PT3444350T - Métodos e composições para a inibição específica de alfa-1 antitripsina através de arn de cadeia dupla - Google Patents
Métodos e composições para a inibição específica de alfa-1 antitripsina através de arn de cadeia duplaInfo
- Publication number
- PT3444350T PT3444350T PT181961061T PT18196106T PT3444350T PT 3444350 T PT3444350 T PT 3444350T PT 181961061 T PT181961061 T PT 181961061T PT 18196106 T PT18196106 T PT 18196106T PT 3444350 T PT3444350 T PT 3444350T
- Authority
- PT
- Portugal
- Prior art keywords
- antitrypsin
- alpha
- compositions
- double
- methods
- Prior art date
Links
- 108091032973 (ribonucleotides)n+m Proteins 0.000 title 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 title 1
- 102000015395 alpha 1-Antitrypsin Human genes 0.000 title 1
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 title 1
- 229940024142 alpha 1-antitrypsin Drugs 0.000 title 1
- 230000005764 inhibitory process Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
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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. ==--==--==--
Landscapes
- 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)
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 |
|---|---|
| PT3444350T true PT3444350T (pt) | 2022-02-04 |
Family
ID=52133228
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT181961061T PT3444350T (pt) | 2013-07-03 | 2014-07-03 | Métodos e composições para a inibição específica de alfa-1 antitripsina através de arn de cadeia dupla |
Country Status (11)
| Country | Link |
|---|---|
| US (11) | US9458457B2 (https=) |
| EP (3) | EP4012031A1 (https=) |
| JP (4) | JP2016528887A (https=) |
| AU (2) | AU2014284298B2 (https=) |
| CA (1) | CA2916499A1 (https=) |
| DK (1) | DK3444350T3 (https=) |
| ES (1) | ES2905257T3 (https=) |
| HU (1) | HUE058794T2 (https=) |
| PL (1) | PL3444350T3 (https=) |
| PT (1) | PT3444350T (https=) |
| WO (1) | WO2015003113A2 (https=) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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) |
| 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) |
| 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) |
| EA038792B1 (ru) | 2013-05-22 | 2021-10-20 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ RNAi Serpina1 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| US9458457B2 (en) | 2013-07-03 | 2016-10-04 | 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 |
| KR20240056729A (ko) * | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| IL308824A (en) | 2016-08-17 | 2024-01-01 | 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 |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| TWI851534B (zh) * | 2016-12-22 | 2024-08-11 | 美商英特利亞醫療公司 | 用於治療α-１抗胰蛋白酶缺乏症之組合物及方法 |
| CN110268060B (zh) | 2017-01-10 | 2024-07-26 | 箭头药业股份有限公司 | α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法 |
| SE1750774A1 (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 |
| CN119570787A (zh) * | 2018-05-11 | 2025-03-07 | 比姆医疗股份有限公司 | 使用可编程碱基编辑器系统遏止病原性突变的方法 |
| MX2021015003A (es) | 2019-06-06 | 2022-01-24 | Arrowhead Pharmaceuticals Inc | Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (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 |
| EP4392556A1 (en) * | 2021-08-25 | 2024-07-03 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting ?lpha-1 antitrypsin expression |
| MX2024002988A (es) * | 2021-09-08 | 2024-06-11 | Flagship Pioneering Innovations Vi Llc | Composiciones y metodos moduladores de la serpina. |
| WO2024229229A2 (en) * | 2023-05-02 | 2024-11-07 | Sirnaomics, Inc. | Products and compositions |
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| 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 |
| ES2061416T3 (es) | 1990-10-12 | 1997-03-01 | Max Planck Gesellschaft | Ribozimas modificadas. |
| 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 | |
| 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 |
| AU2003295600A1 (en) | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| WO2004094636A1 (en) * | 2003-04-24 | 2004-11-04 | Galapagos Genomics N.V. | Effective sirna knock-down constructs |
