KR102670852B1 - 변이형 RNAi - Google Patents

변이형 RNAi Download PDF

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KR102670852B1
KR102670852B1 KR1020177025286A KR20177025286A KR102670852B1 KR 102670852 B1 KR102670852 B1 KR 102670852B1 KR 1020177025286 A KR1020177025286 A KR 1020177025286A KR 20177025286 A KR20177025286 A KR 20177025286A KR 102670852 B1 KR102670852 B1 KR 102670852B1
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delete delete
vector
promoter
strand
nucleic acid
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KR20170110149A (ko
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리사 엠. 스타넥
아담 팔레르모
브렌다 리차드
세르지오 파블로 사르디
캐서린 오리어던
안토니우스 송
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젠자임 코포레이션
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KR1020177025286A 2015-02-10 2016-02-09 변이형 RNAi Active KR102670852B1 (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
US62/114,578 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

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KR20170110149A KR20170110149A (ko) 2017-10-10
KR102670852B1 true KR102670852B1 (ko) 2024-05-29

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US (4) US10450563B2 (enExample)
EP (1) EP3256588A2 (enExample)
JP (2) JP2018506304A (enExample)
KR (1) KR102670852B1 (enExample)
CN (1) CN107438671B (enExample)
AR (1) AR103646A1 (enExample)
AU (3) AU2016219396B2 (enExample)
CA (1) CA3255657A1 (enExample)
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CO (1) CO2017009083A2 (enExample)
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Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2744987C (en) 2008-12-02 2018-01-16 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
WO2013012758A1 (en) 2011-07-19 2013-01-24 Ontorii, Inc. Methods for the synthesis of functionalized nucleic acids
KR102712879B1 (ko) 2012-07-13 2024-10-04 웨이브 라이프 사이언시스 리미티드 키랄 제어
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
CN104684923B (zh) 2012-07-13 2018-09-28 株式会社新日本科学 手性核酸佐剂
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
EP4137572A1 (en) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Chiral design
CN112410339A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
RU2716422C2 (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
JP2018506304A (ja) 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
EP3277811B1 (en) 2015-04-03 2020-12-23 University of Massachusetts Fully stabilized asymmetric sirna
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
CA2995110A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
US20180228830A1 (en) * 2015-10-23 2018-08-16 Rena Therapeutics Inc. Nucleic acid complex having at least one bulge structure
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
CA3252099A1 (en) 2016-05-18 2025-06-05 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CN109831916B (zh) 2016-05-18 2023-07-21 沃雅戈治疗公司 治疗亨廷顿氏舞蹈病的组合物和方法
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
CA3064590A1 (en) 2017-06-23 2018-12-27 University Of Massachusetts Two-tailed self-delivering sirna and related methods
WO2019060649A1 (en) * 2017-09-22 2019-03-28 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
EP3684932A1 (en) * 2017-09-22 2020-07-29 Genzyme Corporation Variant rnai
KR20200119782A (ko) * 2017-12-06 2020-10-20 오비드 테라퓨틱스 인크. 발작 장애 치료 시 mir101 또는 mir128의 용도
CN112368051A (zh) * 2018-03-23 2021-02-12 马萨诸塞大学 用于治疗骨病的基因治疗剂
BR112020023082A2 (pt) 2018-05-15 2021-02-09 Voyager Therapeutics, Inc. composições e métodos para o tratamento de doença de parkinson
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020028816A1 (en) * 2018-08-03 2020-02-06 Genzyme Corporation Variant rnai against alpha-synuclein
JP7627042B2 (ja) 2018-08-23 2025-02-05 ユニバーシティー オブ マサチューセッツ Ｏ－メチルリッチ完全安定化オリゴヌクレオチド
US20210348135A1 (en) * 2018-10-12 2021-11-11 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
MX2021008331A (es) * 2019-01-09 2021-08-05 Univ De Coimbra Arn bicatenario y usos del mismo.
CN113614232A (zh) 2019-01-18 2021-11-05 马萨诸塞大学 动态药代动力学修饰锚
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
US12024706B2 (en) 2019-08-09 2024-07-02 University Of Massachusetts Modified oligonucleotides targeting SNPs
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
WO2021216556A2 (en) * 2020-04-20 2021-10-28 University Of Massachusetts Oligonucleotides for msh3 modulation
US20250051750A1 (en) 2021-11-29 2025-02-13 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
WO2024187106A2 (en) * 2023-03-09 2024-09-12 Leal Therapeutics, Inc. Compositions and methods for modulating grin2a
KR20250129583A (ko) * 2024-02-21 2025-08-29 서울대학교산학협력단 Drosha 절단을 촉진시키는 구조적 모티프를 포함하는 rna 간섭 유도 핵산 분자의 변형 전구체

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009100502A1 (en) 2008-02-14 2009-08-20 Monash University Immunostimulatory sirna molecules
WO2011034811A1 (en) 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
AU6972301A (en) 2000-06-01 2001-12-11 Univ North Carolina Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
WO2004029212A2 (en) 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
CA2526893C (en) 2003-05-14 2010-10-26 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US7902352B2 (en) * 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US20090036395A1 (en) 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
CA2691617C (en) 2007-05-31 2020-07-21 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
CA2711585A1 (en) 2008-02-04 2009-08-13 Galapagos Nv Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
FR2929292A1 (fr) 2008-03-28 2009-10-02 Exonhit Therapeutics S A Sa Procede et methodes de diagnostic de la maladie d'alzheimer
US9102949B2 (en) 2010-04-23 2015-08-11 University Of Massachusetts CNS targeting AAV vectors and methods of use thereof
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
AU2012219380B2 (en) 2011-02-17 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving AAV9-mediated gene transfer
EP2820159B1 (en) 2012-02-29 2019-10-23 Sangamo Therapeutics, Inc. Methods and compositions for treating huntington's disease
US9550989B2 (en) * 2013-10-03 2017-01-24 Washington University Rational design of microRNA-siRNA chimeras for multi-functional target suppression
SI3119437T1 (sl) 2014-03-21 2020-01-31 Genzyme Corporation Genska terapija za retinitis pigmentoza
PE20170260A1 (es) 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
SMT201900347T1 (it) 2014-12-24 2019-07-11 Uniqure Ip Bv Soppressione del gene huntingtina indotta da rnai
JP2018506304A (ja) 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
EP3684932A1 (en) 2017-09-22 2020-07-29 Genzyme Corporation Variant rnai

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009100502A1 (en) 2008-02-14 2009-08-20 Monash University Immunostimulatory sirna molecules
WO2011034811A1 (en) 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Cell,제151권,4호,900-911면(2012.11.9.) 1부.*

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