JP2012509258A5 - - Google Patents

Download PDF

Info

Publication number
JP2012509258A5
JP2012509258A5 JP2011536348A JP2011536348A JP2012509258A5 JP 2012509258 A5 JP2012509258 A5 JP 2012509258A5 JP 2011536348 A JP2011536348 A JP 2011536348A JP 2011536348 A JP2011536348 A JP 2011536348A JP 2012509258 A5 JP2012509258 A5 JP 2012509258A5
Authority
JP
Japan
Prior art keywords
independently
substituted
alkyl
peg
cationic lipid
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Withdrawn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
JP2011536348A
Other languages
English (en)
Japanese (ja)
Other versions
JP2012509258A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2009/052462 external-priority patent/WO2010056403A1/en
Publication of JP2012509258A publication Critical patent/JP2012509258A/ja
Publication of JP2012509258A5 publication Critical patent/JP2012509258A5/ja
Withdrawn legal-status Critical Current

Links

JP2011536348A 2008-11-17 2009-07-31 核酸送達系のための分岐カチオン性脂質 Withdrawn JP2012509258A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11530708P 2008-11-17 2008-11-17
US61/115,307 2008-11-17
PCT/US2009/052462 WO2010056403A1 (en) 2008-11-17 2009-07-31 Branched cationic lipids for nucleic acids delivery system

Publications (2)

Publication Number Publication Date
JP2012509258A JP2012509258A (ja) 2012-04-19
JP2012509258A5 true JP2012509258A5 (https=) 2012-07-19

Family

ID=42170237

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2011536348A Withdrawn JP2012509258A (ja) 2008-11-17 2009-07-31 核酸送達系のための分岐カチオン性脂質

Country Status (7)

