JP2025501682A5 - - Google Patents

Info

Publication number
JP2025501682A5
JP2025501682A5 JP2024532799A JP2024532799A JP2025501682A5 JP 2025501682 A5 JP2025501682 A5 JP 2025501682A5 JP 2024532799 A JP2024532799 A JP 2024532799A JP 2024532799 A JP2024532799 A JP 2024532799A JP 2025501682 A5 JP2025501682 A5 JP 2025501682A5
Authority
JP
Japan
Prior art keywords
gapmer oligonucleotide
inventions
oligonucleotide according
spacer
gapmer
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
JP2024532799A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025501682A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/051663 external-priority patent/WO2023102188A1/en
Publication of JP2025501682A publication Critical patent/JP2025501682A/ja
Publication of JP2025501682A5 publication Critical patent/JP2025501682A5/ja
Pending legal-status Critical Current

Links

JP2024532799A 2021-12-03 2022-12-02 改変された骨格化学を有するギャップマーアンチセンスオリゴヌクレオチド Pending JP2025501682A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202163285696P 2021-12-03 2021-12-03
US202163285692P 2021-12-03 2021-12-03
US202163285888P 2021-12-03 2021-12-03
US202163285705P 2021-12-03 2021-12-03
US202163285665P 2021-12-03 2021-12-03
US63/285,692 2021-12-03
US63/285,696 2021-12-03
US63/285,888 2021-12-03
US63/285,705 2021-12-03
US63/285,665 2021-12-03
PCT/US2022/051663 WO2023102188A1 (en) 2021-12-03 2022-12-02 Gapmer antisense oligonucleotides with modified backbone chemistries

Publications (2)

Publication Number Publication Date
JP2025501682A JP2025501682A (ja) 2025-01-23
JP2025501682A5 true JP2025501682A5 (enExample) 2025-12-10

Family

ID=86613042

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024532799A Pending JP2025501682A (ja) 2021-12-03 2022-12-02 改変された骨格化学を有するギャップマーアンチセンスオリゴヌクレオチド

Country Status (5)

Country Link
EP (1) EP4441224A4 (enExample)
JP (1) JP2025501682A (enExample)
CN (1) CN119452086A (enExample)
CA (1) CA3241316A1 (enExample)
WO (1) WO2023102188A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240126870A (ko) 2021-12-22 2024-08-21 캠프4 테라퓨틱스 코포레이션 조절 rna들을 표적으로 하는 안티센스 올리고뉴클레오티드를 사용한 유전자 전사의 조절

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08504559A (ja) 1992-12-14 1996-05-14 ハネウエル・インコーポレーテッド 個別に制御される冗長巻線を有するモータシステム
ATE227342T1 (de) * 1993-09-02 2002-11-15 Ribozyme Pharm Inc Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
JP4236812B2 (ja) 1997-09-12 2009-03-11 エクシコン エ／エス オリゴヌクレオチド類似体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
AU2003291755A1 (en) 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
CA2538252C (en) 2003-09-18 2014-02-25 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
ZA200707490B (en) * 2005-03-10 2008-12-31 Genentech Inc Methods and compositions for modulatiing vascular integrity
WO2007090071A2 (en) 2006-01-27 2007-08-09 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
KR101221589B1 (ko) * 2006-04-07 2013-01-15 이데라 파마슈티칼즈, 인코포레이티드 Ｔｌｒ７ 및 ｔｌｒ８에 대한 안정화된 면역 조절성 ｒｎａ〔ｓｉｍｒａ〕 화합물
EP2505648A1 (en) * 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of PTP1B
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
DK2152879T3 (da) * 2007-05-01 2013-02-18 Santaris Pharma As RNA antagonistforbindelser til modulering af beta-catenin
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
KR101110013B1 (ko) * 2007-10-05 2012-02-29 (주)바이오니아 서열 내에 어베이직 부분을 포함하는 ｐｃｒ 증폭용프라이머
RU2593939C2 (ru) 2008-11-13 2016-08-10 Ногра Фарма Лимитед Антисмысловые композиции и способы их получения и применения
AU2012308320C1 (en) * 2011-09-14 2018-08-23 Translate Bio Ma, Inc. Multimeric oligonucleotide compounds
US20150247141A1 (en) * 2012-09-14 2015-09-03 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
CN104837996A (zh) * 2012-11-15 2015-08-12 罗氏创新中心哥本哈根有限公司 抗apob反义缀合物化合物
EP2959000B1 (en) * 2013-02-25 2018-08-15 Integrated DNA Technologies Inc. Naphthyl-azo modifications for antisense compounds
DK2992098T3 (da) 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
WO2016024205A1 (en) * 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
PH12018501207B1 (en) * 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression
AR115847A1 (es) * 2018-07-25 2021-03-03 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de la atxn2
JP2022537581A (ja) * 2019-06-21 2022-08-26 クラリス コーポレーション Ｐｐｍ１阻害剤およびその使用方法

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