JP2018506304A5 - - Google Patents

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Publication number
JP2018506304A5
JP2018506304A5 JP2017560478A JP2017560478A JP2018506304A5 JP 2018506304 A5 JP2018506304 A5 JP 2018506304A5 JP 2017560478 A JP2017560478 A JP 2017560478A JP 2017560478 A JP2017560478 A JP 2017560478A JP 2018506304 A5 JP2018506304 A5 JP 2018506304A5
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JP
Japan
Prior art keywords
rnai
vector
guide region
sequence
strand
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
Application number
JP2017560478A
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English (en)
Japanese (ja)
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JP2018506304A (ja
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Publication date
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Priority claimed from PCT/US2016/017207 external-priority patent/WO2016130589A2/en
Publication of JP2018506304A publication Critical patent/JP2018506304A/ja
Publication of JP2018506304A5 publication Critical patent/JP2018506304A5/ja
Priority to JP2021078464A priority Critical patent/JP7526710B2/ja
Pending legal-status Critical Current

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JP2017560478A 2015-02-10 2016-02-09 バリアントRNAi Pending JP2018506304A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2021078464A JP7526710B2 (ja) 2015-02-10 2021-05-06 バリアントRNAi

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

Related Child Applications (1)

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JP2021078464A Division JP7526710B2 (ja) 2015-02-10 2021-05-06 バリアントRNAi

Publications (2)

Publication Number Publication Date
JP2018506304A JP2018506304A (ja) 2018-03-08
JP2018506304A5 true JP2018506304A5 (cg-RX-API-DMAC7.html) 2019-03-22

Family

ID=56614905

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JP2017560478A Pending JP2018506304A (ja) 2015-02-10 2016-02-09 バリアントRNAi
JP2021078464A Active JP7526710B2 (ja) 2015-02-10 2021-05-06 バリアントRNAi

Family Applications After (1)

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JP2021078464A Active JP7526710B2 (ja) 2015-02-10 2021-05-06 バリアントRNAi

Country Status (25)

Country Link
US (4) US10450563B2 (cg-RX-API-DMAC7.html)
EP (1) EP3256588A2 (cg-RX-API-DMAC7.html)
JP (2) JP2018506304A (cg-RX-API-DMAC7.html)
KR (1) KR102670852B1 (cg-RX-API-DMAC7.html)
CN (1) CN107438671B (cg-RX-API-DMAC7.html)
AR (1) AR103646A1 (cg-RX-API-DMAC7.html)
AU (3) AU2016219396B2 (cg-RX-API-DMAC7.html)
CA (1) CA3255657A1 (cg-RX-API-DMAC7.html)
CL (1) CL2017002027A1 (cg-RX-API-DMAC7.html)
CO (1) CO2017009083A2 (cg-RX-API-DMAC7.html)
CR (1) CR20170406A (cg-RX-API-DMAC7.html)
EA (1) EA201791805A1 (cg-RX-API-DMAC7.html)
EC (1) ECSP17059343A (cg-RX-API-DMAC7.html)
HK (1) HK1247641A1 (cg-RX-API-DMAC7.html)
IL (1) IL253893B (cg-RX-API-DMAC7.html)
MA (1) MA40819B1 (cg-RX-API-DMAC7.html)
MX (2) MX2017010369A (cg-RX-API-DMAC7.html)
MY (2) MY194175A (cg-RX-API-DMAC7.html)
PE (1) PE20171382A1 (cg-RX-API-DMAC7.html)
PH (1) PH12017501432A1 (cg-RX-API-DMAC7.html)
SG (3) SG11201706444TA (cg-RX-API-DMAC7.html)
TN (1) TN2017000354A1 (cg-RX-API-DMAC7.html)
TW (1) TWI781079B (cg-RX-API-DMAC7.html)
UY (1) UY36554A (cg-RX-API-DMAC7.html)
WO (1) WO2016130589A2 (cg-RX-API-DMAC7.html)

Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2009323766B2 (en) 2008-12-02 2016-10-06 Wave Life Sciences Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Wave Life Sciences Japan 不斉補助基
JP6246121B2 (ja) 2012-07-13 2017-12-13 株式会社新日本科学 キラル核酸アジュバント
SG10201912895PA (en) 2012-07-13 2020-02-27 Wave Life Sciences Ltd Chiral control
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
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
PT3094728T (pt) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Desenho quiral
CN112375760A (zh) 2014-11-14 2021-02-19 沃雅戈治疗公司 调节性多核苷酸
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
EA201791805A1 (ru) 2015-02-10 2018-05-31 Джензим Корпорейшн ВАРИАНТ СРЕДСТВА ДЛЯ RNAi
JP6892433B2 (ja) 2015-04-03 2021-06-23 ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts 十分に安定化された非対称sirna
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US10633653B2 (en) 2015-08-14 2020-04-28 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
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
IL302748B1 (en) 2016-05-18 2025-09-01 Voyager Therapeutics Inc Modulatory polynucleotides
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
CN110799647A (zh) 2017-06-23 2020-02-14 马萨诸塞大学 双尾自递送sirna及相关的方法
AU2018335410B2 (en) 2017-09-22 2025-04-03 Genzyme Corporation Variant RNAi
WO2019060649A1 (en) * 2017-09-22 2019-03-28 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
US10870855B2 (en) * 2017-12-06 2020-12-22 Ovid Therapeutics Inc. Use of MIR101 or MIR128 in the treatment of seizure disorders
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
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
CN112770812A (zh) 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
PH12021550254A1 (en) * 2018-08-03 2023-02-06 Genzyme Corp Variant rnai against alpha-synuclein
WO2020041769A1 (en) 2018-08-23 2020-02-27 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
JP2022504740A (ja) * 2018-10-12 2022-01-13 ジェンザイム・コーポレーション 肝臓に向けられた遺伝子補充療法によって重度のpkuを処置するための改善されたヒトpahの生成
BR112021013109A2 (pt) * 2019-01-09 2021-10-13 Universidade De Coimbra Rna de fita dupla e seus usos.
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
AU2020329155A1 (en) 2019-08-09 2022-03-10 University Of Massachusetts Chemically modified oligonucleotides targeting SNPs
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
US20210355491A1 (en) * 2020-04-20 2021-11-18 University Of Massachusetts Oligonucleotides for msh3 modulation
WO2023093905A1 (zh) 2021-11-29 2023-06-01 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
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

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2001500015A (ja) 1996-09-06 2001-01-09 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア T7ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
CA2410828C (en) 2000-06-01 2012-01-24 University Of North Carolina At Chapel Hill 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
MX346493B (es) 2001-11-13 2017-03-21 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
ATE513843T1 (de) 2002-09-25 2011-07-15 Univ Massachusetts Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna
US7589189B2 (en) * 2003-05-14 2009-09-15 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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and 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
WO2008150897A2 (en) * 2007-05-31 2008-12-11 University Of Iowa Research Foundation Reduction of off-target rna interference toxicity
WO2009098196A2 (en) 2008-02-04 2009-08-13 Biofocus Dpi B.V. Molecular targets and compounds, methods to identify the same, useful in the treatment of neurodegenerative diseases.
WO2009100502A1 (en) 2008-02-14 2009-08-20 Monash University Immunostimulatory sirna molecules
FR2929292A1 (fr) 2008-03-28 2009-10-02 Exonhit Therapeutics S A Sa Procede et methodes de diagnostic de la maladie d'alzheimer
WO2011034811A1 (en) 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics
CA2833908C (en) 2010-04-23 2021-02-09 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
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
JP6490426B2 (ja) 2012-02-29 2019-03-27 サンガモ セラピューティクス, インコーポレイテッド ハンチントン病を治療するための方法および組成物
US9550989B2 (en) * 2013-10-03 2017-01-24 Washington University Rational design of microRNA-siRNA chimeras for multi-functional target suppression
PT3119437T (pt) 2014-03-21 2019-12-12 Genzyme Corp Terapia genética para retinopatia pigmentar
IL248102B (en) 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
LT3237618T (lt) 2014-12-24 2019-07-10 Uniqure Ip B.V. Rnri sukeltas hantingtino geno slopinimas
EA201791805A1 (ru) 2015-02-10 2018-05-31 Джензим Корпорейшн ВАРИАНТ СРЕДСТВА ДЛЯ RNAi
AU2018335410B2 (en) 2017-09-22 2025-04-03 Genzyme Corporation Variant RNAi

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