JP2016528887A5 - - Google Patents
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- Publication number
- JP2016528887A5 JP2016528887A5 JP2016524365A JP2016524365A JP2016528887A5 JP 2016528887 A5 JP2016528887 A5 JP 2016528887A5 JP 2016524365 A JP2016524365 A JP 2016524365A JP 2016524365 A JP2016524365 A JP 2016524365A JP 2016528887 A5 JP2016528887 A5 JP 2016528887A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- oligonucleotide chain
- nucleotides
- antitrypsin
- target sequence
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 108091034117 Oligonucleotide Proteins 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 102000015395 alpha 1-Antitrypsin Human genes 0.000 claims 2
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims 2
- 229940024142 alpha 1-antitrypsin Drugs 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 230000000295 complement effect Effects 0.000 claims 1
- 230000007423 decrease Effects 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
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)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020094030A Division JP2020188771A (ja) | 2013-07-03 | 2020-05-29 | 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016528887A JP2016528887A (ja) | 2016-09-23 |
| JP2016528887A5 true JP2016528887A5 (https=) | 2017-08-10 |
Family
ID=52133228
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016524365A Pending JP2016528887A (ja) | 2013-07-03 | 2014-07-03 | 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 |
| JP2020094030A Withdrawn JP2020188771A (ja) | 2013-07-03 | 2020-05-29 | 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 |
| JP2022019996A Withdrawn JP2022078069A (ja) | 2013-07-03 | 2022-02-10 | 二本鎖ＲＮＡによるα－１アンチトリプシンの特異的阻害のための方法及び組成物 |
| JP2024045063A Withdrawn JP2024075702A (ja) | 2013-07-03 | 2024-03-21 | 二本鎖ＲＮＡによるα－１アンチトリプシンの特異的阻害のための方法及び組成物 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020094030A Withdrawn JP2020188771A (ja) | 2013-07-03 | 2020-05-29 | 二本鎖ＲＮＡによるα−１アンチトリプシンの特異的阻害のための方法及び組成物 |
| JP2022019996A Withdrawn JP2022078069A (ja) | 2013-07-03 | 2022-02-10 | 二本鎖ＲＮＡによるα－１アンチトリプシンの特異的阻害のための方法及び組成物 |
| JP2024045063A Withdrawn JP2024075702A (ja) | 2013-07-03 | 2024-03-21 | 二本鎖ＲＮＡによるα－１アンチトリプシンの特異的阻害のための方法及び組成物 |
Country Status (11)
| Country | Link |
|---|---|
| US (11) | US9458457B2 (https=) |
| EP (3) | EP3017047A4 (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 |
|---|---|---|---|---|
| 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) |
| TW201936624A (zh) | 2013-05-22 | 2019-09-16 | 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 |
| 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 |
| EA201891338A1 (ru) * | 2015-12-04 | 2018-12-28 | Новартис Аг | Композиции и способы для иммуноонкологии |
| WO2017139616A1 (en) * | 2016-02-10 | 2017-08-17 | Arrowhead Pharmaceuticals, Inc. | Methods of treatment for alpha-1 antitrypsin deficiency |
| CA3024448C (en) * | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| US10576167B2 (en) | 2016-08-17 | 2020-03-03 | 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 |
| EP4035659A1 (en) | 2016-11-29 | 2022-08-03 | PureTech LYT, Inc. | Exosomes for delivery of therapeutic agents |
| AU2017379073B2 (en) * | 2016-12-22 | 2023-12-14 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiency |
| BR112019014282A2 (pt) | 2017-01-10 | 2020-03-03 | Arrowhead Pharmaceuticals, Inc. | Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso |
| SE540888C2 (en) * | 2017-06-19 | 2018-12-11 | Hepgene Medical Ab | Novel nucleic acid molecules and their use in therapy |
| CA3094008A1 (en) * | 2018-04-13 | 2019-10-17 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides |
| 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 |
| MX2021015003A (es) | 2019-06-06 | 2022-01-24 | Arrowhead Pharmaceuticals Inc | Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd). |
| AU2021345026A1 (en) * | 2020-09-15 | 2023-05-11 | Arrowhead Pharmaceuticals, Inc. | Methods for the reduction of z-aat protein levels |
| JP2024532019A (ja) | 2021-08-25 | 2024-09-05 | ノボ ノルディスク エー／エス | アルファ－１アンチトリプシン発現を阻害するための組成物及び方法 |
