HK1208227A1 - Novel tetragalnac containing conjugates and methods for delivery of oligonucleotides - Google Patents
Novel tetragalnac containing conjugates and methods for delivery of oligonucleotides Download PDFInfo
- Publication number
- HK1208227A1 HK1208227A1 HK15108687.4A HK15108687A HK1208227A1 HK 1208227 A1 HK1208227 A1 HK 1208227A1 HK 15108687 A HK15108687 A HK 15108687A HK 1208227 A1 HK1208227 A1 HK 1208227A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- tetragalnac
- oligonucleotides
- delivery
- novel
- methods
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
- C07H15/26—Acyclic or carbocyclic radicals, substituted by hetero rings
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Immunology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Transplantation (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261641761P | 2012-05-02 | 2012-05-02 | |
| US201261641761P | 2012-05-02 | ||
| PCT/US2013/039025 WO2013166121A1 (fr) | 2012-05-02 | 2013-05-01 | Nouveaux conjugués contenant tétragalnac et procédés pour l'administration d'oligonucléotides |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HK1208227A1 true HK1208227A1 (en) | 2016-02-26 |
Family
ID=49514844
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HK15108687.4A HK1208227A1 (en) | 2012-05-02 | 2013-05-01 | Novel tetragalnac containing conjugates and methods for delivery of oligonucleotides |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US9540639B2 (fr) |
| EP (1) | EP2844663B1 (fr) |
| JP (2) | JP6239592B2 (fr) |
| AR (1) | AR090906A1 (fr) |
| AU (1) | AU2013256400B2 (fr) |
| CA (1) | CA2872100A1 (fr) |
| HK (1) | HK1208227A1 (fr) |
| TW (2) | TWI595885B (fr) |
| WO (1) | WO2013166121A1 (fr) |
Families Citing this family (58)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10023861B2 (en) | 2011-08-29 | 2018-07-17 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| AR090905A1 (es) * | 2012-05-02 | 2014-12-17 | Merck Sharp & Dohme | Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica |
| US9127276B2 (en) | 2013-05-01 | 2015-09-08 | Isis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| US10808246B2 (en) * | 2013-07-11 | 2020-10-20 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
| US10239957B2 (en) * | 2013-11-06 | 2019-03-26 | Merck Sharp & Dohme Corp. | Peptide containing conjugates for dual molecular delivery of oligonucleotides |
| EP3065783A4 (fr) * | 2013-11-06 | 2017-06-21 | Merck Sharp & Dohme Corp. | Double distribution moléculaire de conjugués contenant des oligonucléotides et des peptides |
| JP6466637B2 (ja) * | 2013-11-28 | 2019-02-06 | ソマール株式会社 | 糖鎖含有ポリマー、および、糖鎖含有ポリマー複合体 |
| DK3137476T3 (da) | 2014-04-28 | 2019-11-18 | Ionis Pharmaceuticals Inc | Linker-modificerede oligomerforbindelser |
| CN110903337A (zh) | 2014-05-01 | 2020-03-24 | Ionis制药公司 | 用于调节生长激素受体表达的组合物和方法 |
| CN106255755B (zh) | 2014-05-01 | 2020-07-24 | Ionis制药公司 | 用于调节血管生成素样蛋白3表达的组合物和方法 |
| PH12016502062B1 (en) * | 2014-05-01 | 2023-01-27 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating complement factor b expression |
