JPWO2020206350A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2020206350A5
JPWO2020206350A5 JP2021559265A JP2021559265A JPWO2020206350A5 JP WO2020206350 A5 JPWO2020206350 A5 JP WO2020206350A5 JP 2021559265 A JP2021559265 A JP 2021559265A JP 2021559265 A JP2021559265 A JP 2021559265A JP WO2020206350 A5 JPWO2020206350 A5 JP WO2020206350A5
Authority
JP
Japan
Prior art keywords
oligonucleotide
gaaa
sequence
modification
aldh2
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
JP2021559265A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022526419A (ja
JP2022526419A5 (https=
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/026717 external-priority patent/WO2020206350A1/en
Publication of JP2022526419A publication Critical patent/JP2022526419A/ja
Publication of JPWO2020206350A5 publication Critical patent/JPWO2020206350A5/ja
Publication of JP2022526419A5 publication Critical patent/JP2022526419A5/ja
Abandoned legal-status Critical Current

Links

Images

JP2021559265A 2019-04-04 2020-04-03 中枢神経系における遺伝子発現を阻害するための組成物及び方法 Abandoned JP2022526419A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962829595P 2019-04-04 2019-04-04
US62/829,595 2019-04-04
PCT/US2020/026717 WO2020206350A1 (en) 2019-04-04 2020-04-03 Compositions and methods for inhibiting gene expression in the central nervous system

Publications (3)

Publication Number Publication Date
JP2022526419A JP2022526419A (ja) 2022-05-24
JPWO2020206350A5 true JPWO2020206350A5 (https=) 2023-07-11
JP2022526419A5 JP2022526419A5 (https=) 2023-07-11

Family

ID=70482804

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021559265A Abandoned JP2022526419A (ja) 2019-04-04 2020-04-03 中枢神経系における遺伝子発現を阻害するための組成物及び方法

Country Status (13)

Country Link
US (1) US20220170025A1 (https=)
EP (1) EP3947683A1 (https=)
JP (1) JP2022526419A (https=)
KR (1) KR20210148264A (https=)
CN (1) CN113748208A (https=)
AU (1) AU2020252560A1 (https=)
BR (1) BR112021019793A2 (https=)
CA (1) CA3135402A1 (https=)
CL (1) CL2021002585A1 (https=)
IL (1) IL286868A (https=)
MX (1) MX2021012126A (https=)
SG (1) SG11202110896WA (https=)
WO (1) WO2020206350A1 (https=)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019143621A1 (en) 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression
CN119499394A (zh) 2019-11-01 2025-02-25 阿尔尼拉姆医药品有限公司 亨廷顿（HTT）iRNA药剂组合物及其使用方法
EP4073251A1 (en) 2019-12-13 2022-10-19 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
EP4244356A1 (en) * 2020-11-13 2023-09-20 Dicerna Pharmaceuticals, Inc. Chemical modifications for inhibiting expression of aldh2
AR124713A1 (es) * 2021-01-28 2023-04-26 Dicerna Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
CN117222739A (zh) 2021-02-25 2023-12-12 阿尔尼拉姆医药品有限公司 朊病毒蛋白(prnp)irna组合物和其使用方法
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
WO2022212231A2 (en) 2021-03-29 2022-10-06 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
PE20250074A1 (es) * 2021-05-28 2025-01-13 Novo Nordisk As Composiciones y metodos para inhibir la expresion del componente 1 de reduccion de amidoxima mitocondrial (marc1)
CN117561334A (zh) 2021-06-04 2024-02-13 阿尔尼拉姆医药品有限公司 人染色体9开放阅读框72（C9ORF72）iRNA药剂组合物和其使用方法
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
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
AR127587A1 (es) * 2021-11-05 2024-02-07 Dicerna Pharmaceuticals Inc Conjugación de lípidos para dirigir oligodendrocitos del sistema nervioso central
TW202335674A (zh) * 2021-11-05 2023-09-16 美商黛瑟納製藥公司 靶定中樞神經系統的星狀細胞之脂質結合物
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
CN119173631A (zh) * 2022-05-12 2024-12-20 迪克纳制药公司 用于抑制mapt表达的组合物和方法
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
US20050019915A1 (en) * 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
JP2005508196A (ja) 2001-11-07 2005-03-31 アプレラ コーポレイション 核酸分析の汎用ヌクレオチド
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
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
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
US8691971B2 (en) 2008-09-23 2014-04-08 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
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
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
US8999950B2 (en) * 2011-10-05 2015-04-07 Protiva Biotherapeutics Inc. Compositions and methods for silencing aldehyde dehydrogenase
JP2015501155A (ja) 2011-10-25 2015-01-15 アイシス ファーマシューティカルズ， インコーポレーテッド Ｇｃｃｒ発現のアンチセンス調整
EP2928877B1 (en) 2012-12-06 2020-01-22 Merck Sharp & Dohme Corp. Disulfide masked prodrug compositions and methods
JP2017522046A (ja) 2014-06-06 2017-08-10 ソルスティス バイオロジクス，リミティッド 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物
JP7105065B2 (ja) * 2014-12-15 2022-07-22 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 リガンド修飾二本鎖核酸
JP7249080B2 (ja) 2016-08-23 2023-03-30 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 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用
KR102350647B1 (ko) 2016-09-02 2022-01-14 다이서나 파마수이티컬, 인크. 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드
WO2018094116A1 (en) * 2016-11-17 2018-05-24 Retrotope, Inc. Isotopically modified components and therapeutic uses thereof
JP7155239B2 (ja) * 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
EP4029942A1 (en) * 2017-04-05 2022-07-20 Silence Therapeutics GmbH Products and compositions
CA3118327A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of aldh2 in a cell
MX2020004897A (es) * 2017-11-13 2020-10-05 Silence Therapeutics Gmbh Acidos nucleicos para inhibir la expresion de lpa en una celula.
WO2019143621A1 (en) * 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression