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| 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 |
| WO2005116204A1 (ja) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
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| 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 |
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| WO2008143633A2 (en) | 2007-01-10 | 2008-11-27 | University Of Florida | Compounds and methods for treatment of alpha-1 antitrypsin deficiency |
| DK2341943T3 (en) * | 2008-09-22 | 2019-02-25 | Dicerna Pharmaceuticals Inc | COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS |
| KR101728655B1 (ko) | 2008-12-18 | 2017-04-19 | 다이서나 파마수이티컬, 인크. | 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법 |
| US20100249214A1 (en) | 2009-02-11 | 2010-09-30 | Dicerna Pharmaceuticals | Multiplex dicer substrate rna interference molecules having joining sequences |
| US20100173973A1 (en) * | 2008-12-18 | 2010-07-08 | Dicerna Pharmaceuticals, Inc. | Extended dicer substrate agents and methods for the specific inhibition of gene expression |
| CN103555722B (zh) * | 2009-04-03 | 2016-08-31 | 戴瑟纳制药公司 | 利用不对称双链rna特异性抑制kras的方法和组合物 |
| 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 |
| WO2012178033A2 (en) | 2011-06-23 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Serpina1 sirnas: compositions of matter and methods of treatment |
| EA038792B1 (ru) * | 2013-05-22 | 2021-10-20 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ RNAi Serpina1 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| US9458457B2 (en) | 2013-07-03 | 2016-10-04 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA |
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2014
- 2014-07-03 US US14/323,299 patent/US9458457B2/en active Active
- 2014-07-03 JP JP2016524365A patent/JP2016528887A/ja active Pending
- 2014-07-03 PT PT181961061T patent/PT3444350T/pt unknown
- 2014-07-03 EP EP21210315.4A patent/EP4012031A1/en not_active Withdrawn
- 2014-07-03 EP EP18196106.1A patent/EP3444350B1/en active Active
- 2014-07-03 CA CA2916499A patent/CA2916499A1/en active Pending
- 2014-07-03 EP EP14820312.8A patent/EP3017047A4/en not_active Withdrawn
- 2014-07-03 DK DK18196106.1T patent/DK3444350T3/da active
- 2014-07-03 AU AU2014284298A patent/AU2014284298B2/en not_active Ceased
- 2014-07-03 HU HUE18196106A patent/HUE058794T2/hu unknown
- 2014-07-03 WO PCT/US2014/045365 patent/WO2015003113A2/en not_active Ceased
- 2014-07-03 PL PL18196106T patent/PL3444350T3/pl unknown
- 2014-07-03 ES ES18196106T patent/ES2905257T3/es active Active
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2016
- 2016-08-25 US US15/247,201 patent/US10370655B2/en active Active
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2019
- 2019-06-28 US US16/457,394 patent/US10844381B2/en active Active
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2020
- 2020-05-29 JP JP2020094030A patent/JP2020188771A/ja not_active Withdrawn
- 2020-07-01 US US16/918,205 patent/US11208658B2/en not_active Expired - Fee Related
- 2020-10-29 AU AU2020260479A patent/AU2020260479B2/en not_active Ceased
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2021
- 2021-08-12 US US17/400,259 patent/US11312960B1/en active Active
- 2021-08-12 US US17/400,317 patent/US11408002B1/en active Active
- 2021-09-10 US US17/472,255 patent/US11312961B1/en active Active
- 2021-09-10 US US17/472,227 patent/US11459566B1/en active Active
- 2021-11-12 US US17/454,694 patent/US20230069235A1/en not_active Abandoned
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2022
- 2022-02-10 JP JP2022019996A patent/JP2022078069A/ja not_active Withdrawn
- 2022-03-30 US US17/709,087 patent/US11912996B2/en active Active
- 2022-06-09 US US17/836,789 patent/US11851658B2/en active Active
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2024
- 2024-03-21 JP JP2024045063A patent/JP2024075702A/ja not_active Withdrawn
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