JP2019526555A5 - - Google Patents

Download PDF

Info

Publication number
JP2019526555A5
JP2019526555A5 JP2019508973A JP2019508973A JP2019526555A5 JP 2019526555 A5 JP2019526555 A5 JP 2019526555A5 JP 2019508973 A JP2019508973 A JP 2019508973A JP 2019508973 A JP2019508973 A JP 2019508973A JP 2019526555 A5 JP2019526555 A5 JP 2019526555A5
Authority
JP
Japan
Prior art keywords
substituted
unsubstituted
formula
hydrogen
glutathione
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.)
Granted
Application number
JP2019508973A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019526555A (ja
JP7249080B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2017/048239 external-priority patent/WO2018039364A1/en
Publication of JP2019526555A publication Critical patent/JP2019526555A/ja
Publication of JP2019526555A5 publication Critical patent/JP2019526555A5/ja
Priority to JP2022076983A priority Critical patent/JP2022106928A/ja
Application granted granted Critical
Publication of JP7249080B2 publication Critical patent/JP7249080B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2019508973A 2016-08-23 2017-08-23 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 Active JP7249080B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022076983A JP2022106928A (ja) 2016-08-23 2022-05-09 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

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662378635P 2016-08-23 2016-08-23
US62/378,635 2016-08-23
PCT/US2017/048239 WO2018039364A1 (en) 2016-08-23 2017-08-23 Compositions comprising reversibly modified oligonucleotides and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022076983A Division JP2022106928A (ja) 2016-08-23 2022-05-09 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

Publications (3)

Publication Number Publication Date
JP2019526555A JP2019526555A (ja) 2019-09-19
JP2019526555A5 true JP2019526555A5 (enExample) 2020-10-01
JP7249080B2 JP7249080B2 (ja) 2023-03-30

Family

ID=61245366

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019508973A Active JP7249080B2 (ja) 2016-08-23 2017-08-23 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
JP2022076983A Withdrawn JP2022106928A (ja) 2016-08-23 2022-05-09 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022076983A Withdrawn JP2022106928A (ja) 2016-08-23 2022-05-09 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

Country Status (10)

Country Link
US (2) US11390642B2 (enExample)
EP (1) EP3503730B1 (enExample)
JP (2) JP7249080B2 (enExample)
KR (1) KR102530513B1 (enExample)
CN (2) CN114891053A (enExample)
AU (2) AU2017315670B2 (enExample)
CA (1) CA3032165C (enExample)
IL (2) IL264685B (enExample)
MX (2) MX2019002075A (enExample)
WO (1) WO2018039364A1 (enExample)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
JP2021509669A (ja) 2018-01-05 2021-04-01 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 免疫療法を強化するためのベータ−カテニン及びidoの発現の低減
WO2019143621A1 (en) 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression
WO2020206350A1 (en) 2019-04-04 2020-10-08 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression in the central nervous system
WO2021177657A1 (ko) * 2020-03-03 2021-09-10 주식회사 넥스모스 글루타치온에 특이적으로 결합하는 dna 압타머 및 그의 용도
CA3187220A1 (en) 2020-08-04 2022-02-10 Dicerna Pharmaceuticals, Inc. Systemic delivery of oligonucleotides
MX2023004109A (es) 2020-10-08 2023-06-28 Dicerna Pharmaceuticals Inc Administracion selectiva de oligonucleótidos a células gliales.
EP4271695A2 (en) * 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
WO2022221430A1 (en) 2021-04-14 2022-10-20 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating pnpla3 expression
US12465647B2 (en) 2021-08-05 2025-11-11 Sanegene Bio Usa Inc. 1′-alkyl modified ribose derivatives and methods of use
AU2022352676A1 (en) 2021-09-22 2024-03-14 Sanegene Bio Usa Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
WO2023047338A1 (en) * 2021-09-24 2023-03-30 Crispr Therapeutics Ag PRODRUG INCORPORATED sgRNA SYNTHESIS
JP2024542929A (ja) 2021-10-05 2024-11-19 セーンジーン バイオ ユーエスエー インコーポレイティド ポリヒドロキシル化されたシクロペンタン誘導体及び使用方法
EP4441218A2 (en) 2021-12-01 2024-10-09 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating apoc3 expression
JP2025508785A (ja) 2022-02-22 2025-04-10 セーンジーン バイオ ユーエスエー インコーポレイティド 5’修飾炭素環式リボヌクレオチド誘導体及び使用方法
CA3245064A1 (en) 2022-04-15 2023-10-19 Dicerna Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF MODULATING THE ACTIVITY OF STRUCTURAL AND CYTOSKELETON-ASSOCIATED PROTEINS
WO2023220561A1 (en) 2022-05-09 2023-11-16 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
WO2024015796A1 (en) 2022-07-11 2024-01-18 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
AU2023320578A1 (en) 2022-08-05 2025-02-13 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
IL320218A (en) 2022-10-14 2025-06-01 Sanegene Bio Usa Inc Small interfering RNA targeting C3 and its uses
WO2024112800A1 (en) * 2022-11-22 2024-05-30 Ionis Pharmaceuticals, Inc. Methods for deprotecting and purifying oligonucleotide compounds
CN120569477A (zh) 2022-12-19 2025-08-29 美国圣因生物股份有限公司 靶向cfb的小干扰rna及其用途
US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
WO2024186673A2 (en) 2023-03-03 2024-09-12 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
EP4731764A2 (en) 2023-06-21 2026-04-29 Sanegene Bio USA Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
US20250064939A1 (en) 2023-07-24 2025-02-27 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
WO2026017140A2 (en) 2024-07-18 2026-01-22 Sanegene Bio Usa Inc. Small interfering rna targeting fxi and uses thereof
WO2026041067A2 (en) 2024-08-20 2026-02-26 Sanegene Bio Usa Inc. Small interfering rna targeting myostatin (mstn) and uses thereof
WO2026046411A1 (en) 2024-09-02 2026-03-05 Sanegene Bio Usa Inc. Small interfering rna targeting plasminogen (plg) and uses thereof
WO2026092658A2 (en) 2024-10-31 2026-05-07 Sanegene Bio Usa Inc. Small interfering rna targeting amyloid beta precursor protein (app) and uses thereof

