AU2014284298B2 - Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA - Google Patents

Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA Download PDF

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Publication number
AU2014284298B2
AU2014284298B2 AU2014284298A AU2014284298A AU2014284298B2 AU 2014284298 B2 AU2014284298 B2 AU 2014284298B2 AU 2014284298 A AU2014284298 A AU 2014284298A AU 2014284298 A AU2014284298 A AU 2014284298A AU 2014284298 B2 AU2014284298 B2 AU 2014284298B2
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strand
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rna
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AU2014284298A1 (en
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Bob D. Brown
Henryk T. Dudek
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Novo Nordisk AS
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Novo Nordisk AS
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Assigned to NOVO NORDISK A/S reassignment NOVO NORDISK A/S Request for Assignment Assignors: 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)
AU2014284298A 2013-07-03 2014-07-03 Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA Ceased AU2014284298B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2020260479A AU2020260479B2 (en) 2013-07-03 2020-10-29 Methods and Compositions for the Specific Inhibition of Alpha-1 Antitrypsin by Double-Stranded RNA

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361842551P 2013-07-03 2013-07-03
US61/842,551 2013-07-03
US201361891548P 2013-10-16 2013-10-16
US61/891,548 2013-10-16
PCT/US2014/045365 WO2015003113A2 (en) 2013-07-03 2014-07-03 Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna

Related Child Applications (1)

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AU2020260479A Division AU2020260479B2 (en) 2013-07-03 2020-10-29 Methods and Compositions for the Specific Inhibition of Alpha-1 Antitrypsin by Double-Stranded RNA

Publications (2)

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AU2014284298A1 AU2014284298A1 (en) 2016-01-21
AU2014284298B2 true AU2014284298B2 (en) 2020-12-10

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AU2020260479A Ceased AU2020260479B2 (en) 2013-07-03 2020-10-29 Methods and Compositions for the Specific Inhibition of Alpha-1 Antitrypsin by Double-Stranded RNA

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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)

Families Citing this family (23)

* 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)
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
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
US10584337B2 (en) * 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL264439B2 (en) 2016-08-17 2024-08-01 Factor Bioscience Inc non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment
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
SE540888C2 (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
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1410803A1 (de) * 2002-10-16 2004-04-21 Scil proteins GmbH MIA-2 Proteine

Family Cites Families (34)

* 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
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
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)
AU2003261449A1 (en) 2002-08-07 2004-02-25 Compositions for rna interference and methods of use thereof
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
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
CA2559955C (en) 2004-03-15 2016-02-16 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
CA2566286A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere 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
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
WO2008016391A2 (en) * 2006-01-31 2008-02-07 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
WO2008143633A2 (en) * 2007-01-10 2008-11-27 University Of Florida Compounds and methods for treatment of alpha-1 antitrypsin deficiency
WO2010033225A2 (en) * 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
KR20110110776A (ko) 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
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
JP6010458B2 (ja) 2009-04-03 2016-10-19 ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. 非対称二本鎖rnaによる特異的阻害のための、方法および組成物
CN102753187A (zh) * 2009-06-03 2012-10-24 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
AU2011276363A1 (en) * 2010-07-06 2013-01-10 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
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1410803A1 (de) * 2002-10-16 2004-04-21 Scil proteins GmbH MIA-2 Proteine

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
US10844381B2 (en) 2020-11-24
EP3017047A2 (en) 2016-05-11
US11312960B1 (en) 2022-04-26
ES2905257T3 (es) 2022-04-07

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