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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Prior art keywords
strand
rna
methyl rna
dsrna
antitrypsin
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ES18196106T
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English (en)
Spanish (es)
Inventor
Bob D Brown
Henryk T Dudek
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (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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Family Applications (1)

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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) EP3444350B1 (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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US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2012058210A1 (en) 2010-10-29 2012-05-03 Merck Sharp & Dohme Corp. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA)
TWI664187B (zh) 2013-05-22 2019-07-01 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
CA2916499A1 (en) 2013-07-03 2015-01-08 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
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WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
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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
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
JP7383478B2 (ja) * 2016-12-22 2023-11-20 インテリア セラピューティクス,インコーポレイテッド アルファ1アンチトリプシン欠乏症を治療するための組成物および方法
US10450565B2 (en) 2017-01-10 2019-10-22 Arrowhead Pharmaceuticals, Inc. Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
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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
KR102959497B1 (ko) * 2018-05-11 2026-05-04 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 돌연변이를 억제하는 방법
AU2020286462B2 (en) 2019-06-06 2026-07-02 Arrowhead Pharmaceuticals, Inc. Methods for the treatment of alpha-1 antitrypsin deficiency (AATD)
WO2022060721A2 (en) * 2020-09-15 2022-03-24 Arrowhead Pharmaceuticals, Inc. Methods for the reduction of z-aat protein levels
KR20240046843A (ko) 2021-08-25 2024-04-11 노보 노르디스크 에이/에스 알파-1 항트립신 발현을 억제하기 위한 조성물 및 방법
WO2023039447A2 (en) * 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Serpina-modulating compositions and methods
TW202430637A (zh) 2022-11-16 2024-08-01 美商戴瑟納製藥股份有限公司 Stat3靶向性寡核苷酸及其用途
WO2024229229A2 (en) * 2023-05-02 2024-11-07 Sirnaomics, Inc. Products and compositions

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Also Published As

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

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