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6114513A (en) * 1990-01-11 2000-09-05 Isis Pharmaceuticals, Inc. Thiol-derivatized oligonucleotides
JPH05505941A (ja) 1990-03-29 1993-09-02 ギリード サイエンシズ インコーポレーテッド オリゴヌクレオチド―輸送剤ジスルフィド結合体
US7282590B2 (en) 2004-02-12 2007-10-16 The Research Foundation Of State University Of New York Drug conjugates
KR20100037120A (ko) 2007-07-06 2010-04-08 노스이스턴 유니버시티 Rna 작용제의 양쪽성 컨주게이트를 포함하는 혼합 미쎌, 및 이의 용도
EP3067359A1 (en) 2008-09-23 2016-09-14 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2013188607A1 (en) * 2012-06-14 2013-12-19 The Trustees Of The University Of Pennsylvania One step n-terminal tagging of proteins
JP2016507484A (ja) 2012-12-06 2016-03-10 メルク・シャープ・アンド・ドーム・コーポレーションMerck Sharp & Dohme Corp. ジスルフィドマスキングプロドラッグ組成物および方法
CA2892676A1 (en) 2012-12-06 2014-06-12 Merck Sharp & Dohme Corp. Nucleoside kinase bypass compositions and methods
EP2845607A1 (en) 2013-09-09 2015-03-11 University of Vienna Antisense oligonucleotides with improved pharmacokinetic properties
JP2016537027A (ja) * 2013-11-06 2016-12-01 ソルスティス バイオロジクス,リミティッド ジスルフィド基を有するポリヌクレオチド構築物
EP3152308A4 (en) * 2014-06-06 2017-12-27 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups

Similar Documents

Publication Publication Date Title
JP6884250B2 (ja) ペプチドオリゴヌクレオチドコンジュゲート
US20220402883A1 (en) Compounds and methods for modulating rna function
US10041074B2 (en) Euchromatic region targeting methods for modulating gene expression
BR112019026508A2 (pt) métodos para modificar o splicing do rna
JP2022547888A5 (enExample)
JP7348922B2 (ja) B型肝炎感染の治療用のPAPD5及びPAPD7 mRNAを低減させるための核酸分子
CN113840602A (zh) 用于校正异常剪接的组合物和方法
RU2017101172A (ru) Антисмысловые нуклеиновые кислоты
JP2020534826A (ja) 真核細胞において遺伝子発現をサイレンシングするための非コードrna分子の特異性の改変
CA3072205A1 (en) Methods and compositions for modulating splicing
CN114007612A (zh) 用于调节剪接的方法和组合物
RS54649B1 (sr) Antisens nukleinske kiseline
KR20190040033A (ko) 가역적으로 변형된 올리고뉴클레오티드를 포함하는 조성물 및 그의 용도
JP2021517126A5 (enExample)
JP2015504650A5 (enExample)
CA2939822A1 (en) Crosslinked nucleoside and nucleotide
JP2011529500A5 (enExample)
JP2015512254A (ja) オリゴヌクレオチドアナログのボロン酸結合体
JP2022513111A5 (enExample)
CY1105752T1 (el) Παραγωγα ιμιδαζοπυριδινης μεθοδος παρασκευης αυτων και οι φαρμακευτικες συνθεσεις που τα πepιεχουν
ES2586599T3 (es) Ensayo para cribar compuestos que disminuyen selectivamente el número de células madre de cáncer
Pandey RNA-based regulation in human health and disease
Andersen et al. Efficient RNA-targeting by the introduction of aromatic stacking in the duplex major groove via 5-(1-phenyl-1, 2, 3-triazol-4-yl)-2′-deoxyuridines
Bertrand et al. The snoRNPs and Related Machines: Ancient Devices That Mediate Maturation of rRNA
TW202521694A (zh) 凝血因子xi調節組合物及其使用方法