| EP3137091B1 (fr) | 2014-05-01 | 2020-12-02 | Ionis Pharmaceuticals, Inc. | Conjugués d'oligonucléotides antisens et leur utilisation pour moduler l'expression de pkk |
| WO2015179693A1 (fr) | 2014-05-22 | 2015-11-26 | Isis Pharmaceuticals, Inc. | Composés antisens conjugués et leur utilisation |
| WO2016040748A1 (fr) | 2014-09-12 | 2016-03-17 | Ionis Pharmaceuticals, Inc. | Compositions et procédés de détection d'une protéine smn chez un patient et traitement d'un patient |
| EP3204497B1 (fr) | 2014-10-10 | 2020-03-11 | Dicerna Pharmaceuticals, Inc. | Inhibition thérapeutique de la lactate-déshydrogénase et agents associés |
| US20180312845A1 (en) | 2015-07-10 | 2018-11-01 | Ionis Pharmaceuticals, Inc. | Modulators of diacyglycerol acyltransferase 2 (dgat2) |
| CN108513588A (zh) | 2015-09-24 | 2018-09-07 | Ionis制药公司 | Kras表达的调节剂 |
| CN113952353A (zh) | 2015-11-06 | 2022-01-21 | Ionis制药公司 | 调节载脂蛋白(a)表达 |
| PT4119569T (pt) | 2015-11-06 | 2024-10-23 | Ionis Pharmaceuticals Inc | Compostos antissentido conjugados para utilização em terapia |
| CA3012967A1 (fr) | 2016-01-29 | 2017-08-03 | Kyowa Hakko Kirin Co., Ltd. | Conjugue d'acide nucleique |
| KR102515329B1 (ko) | 2016-03-07 | 2023-03-29 | 애로우헤드 파마슈티컬스 인코포레이티드 | 치료용 화합물을 위한 표적화 리간드 |
| WO2018013525A1 (fr) | 2016-07-11 | 2018-01-18 | Translate Bio Ma, Inc. | Conjugués de type acide nucléique et leurs utilisations |
| DK3484524T3 (da) | 2016-07-15 | 2022-12-12 | Ionis Pharmaceuticals Inc | Forbindelser og fremgangsmåder til modulation af smn2 |
| JOP20170056B1 (ar) | 2016-09-02 | 2021-08-17 | Arrowhead Pharmaceuticals Inc | مركبات ترابطية مستهدفة |
| KR20190065341A (ko) | 2016-10-06 | 2019-06-11 | 아이오니스 파마수티컬즈, 인코포레이티드 | 올리고머 화합물들의 접합 방법 |
| JOP20190215A1 (ar) | 2017-03-24 | 2019-09-19 | Ionis Pharmaceuticals Inc | مُعدّلات التعبير الوراثي عن pcsk9 |
| KR20230169413A (ko) | 2017-10-13 | 2023-12-15 | 노보 노르디스크 헬스 케어 악티엔게젤샤프트 | Ldha의 발현을 억제하기 위한 방법 및 조성물 |
| EP3737424A4 (fr) | 2018-01-10 | 2021-10-27 | Translate Bio MA, Inc. | Compositions et méthodes pour faciliter l'administration d'acides nucléiques synthétiques dans des cellules |
| BR112020014425A2 (pt) | 2018-01-15 | 2020-12-29 | Ionis Pharmaceuticals, Inc. | Moduladores de expressão de dnm2 |
| WO2019157531A1 (fr) | 2018-02-12 | 2019-08-15 | Ionis Pharmaceuticals, Inc. | Composés modifiés et leurs utilisations |
| US20210155926A1 (en) * | 2018-04-05 | 2021-05-27 | Silence Therapeutics Gmbh | siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS |
| KR20210018267A (ko) | 2018-05-07 | 2021-02-17 | 알닐람 파마슈티칼스 인코포레이티드 | 간외 전달 |
| PE20210393A1 (es) | 2018-05-09 | 2021-03-02 | Ionis Pharmaceuticals Inc | Compuestos y metodos para la reduccion de la expresion de fxi |
| US12496311B2 (en) | 2018-07-17 | 2025-12-16 | Aronora, Inc. | Methods for safely reducing thrombopoietin |
| EP3833397A4 (fr) | 2018-08-08 | 2023-06-14 | Arcturus Therapeutics, Inc. | Compositions et agents contre la stéatohépatite non alcoolique |