Country Link
US (1) US20110305769A1 (https=)
EP (1) EP2350296A4 (https=)
JP (1) JP2012509258A (https=)
CN (1) CN102216462A (https=)
CA (1) CA2742689A1 (https=)
TW (1) TW201019969A (https=)
WO (1) WO2010056403A1 (https=)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201021853A (en) * 2008-11-17 2010-06-16 Enzon Pharmaceuticals Inc Releasable cationic lipids for nucleic acids delivery systems
HUE042177T2 (hu) 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
EP2527440A1 (en) * 2011-05-27 2012-11-28 Institut Curie Cancer treatment by combining DNA molecules mimicking double strand breaks with hyperthermia
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
JP5950477B2 (ja) 2011-08-10 2016-07-13 アドシア 少なくとも1種の基礎インスリンの注射溶液
CA2851280C (en) 2011-10-11 2021-05-18 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
US9579338B2 (en) * 2011-11-04 2017-02-28 Nitto Denko Corporation Method of producing lipid nanoparticles for drug delivery
CN102516534B (zh) * 2011-11-14 2013-11-20 上海交通大学 可降解为精胺的聚阳离子及其合成方法、纳米颗粒
WO2013104861A1 (fr) 2012-01-09 2013-07-18 Adocia Solution injectable a ph 7 comprenant au moins une insuline basale dont le pi est compris entre 5,8 et 8,5 et un co-polyaminoacide substitue
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
US10245229B2 (en) * 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
US20150314003A2 (en) 2012-08-09 2015-11-05 Adocia Injectable solution at ph 7 comprising at least one basal insulin the isoelectric point of which is between 5.8 and 8.5 and a hydrophobized anionic polymer
GB201215289D0 (en) * 2012-08-28 2012-10-10 Medical Res Council Nanoparticle formulation
US9687448B2 (en) 2012-12-07 2017-06-27 Alnylam Pharmaceuticals, Inc. Nucleic acid lipid particle formulations
PL3467108T3 (pl) 2013-03-14 2024-09-30 Translate Bio, Inc. Sposoby oczyszczania informacyjnego rna
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
MX2016005238A (es) * 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
PT3134506T (pt) 2014-04-25 2019-10-31 Translate Bio Inc Métodos de purificação de rna mensageiro
CN104922068B (zh) * 2015-05-22 2017-10-13 江苏凯基生物技术股份有限公司 一种Decoy核酸阳离子脂质体载体及其制备方法
JP6457696B2 (ja) 2015-07-23 2019-01-23 アンスティテュ・キュリInstitut Curie 癌を処置するためのDbait分子とPARPインヒビターとの組合せの使用
EP3349802B1 (en) * 2015-09-14 2021-08-04 The Board of Regents of the University of Texas System Lipocationic dendrimers and uses thereof
EP3368089B1 (en) 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
US10654875B2 (en) 2015-11-12 2020-05-19 The Board Of Trustees Of The Leland Stanford Junior University Cell-penetrating, guanidinium-rich oligophosphotriesters for drug and probe delivery
CN107951862B (zh) * 2016-10-17 2021-03-12 南京绿叶制药有限公司 一种抑制bcl-2的反义寡聚核酸的脂质纳米粒及其制备方法
CA3037046A1 (en) 2016-10-31 2018-05-03 University Of Massachusetts Targeting microrna-101-3p in cancer therapy
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
KR102778110B1 (ko) * 2017-04-20 2025-03-10 신테나 아게 트리시클로-dna 뉴클레오시드를 포함하는 변형 올리고머 화합물 및 그의 용도
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
EP3662913A4 (en) * 2017-08-04 2021-06-30 Kyowa Kirin Co., Ltd. Nucleic-acid-containing lipid nanoparticles
US10435429B2 (en) 2017-10-03 2019-10-08 Nucorion Pharmaceuticals, Inc. 5-fluorouridine monophosphate cyclic triester compounds
EP3752511A4 (en) * 2018-01-10 2021-12-29 Nucorion Pharmaceuticals, Inc. Phosphor(n)amidatacetal and phosph(on)atalcetal compounds
US11427550B2 (en) 2018-01-19 2022-08-30 Nucorion Pharmaceuticals, Inc. 5-fluorouracil compounds
WO2019183000A1 (en) * 2018-03-19 2019-09-26 University Of Massachusetts Modified guide rnas for crispr genome editing
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
CN112789031B (zh) * 2018-09-28 2023-03-24 胡桃钳医疗公司 用于核酸递送的包含脂化的阳离子肽化合物的脂质纳米颗粒制剂
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
CN114206896B (zh) 2019-07-17 2024-12-17 纽科利制药公司 环状脱氧核糖核苷酸化合物
US20230098031A1 (en) * 2019-12-11 2023-03-30 The General Hospital Corporation Methods for cell imaging
EP4087619A4 (en) * 2020-01-07 2024-02-21 Translate Bio MA, Inc. FORMULATIONS FOR THE DELIVERY OF OLIGONUCLEOTIDES TO LUNG CELLS
KR20220130696A (ko) * 2020-01-08 2022-09-27 마이크로바스큘러 테라퓨틱스 엘엘씨 알킬화 뉴클레오사이드, 및 핵산 전달을 위한 조성물 및 그 방법
KR20230002300A (ko) * 2020-01-21 2023-01-05 이더알엔에이 이뮤노테라피스 엔브이 지질 나노입자
EP3865122A1 (en) * 2020-02-11 2021-08-18 Pantherna Therapeutics GmbH Lipid composition and use thereof for delivery of a therapeutically active agent to endothelium
US12552820B2 (en) 2020-04-21 2026-02-17 Ligand Pharmaceuticals Incorporated Benzyloxy phosph(on)ate compounds
CN115605492B (zh) 2020-04-21 2025-09-16 配体制药股份有限公司 核苷酸前药化合物
CN114315606B (zh) * 2020-12-29 2023-12-26 李斌 一种脂样分子及其应用
KR20240008872A (ko) * 2021-05-06 2024-01-19 아크투루스 쎄라퓨틱스, 인크. Rna 전달을 위한 이온화 가능한 양이온성 지질
US20250127719A1 (en) * 2021-12-07 2025-04-24 The Trustees Of The University Of Pennsylvania Anisamide-containing lipids and compositions and methods of use thereof
CN114306369B (zh) * 2021-12-23 2023-12-26 北京悦康科创医药科技股份有限公司 一种硫代寡核苷酸注射液及其制备方法