Similar Documents

Publication Publication Date Title
JPWO2020206350A5 (https=)
JP6752151B2 (ja) 遺伝子サイレンシングにおいて低下したｏｆｆ−ｔａｒｇｅｔ効果を有するｕｎａオリゴマー
CN113748208A (zh) 用于抑制中枢神经系统中的基因表达的组合物和方法
TW201803989A (zh) 拮抗ｂ型肝炎病毒之組成物和藥劑及彼等之用途
JP2020529197A5 (https=)
JPWO2021202511A5 (https=)
JPWO2021188795A5 (https=)
US20240191228A1 (en) Antisense oligonucleotides for treatment of neurological disorders
BR112021003609A2 (pt) produtos e composições
TW202228729A (zh) 選擇性遞送寡核苷酸至膠細胞
JPWO2021119226A5 (https=)
WO2025098117A1 (zh) 一种抑制甲状腺素转运蛋白(TTR)表达的RNAi试剂、组合物及应用
JP2022520885A (ja) 神経変性疾患を処置するための組成物および方法
JPWO2021087036A5 (https=)
JPWO2022031847A5 (https=)
KR101783444B1 (ko) miR-33-5p 를 이용한 뇌신경세포 보호 물질 스크리닝 방법
JP2025531512A (ja) アルファシヌクレイン核酸を標的化するｓｉＲＮＡ組成物及び方法
KR20230161366A (ko) 금속 나노 입자-핵산 결합체를 기반으로 하는 유전자 운반체
CN119630791A (zh) 用于抑制snca表达的组合物和方法
JPWO2022218941A5 (https=)
CN116670276A (zh) 用于通过多个靶标治疗疼痛的治疗组合物
TW202136510A (zh) 用於抑制ｓｃｎ９ａ表現之增強型寡核苷酸
US20240287512A1 (en) Lipid conjugation for targeting neurons of the central nervous system
WO2007072909A1 (ja) 新規オリゴヌクレオチド及びそれから成るNF-κBデコイ
JPWO2023278607A5 (https=)