우헤드 파마슈티컬스 인코포레이티드 Z-aat 단백질 수준을 감소시키기 위한 방법
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
WO2022162161A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Conjugated oligonucleotide compounds, methods of making and uses thereof
WO2024062413A1 (en) * 2022-09-21 2024-03-28 Janssen Biotech, Inc. Novel stabilized nucleoside phosphates and analogues thereof
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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