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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    • 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)
  • Medicinal Preparation (AREA)
  • Electric Double-Layer Capacitors Or The Like (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)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
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)
EP3327125B1 (en) 2010-10-29 2020-08-05 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
LT2999785T (lt) 2013-05-22 2018-07-25 Alnylam Pharmaceuticals, Inc. Serpina1 irna kompozicijos ir jų panaudojimo būdai
DK3444350T3 (da) 2013-07-03 2022-02-07 Dicerna Pharmaceuticals Inc Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget 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
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
KR20240056729A (ko) * 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US10576167B2 (en) 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
TWI788312B (zh) 2016-11-23 2023-01-01 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
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
BR112019012825A2 (pt) 2016-12-22 2019-11-26 Intellia Therapeutics Inc composições e métodos para tratar deficiência de alfa-1 antitripsina
TWI826365B (zh) 2017-01-10 2023-12-21 美商愛羅海德製藥公司 α-1抗胰蛋白酶 (AAT) RNAi 藥劑、包含AAT RNAi 藥劑之組合物及使用方法
SE540888C2 (en) * 2017-06-19 2018-12-11 Hepgene Medical Ab Novel nucleic acid molecules and their use in therapy
JP2021520355A (ja) * 2018-04-13 2021-08-19 ディセルナ ファーマシューティカルズ インコーポレイテッド Tm上昇ヌクレオチドで修飾された二本鎖核酸阻害剤分子
JP7558929B2 (ja) * 2018-05-11 2024-10-01 ビーム セラピューティクス インク. プログラム可能塩基エディターシステムを用いて病原性変異を抑制する方法
KR20230067648A (ko) * 2020-09-15 2023-05-16 애로우헤드 파마슈티컬스 인코포레이티드 Z-aat 단백질 수준을 감소시키기 위한 방법
KR20240046843A (ko) 2021-08-25 2024-04-11 노보 노르디스크 에이/에스 알파-1 항트립신 발현을 억제하기 위한 조성물 및 방법
WO2024229229A2 (en) * 2023-05-02 2024-11-07 Sirnaomics, Inc. Products and compositions

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1975A (en) 1841-02-12 Manner of constructing corn-shellers
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
PL169576B1 (pl) 1990-10-12 1996-08-30 Max Planck Gesellschaft Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US6291438B1 (en) 1993-02-24 2001-09-18 Jui H. Wang Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use
US5858988A (en) 1993-02-24 1999-01-12 Wang; Jui H. Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
US6218108B1 (en) 1997-05-16 2001-04-17 Research Corporation Technologies, Inc. Nucleoside analogs with polycyclic aromatic groups attached, methods of synthesis and uses therefor
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
US5985836A (en) 1998-07-31 1999-11-16 Bayer Corporation Alpha-1 proteinase inhibitor binding peptides
US20050137153A1 (en) * 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
US8729036B2 (en) 2002-08-07 2014-05-20 University Of Massachusetts Compositions for RNA interference and methods of use thereof
US20040076965A1 (en) * 2002-10-16 2004-04-22 Bosserhoff Anja Katrin MIA-2 protein
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
AU2003224132A1 (en) * 2003-04-24 2004-11-19 Galapagos Genomics N.V. Effective sirna knock-down constructs
US20050042641A1 (en) 2003-05-27 2005-02-24 Cold Spring Harbor Laboratory In vivo high throughput selection of RNAi probes
US8084599B2 (en) 2004-03-15 2011-12-27 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
US20080213891A1 (en) 2004-07-21 2008-09-04 Alnylam Pharmaceuticals, Inc. RNAi Agents Comprising Universal Nucleobases
US20090018097A1 (en) 2005-09-02 2009-01-15 Mdrna, Inc Modification of double-stranded ribonucleic acid molecules
US9150882B2 (en) * 2006-01-31 2015-10-06 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
JP4644619B2 (ja) 2006-03-27 2011-03-02 富士通株式会社 基地局装置、端末および帯域制御方法
EP2114141A2 (en) 2007-01-10 2009-11-11 University of Florida Compounds and methods for treatment of alpha-1 antitrypsin deficiency
US20100331389A1 (en) * 2008-09-22 2010-12-30 Bob Dale Brown Compositions and methods for the specific inhibition of gene expression by dsRNA containing modified nucleotides
US8513207B2 (en) 2008-12-18 2013-08-20 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
US20100173973A1 (en) * 2008-12-18 2010-07-08 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
ES2921573T3 (es) * 2009-04-03 2022-08-29 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de KRAS por RNA bicatenario asimétrico
CN102497870A (zh) 2009-06-03 2012-06-13 戴瑟纳制药公司 肽-dicer底物试剂及其特异性抑制基因表达的方法
CA2804210A1 (en) * 2010-07-06 2012-01-12 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna
US20140235693A1 (en) 2011-06-23 2014-08-21 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
LT2999785T (lt) * 2013-05-22 2018-07-25 Alnylam Pharmaceuticals, Inc. Serpina1 irna kompozicijos ir jų panaudojimo būdai
DK3444350T3 (da) 2013-07-03 2022-02-07 Dicerna Pharmaceuticals Inc Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget rna

Also Published As

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

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