ES2905257T3 - Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario - Google Patents

Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario Download PDF

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Publication number
ES2905257T3
ES2905257T3 ES18196106T ES18196106T ES2905257T3 ES 2905257 T3 ES2905257 T3 ES 2905257T3 ES 18196106 T ES18196106 T ES 18196106T ES 18196106 T ES18196106 T ES 18196106T ES 2905257 T3 ES2905257 T3 ES 2905257T3
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strand
rna
methyl rna
dsrna
antitrypsin
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ES18196106T
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English (en)
Spanish (es)
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Bob D Brown
Henryk T Dudek
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Electric Double-Layer Capacitors Or The Like (AREA)
  • Medicinal Preparation (AREA)
ES18196106T 2013-07-03 2014-07-03 Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario Active ES2905257T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361842551P 2013-07-03 2013-07-03
US201361891548P 2013-10-16 2013-10-16

Publications (1)

Publication Number Publication Date
ES2905257T3 true ES2905257T3 (es) 2022-04-07

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ES18196106T Active ES2905257T3 (es) 2013-07-03 2014-07-03 Métodos y composiciones para la inhibición específica de alfa-1 antitripsina mediante ARN bicatenario

Country Status (11)

Country Link
US (11) US9458457B2 (enExample)
EP (3) EP3017047A4 (enExample)
JP (4) JP2016528887A (enExample)
AU (2) AU2014284298B2 (enExample)
CA (1) CA2916499A1 (enExample)
DK (1) DK3444350T3 (enExample)
ES (1) ES2905257T3 (enExample)
HU (1) HUE058794T2 (enExample)
PL (1) PL3444350T3 (enExample)
PT (1) PT3444350T (enExample)
WO (1) WO2015003113A2 (enExample)

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EP3766975A1 (en) 2010-10-29 2021-01-20 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acid (sina)
KR102463973B1 (ko) 2013-05-22 2022-11-07 알닐람 파마슈티칼스 인코포레이티드 SERPINA1 iRNA 조성물 및 이의 사용 방법
EP3017047A4 (en) 2013-07-03 2017-06-14 Dicerna Pharmaceuticals Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α-1抗胰蛋白酶基因表現之組合物及方法
CN108699557B (zh) * 2015-12-04 2025-08-15 诺华股份有限公司 用于免疫肿瘤学的组合物和方法
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
SG11201809699XA (en) * 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
IL308824A (en) 2016-08-17 2024-01-01 Factor Bioscience Inc Nucleic acid products and methods of administration thereof
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
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
CA3047415A1 (en) * 2016-12-22 2018-06-28 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
UY37565A (es) 2017-01-10 2018-07-31 Arrowhead Pharmaceuticals Inc Agentes de iarn alfa-1 antitripsina (aat), composiciones que incluyen agentes de iarn aat y métodos de uso
SE1750774A1 (en) * 2017-06-19 2018-12-11 Hepgene Medical Ab Novel nucleic acid molecules and their use in therapy
CN111971051A (zh) * 2018-04-13 2020-11-20 迪克纳制药公司 用增加tm的核苷酸修饰的双链核酸抑制剂分子
EP3790964A4 (en) * 2018-05-11 2022-06-08 Beam Therapeutics, Inc. METHODS FOR REMOVAL OF PATHOGENIC MUTATIONS USING PROGRAMMABLE BASE EDITOR SYSTEMS
EP3980540A4 (en) 2019-06-06 2023-11-01 Arrowhead Pharmaceuticals, Inc. METHOD FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY (AATD)
JP7846095B2 (ja) * 2020-09-15 2026-04-14 アローヘッド ファーマシューティカルズ インコーポレイテッド Z-aatタンパク質のレベルを低下させる方法
TW202308660A (zh) * 2021-08-25 2023-03-01 美商戴瑟納製藥股份有限公司 用於抑制α-1抗胰蛋白酶表現之組合物及方法
JP2024533312A (ja) * 2021-09-08 2024-09-12 フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー Serpina調節組成物及び方法
WO2024229229A2 (en) * 2023-05-02 2024-11-07 Sirnaomics, Inc. Products and compositions

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Also Published As

Publication number Publication date
US11912996B2 (en) 2024-02-27
PT3444350T (pt) 2022-02-04
US11459566B1 (en) 2022-10-04
AU2014284298B2 (en) 2020-12-10
US20190390198A1 (en) 2019-12-26
CA2916499A1 (en) 2015-01-08
US11851658B2 (en) 2023-12-26
US11408002B1 (en) 2022-08-09
US11312960B1 (en) 2022-04-26
JP2024075702A (ja) 2024-06-04
EP3444350A1 (en) 2019-02-20
US20170130221A1 (en) 2017-05-11
US20150011607A1 (en) 2015-01-08
US20220228149A1 (en) 2022-07-21
JP2020188771A (ja) 2020-11-26
US10370655B2 (en) 2019-08-06
US20220396793A1 (en) 2022-12-15
PL3444350T3 (pl) 2022-03-21
US11312961B1 (en) 2022-04-26
AU2020260479A1 (en) 2020-11-26
EP3017047A4 (en) 2017-06-14
EP4012031A1 (en) 2022-06-15
DK3444350T3 (da) 2022-02-07
WO2015003113A3 (en) 2015-02-26
AU2020260479B2 (en) 2023-06-15
AU2014284298A1 (en) 2016-01-21
HUE058794T2 (hu) 2022-09-28
US20230069235A1 (en) 2023-03-02
US10844381B2 (en) 2020-11-24
US9458457B2 (en) 2016-10-04
US11208658B2 (en) 2021-12-28
US20210180061A1 (en) 2021-06-17
JP2016528887A (ja) 2016-09-23
EP3444350B1 (en) 2021-12-01
WO2015003113A2 (en) 2015-01-08
EP3017047A2 (en) 2016-05-11
JP2022078069A (ja) 2022-05-24

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