JP2022551269A5 - - Google Patents

Info

Publication number
JP2022551269A5
JP2022551269A5 JP2022520472A JP2022520472A JP2022551269A5 JP 2022551269 A5 JP2022551269 A5 JP 2022551269A5 JP 2022520472 A JP2022520472 A JP 2022520472A JP 2022520472 A JP2022520472 A JP 2022520472A JP 2022551269 A5 JP2022551269 A5 JP 2022551269A5
Authority
JP
Japan
Prior art keywords
nucleotides
modified
positions
oligonucleotide according
antisense
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.)
Withdrawn
Application number
JP2022520472A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022551269A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/053999 external-priority patent/WO2021067744A1/en
Publication of JP2022551269A publication Critical patent/JP2022551269A/ja
Publication of JP2022551269A5 publication Critical patent/JP2022551269A5/ja
Withdrawn legal-status Critical Current

Links

JP2022520472A 2019-10-02 2020-10-02 最小フッ素含有量を用いた低分子干渉rnaの化学修飾 Withdrawn JP2022551269A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962909278P 2019-10-02 2019-10-02
US62/909,278 2019-10-02
PCT/US2020/053999 WO2021067744A1 (en) 2019-10-02 2020-10-02 Chemical modifications of small interfering rna with minimal fluorine content

Publications (2)

Publication Number Publication Date
JP2022551269A JP2022551269A (ja) 2022-12-08
JP2022551269A5 true JP2022551269A5 (https=) 2023-10-25

Family

ID=73038388

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022520472A Withdrawn JP2022551269A (ja) 2019-10-02 2020-10-02 最小フッ素含有量を用いた低分子干渉rnaの化学修飾

Country Status (12)

Country Link
US (1) US20220389430A1 (https=)
EP (1) EP4038191A1 (https=)
JP (1) JP2022551269A (https=)
KR (1) KR20220069103A (https=)
CN (1) CN114761557B (https=)
AU (1) AU2020358016A1 (https=)
CA (1) CA3153026A1 (https=)
CL (1) CL2022000825A1 (https=)
IL (1) IL291841A (https=)
MX (1) MX2022004032A (https=)
TW (1) TW202126809A (https=)
WO (1) WO2021067744A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PE20250400A1 (es) 2021-04-12 2025-02-11 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir cetohexoquinasa
CA3209418A1 (en) * 2021-04-14 2022-10-20 Utsav SAXENA Compositions and methods for modulating pnpla3 expression
EP4372085A4 (en) * 2021-07-16 2026-01-21 Suzhou Ribo Life Science Co Ltd Nucleic acid, composition and conjugate, container, preparation process and use
EP4405368A1 (en) * 2021-09-22 2024-07-31 Sanegene Bio USA Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
WO2023069495A1 (en) * 2021-10-19 2023-04-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides with 2'-deoxy-2'-f-2'-c-methyl nucleotides
JP2024543195A (ja) 2021-12-01 2024-11-19 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 Apoc3発現を調節するための組成物及び方法
CN119183476A (zh) * 2022-04-26 2024-12-24 上海拓界生物医药科技有限公司 氘代化学修饰和包含其的寡核苷酸
TW202526017A (zh) * 2023-09-14 2025-07-01 美商Ionis製藥公司 用於減少apociii表現的化合物及方法
WO2025191337A2 (en) * 2024-03-11 2025-09-18 Synerk Biotech Limited Oligonucleotide terminal phosphate derivatives and method of use

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
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
DK2341943T3 (en) 2008-09-22 2019-02-25 Dicerna Pharmaceuticals Inc COMPOSITIONS AND METHODS OF SPECIFIC INHIBITION OF DSRNA REPRINT WITH MODIFICATIONS
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
KR101728655B1 (ko) 2008-12-18 2017-04-19 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
EP2928877B1 (en) 2012-12-06 2020-01-22 Merck Sharp & Dohme Corp. Disulfide masked prodrug compositions and methods
CA2950960A1 (en) 2014-06-06 2015-12-10 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
WO2019075419A1 (en) * 2017-10-13 2019-04-18 Dicerna Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION
UA128786C2 (uk) * 2017-10-20 2024-10-23 Дайсерна Фармасьютикалз, Інк Олігонуклеотид для лікування інфекції гепатиту в

Similar Documents

Publication Publication Date Title
JP2022551269A5 (https=)
TWI823866B (zh) 用於抑制類血管生成素蛋白-3(ANGPTL3)表現的RNAi藥劑及組合物及使用方法
CN113164509B (zh) 用于抑制17β-HSD 13型(HSD17B13)表达的RNAi试剂、其组合物和使用方法
KR102473431B1 (ko) 안티센스 핵산
ES2318106T3 (es) Oligonucleotidos que comprenden segmentos alternos y usos de los mismos.
CN110832077B (zh) 用于抑制α-ENaC表达的RNAi剂及使用方法
TW202307207A (zh) 用於抑制黃嘌呤脫氫酶(XDH)之表現之RNAi藥劑、其醫藥組合物及使用方法
JP2022551269A (ja) 最小フッ素含有量を用いた低分子干渉rnaの化学修飾
CN115397436A (zh) 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
JP2024505035A (ja) 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および方法
JP2018109015A (ja) 三環ヌクレオシドおよびこれらから調製したオリゴマー化合物
TW202334416A (zh) 用於抑制基質金屬蛋白酶7 (MMP7)之表現的RNAi藥劑、其組成物及使用方法
JP2025522429A (ja) 中枢神経系組織への治療薬の送達のための脂質結合体
CN111212909A (zh) 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物
WO2023011360A1 (zh) 一种增强核酸药物缓释能力的方法
US20260049309A1 (en) Regulation of activity of rnai molecules
WO2020181130A2 (en) Polynucleotide conjugates and uses thereof
JP6826984B2 (ja) アシル−アミノ−lnaオリゴヌクレオチドおよび/またはヒドロカルビル−アミノ−lnaオリゴヌクレオチド
JP2022520653A5 (https=)
US20260125682A1 (en) Chemical modifications of small interfering rna with minimal fluorine content
JP7216381B2 (ja) Rna作用抑制剤及びその利用
EP4219717A2 (en) Exon skipping oligomers for muscular dystrophy
WO2024233864A2 (en) Galnac-conjugated rnai oligonucleotides
TW202430635A (zh) 用於抑制補體成分C3(C3)表現之RNAi藥劑、其醫藥組合物及使用方法
HK40084771B (zh) 用於抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法