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5972600A (en) * 1992-04-03 1999-10-26 The Regents Of The University Of California Separation of active complexes
US5777153A (en) * 1994-07-08 1998-07-07 Gilead Sciences, Inc. Cationic lipids
US5830430A (en) * 1995-02-21 1998-11-03 Imarx Pharmaceutical Corp. Cationic lipids and the use thereof
ATE198599T1 (de) * 1996-03-01 2001-01-15 Centre Nat Rech Scient Guanidinium-verbindungen, pharmazeutische präparate davon und ihre verwendungen
DE69815707T2 (de) * 1997-11-20 2004-07-01 Vical, Inc., San Diego Behandlung von krebs durch verwendung von zytokin-exprimierender polynukleotiden und zusammensetzungen dafür
US6320017B1 (en) * 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
EP1572970A4 (en) * 2002-11-14 2006-11-15 Genta Salus Llc OLIOGONUCLEOTIDES INHIBITORS DIRECTED ON BCL-2
US7713738B2 (en) * 2003-02-10 2010-05-11 Enzon Pharmaceuticals, Inc. Oligomeric compounds for the modulation of survivin expression
TWI257924B (en) * 2003-09-22 2006-07-11 Lipotek Inc Cationic lipid for delivery function, and nanoparticle comprising the same
WO2007056861A1 (en) * 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Sirna silencing of influenza virus gene expression
WO2008043366A2 (en) * 2006-10-13 2008-04-17 Københavns Universitet Three-domain compounds for transmembrane delivery
CN102231950A (zh) * 2008-11-17 2011-11-02 安龙制药公司 用于核酸递送系统的可释放聚合脂质
TW201021853A (en) * 2008-11-17 2010-06-16 Enzon Pharmaceuticals Inc Releasable cationic lipids for nucleic acids delivery systems

Similar Documents

Publication Publication Date Title
JP2012509258A5 (https=)
JP2011529912A5 (https=)
JP2011520983A5 (https=)
JP2012509366A5 (https=)
JP2012509273A5 (https=)
DK2431466T3 (en) Single-stranded nucleic acid molecule for the control of gene expression
US9074208B2 (en) Modified siRNA molecules and uses thereof
US9006191B2 (en) Silencing of polo-like kinase expression using interfering RNA
JP2012509272A5 (https=)
WO2007056861A1 (en) Sirna silencing of influenza virus gene expression
AU2016201913A1 (en) Compositions and methods for silencing aldehyde dehydrogenase
CA2979998A1 (en) Compositions and methods for treating hypertriglyceridemia
US9035039B2 (en) Compositions and methods for silencing SMAD4
JP2012532839A5 (https=)
RU2010144531A (ru) Модифицированная липидами двухцепочечная рнк, обладающая мощным эффектом рнк-интерференции
JPWO2020041769A5 (https=)
JP2017500277A5 (https=)
AU2013203219B2 (en) MODIFIED siRNA MOLECULES AND USES THEREOF
RU2010136047A (ru) КАТИОННЫЕ siPНК, СИНТЕЗ И ПРИМЕНЕНИЕ В РНК-ИНТЕРФЕРЕНЦИИ
CN120077134A (zh) 靶向apoc3的多核酸分子及其用途
Zenkova et al. Site-specific artificial ribonucleases: conjugates of oligonucleotides with catalytic groups
JPWO2023165595A5 (https=)
AU2013202970A1 (en) Silencing of polo-like kinase expression using interfering RNA
RU2013114396A (ru) Антисмысловые нуклеиновые кислоты