JP2025096283A5 - - Google Patents

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Publication number
JP2025096283A5
JP2025096283A5 JP2025039288A JP2025039288A JP2025096283A5 JP 2025096283 A5 JP2025096283 A5 JP 2025096283A5 JP 2025039288 A JP2025039288 A JP 2025039288A JP 2025039288 A JP2025039288 A JP 2025039288A JP 2025096283 A5 JP2025096283 A5 JP 2025096283A5
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JP
Japan
Prior art keywords
pharmaceutical composition
drug substance
dose
aat rnai
rnai drug
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Pending
Application number
JP2025039288A
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English (en)
Japanese (ja)
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JP2025096283A (ja
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Priority claimed from PCT/US2020/036359 external-priority patent/WO2020247774A1/en
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Publication of JP2025096283A publication Critical patent/JP2025096283A/ja
Publication of JP2025096283A5 publication Critical patent/JP2025096283A5/ja
Pending legal-status Critical Current

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JP2025039288A 2019-06-06 2025-03-12 アルファ-1アンチトリプシン欠乏症(aatd)の治療法 Pending JP2025096283A (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962858059P 2019-06-06 2019-06-06
US62/858,059 2019-06-06
PCT/US2020/036359 WO2020247774A1 (en) 2019-06-06 2020-06-05 Methods for the treatment of alpha-1 antitrypsin deficiency (aatd)
JP2021572103A JP7697889B2 (ja) 2019-06-06 2020-06-05 アルファ-1アンチトリプシン欠乏症(aatd)の治療法

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2021572103A Division JP7697889B2 (ja) 2019-06-06 2020-06-05 アルファ-1アンチトリプシン欠乏症(aatd)の治療法

Publications (2)

Publication Number Publication Date
JP2025096283A JP2025096283A (ja) 2025-06-26
JP2025096283A5 true JP2025096283A5 (https=) 2025-10-24

Family

ID=73652297

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021572103A Active JP7697889B2 (ja) 2019-06-06 2020-06-05 アルファ-1アンチトリプシン欠乏症(aatd)の治療法
JP2025039288A Pending JP2025096283A (ja) 2019-06-06 2025-03-12 アルファ-1アンチトリプシン欠乏症(aatd)の治療法

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2021572103A Active JP7697889B2 (ja) 2019-06-06 2020-06-05 アルファ-1アンチトリプシン欠乏症(aatd)の治療法

Country Status (12)

Country Link
US (1) US12582668B2 (https=)
EP (1) EP3980540A4 (https=)
JP (2) JP7697889B2 (https=)
KR (1) KR20220019255A (https=)
CN (1) CN114222820A (https=)
AU (1) AU2020286462A1 (https=)
BR (1) BR112021024534A2 (https=)
CA (1) CA3141762A1 (https=)
CO (1) CO2022000016A2 (https=)
MX (1) MX2021015003A (https=)
TW (1) TW202113081A (https=)
WO (1) WO2020247774A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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
CA3264922A1 (en) * 2022-08-22 2024-02-29 Arrowhead Pharmaceuticals, Inc. METHODS FOR THE TREATMENT OF DISEASES AND DISORDERS RELATED TO ANGPTL3
WO2025219443A1 (en) * 2024-04-19 2025-10-23 Uniqure Biopharma B.V. Nucleic acid for a1at regulation

Family Cites Families (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
EP0366685B1 (en) 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
DE69034150T2 (de) 1989-10-24 2005-08-25 Isis Pharmaceuticals, Inc., Carlsbad 2'-Modifizierte Oligonukleotide
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5212295A (en) 1990-01-11 1993-05-18 Isis Pharmaceuticals Monomers for preparation of oligonucleotides having chiral phosphorus linkages
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
DE69233046T2 (de) 1991-10-24 2004-03-04 Isis Pharmaceuticals, Inc., Carlsfeld Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US6077705A (en) 1996-05-17 2000-06-20 Thomas Jefferson University Ribozyme-mediated gene replacement
US6172209B1 (en) 1997-02-14 2001-01-09 Isis Pharmaceuticals Inc. Aminooxy-modified oligonucleotides and methods for making same
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ/エス オリゴヌクレオチド類似体
FR2774379B1 (fr) 1998-01-30 2002-03-29 Groupe Limagrain Holding Procede de production, par des cellules vegetales, d'alpha 1-antitrypsine et de ses variantes, et produits contenant l'alpha-antitrypsine ainsi obtenue
US6271358B1 (en) 1998-07-27 2001-08-07 Isis Pharmaceuticals, Inc. RNA targeted 2′-modified oligonucleotides that are conformationally preorganized
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
DE60040274D1 (de) 1999-03-10 2008-10-30 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc RNA inhibition mediated inhibition of MAP KINASE GENES
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)
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
ATE443765T1 (de) 2003-03-21 2009-10-15 Santaris Pharma As Analoga kurzer interferierender rna (sirna)
US8053232B2 (en) 2004-01-23 2011-11-08 Virxsys Corporation Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated RNA trans splicing
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
AU2005327506B2 (en) * 2004-10-20 2010-07-08 Antisense Therapeutics Ltd Antisense modulation of integrin alpha4 expression
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
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
WO2008086524A2 (en) 2007-01-11 2008-07-17 University Of Florida Research Foundation, Inc. Alpha-1-antitrypsin variants and uses thereof
KR101629017B1 (ko) 2007-05-22 2016-06-10 아크투루스 쎄라퓨틱스, 인크. 히드록시메틸 치환된 rna 올리고뉴클레오티드 및 rna 복합체
WO2009073809A2 (en) 2007-12-04 2009-06-11 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2718765A1 (en) 2008-03-20 2009-09-24 Quark Pharmaceuticals, Inc. Novel sirna compounds for inhibiting rtp801
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
SI2539451T1 (sl) 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna
AU2011299233B2 (en) 2010-09-07 2016-09-15 Integrated Dna Technologies, Inc. Modifications for antisense compounds
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
CA3131967A1 (en) 2010-12-29 2012-07-05 F. Hoffman-La Roche Ag Small molecule conjugates for intracellular delivery of nucleic acids
WO2012178033A2 (en) 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
WO2013032829A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
CA2842041A1 (en) 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
EA038792B1 (ru) 2013-05-22 2021-10-20 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ RNAi Serpina1 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
WO2014197524A2 (en) 2013-06-06 2014-12-11 Serpin Pharma, Llc Peptide therapy for treatment of alpha-1 antitrypsin deficiency and associated pathologies
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
CA2950960A1 (en) 2014-06-06 2015-12-10 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups
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
AU2016306275A1 (en) 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
WO2017139616A1 (en) 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
CA3011946A1 (en) 2016-03-07 2017-09-14 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
EP3469083A1 (en) * 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
SG11201901841TA (en) 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
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
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
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

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