| CA3231594A1 (en) * | 2021-09-08 | 2023-03-16 | Flagship Pioneering Innovations Vi, Llc | Serpina-modulating compositions and methods |
| WO2024229229A2 (en) * | 2023-05-02 | 2024-11-07 | Sirnaomics, Inc. | Products and compositions |
Family Cites Families (35)
| 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 |
| JP3257675B2 (ja) | 1990-10-12 | 2002-02-18 | マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー．ファウ． | 修飾リボザイム |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| 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 |
| US6291438B1 (en) | 1993-02-24 | 2001-09-18 | Jui H. Wang | Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their 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) |
| WO2004014933A1 (en) | 2002-08-07 | 2004-02-19 | 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 |
| EP2305812A3 (en) | 2002-11-14 | 2012-06-06 | 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 |
| WO2004094636A1 (en) * | 2003-04-24 | 2004-11-04 | 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 |
| AU2005222965B8 (en) | 2004-03-15 | 2010-07-01 | 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 |
| 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 |
| EP2114141A2 (en) * | 2007-01-10 | 2009-11-11 | University of Florida | Compounds and methods for treatment of alpha-1 antitrypsin deficiency |
| JP2012504389A (ja) * | 2008-09-22 | 2012-02-23 | 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 | 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法 |
| US20100173973A1 (en) * | 2008-12-18 | 2010-07-08 | Dicerna Pharmaceuticals, Inc. | Extended dicer substrate agents and methods for the specific inhibition of gene expression |
| AU2009336191B2 (en) | 2008-12-18 | 2017-08-24 | Novo Nordisk A/S | Extended dicer substrate agents and methods for the specific inhibition of gene expression |
| US20100249214A1 (en) | 2009-02-11 | 2010-09-30 | Dicerna Pharmaceuticals | Multiplex dicer substrate rna interference molecules having joining sequences |
| CN104651362A (zh) | 2009-04-03 | 2015-05-27 | 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 |
| JP2012528596A (ja) * | 2009-06-03 | 2012-11-15 | 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 | ペプチドダイサー基質剤およびその特異的な遺伝子発現阻害のための方法 |
| WO2012006241A2 (en) * | 2010-07-06 | 2012-01-12 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna |
| EP3366312A1 (en) * | 2011-06-23 | 2018-08-29 | Alnylam Pharmaceuticals, Inc. | Serpina 1 sirnas: compositions of matter and methods of treatment |
| TW201936624A (zh) * | 2013-05-22 | 2019-09-16 | 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 |
| DK3444350T3 (da) | 2013-07-03 | 2022-02-07 | Dicerna Pharmaceuticals Inc | Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget rna |
-
2014
- 2014-07-03 DK DK18196106.1T patent/DK3444350T3/da active
- 2014-07-03 EP EP14820312.8A patent/EP3017047A4/en not_active Withdrawn
- 2014-07-03 HU HUE18196106A patent/HUE058794T2/hu unknown
- 2014-07-03 EP EP21210315.4A patent/EP4012031A1/en not_active Withdrawn
- 2014-07-03 PL PL18196106T patent/PL3444350T3/pl unknown
- 2014-07-03 ES ES18196106T patent/ES2905257T3/es active Active
- 2014-07-03 CA CA2916499A patent/CA2916499A1/en active Pending
- 2014-07-03 EP EP18196106.1A patent/EP3444350B1/en active Active
- 2014-07-03 US US14/323,299 patent/US9458457B2/en active Active
- 2014-07-03 AU AU2014284298A patent/AU2014284298B2/en not_active Ceased
- 2014-07-03 JP JP2016524365A patent/JP2016528887A/ja active Pending
- 2014-07-03 WO PCT/US2014/045365 patent/WO2015003113A2/en not_active Ceased
- 2014-07-03 PT PT181961061T patent/PT3444350T/pt unknown
-
2016
- 2016-08-25 US US15/247,201 patent/US10370655B2/en active Active
-
2019
- 2019-06-28 US US16/457,394 patent/US10844381B2/en active Active
-
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
-
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
-
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
-
2024
- 2024-03-21 JP JP2024045063A patent/JP2024075702A/ja not_active Withdrawn
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