| TWI869213B (zh) | 2018-09-19 | 2025-01-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
| AU2019403447B2 (en) | 2018-12-21 | 2023-07-27 | Ionis Pharmaceuticals, Inc. | Modulators of HSD17B13 expression |
| CA3126947A1 (fr) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Oligonucleotides d'edition d'arn et leurs utilisations |
| WO2020154343A1 (fr) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Oligonucléotides d'édition d'arn et leurs utilisations |
| CA3127243A1 (fr) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Oligonucleotides d'edition d'arn et leurs utilisations |
| WO2021074772A1 (fr) | 2019-10-14 | 2021-04-22 | Astrazeneca Ab | Modulateurs de l'expression de pnpla3 |
| CA3173034A1 (fr) | 2020-02-28 | 2021-09-02 | Ionis Pharmaceuticals, Inc. | Composes et procedes de modulation de smn2 |
| KR20220160647A (ko) | 2020-03-27 | 2022-12-06 | 제너레이션 바이오 컴퍼니 | 신규한 지질 및 이의 나노입자 조성물 |
| JP2023522961A (ja) | 2020-04-21 | 2023-06-01 | フラッグシップ パイオニアリング， インコーポレイテッド | 二機能性分子およびその使用方法 |
| CN111748005B (zh) * | 2020-06-24 | 2022-02-11 | 河北大学 | GalNAc修饰的美兰衍生物、制备方法与应用及肝靶向荧光探针、HClO检测方法 |
| CN116546989A (zh) * | 2020-10-09 | 2023-08-04 | 阿达尔克斯制药有限公司 | N-乙酰半乳糖胺（GalNAc）来源的化合物和寡核苷酸 |
| LT4136092T (lt) | 2020-11-18 | 2024-09-25 | Ionis Pharmaceuticals, Inc. | Junginiai ir būdai, skirti angiotenzinogeno raiškos moduliavimui |
| KR20230135585A (ko) | 2020-12-23 | 2023-09-25 | 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 | 변형된 trem의 조성물 및 이의 용도 |
| EP4355727A1 (fr) | 2021-06-14 | 2024-04-24 | Generation Bio Co. | Lipides cationiques et compositions de ceux-ci |
| JP2024536147A (ja) | 2021-10-01 | 2024-10-04 | エーダーエックス ファーマシューティカルズ， インコーポレイテッド | プレカリクレインを調節する組成物及びその使用方法 |
| JP7776924B2 (ja) * | 2022-03-08 | 2025-11-27 | 大原薬品工業株式会社 | 変異ＦＵＳの発現を選択的に抑制する修飾ｓｉＲＮＡ |
| WO2023250112A1 (fr) | 2022-06-22 | 2023-12-28 | Flagship Pioneering Innovations Vi, Llc | Compositions de trem modifiées et leurs utilisations |
| WO2024040222A1 (fr) | 2022-08-19 | 2024-02-22 | Generation Bio Co. | Adn à extrémités fermées clivable (adnce) et ses procédés d'utilisation |
| WO2024166805A1 (fr) * | 2023-02-06 | 2024-08-15 | 日油株式会社 | Chlorhydrate d'amine ayant un groupe maléimide et son procédé de production |
| WO2024220930A2 (fr) | 2023-04-20 | 2024-10-24 | Adarx Pharmaceuticals, Inc. | Compositions de modulation de mapt et leurs procédés d'utilisation |
| TW202502385A (zh) | 2023-05-12 | 2025-01-16 | 美商雅迪克斯製藥公司 | Ｎｍｄａ配位體結合之化合物及其用途 |
| AU2024279767A1 (en) | 2023-05-26 | 2025-12-04 | Adarx Pharmaceuticals, Inc. | Sod1-modulating compositions and methods of use thereof |
| TW202506137A (zh) | 2023-06-20 | 2025-02-16 | 美商雅迪克斯製藥公司 | Ｌｒｒｋ２調節組合物及其使用方法 |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3222294A1 (fr) | 2003-04-30 | 2017-09-27 | Sirna Therapeutics, Inc. | Conjugués et compositions pour délivrer des composés au niveau cellulaire |
| JP2007535317A (ja) * | 2004-04-15 | 2007-12-06 | グライコフィ， インコーポレイテッド | 下等真核生物におけるガラクトシル化された糖タンパク質の産生 |
| CA2650668A1 (fr) | 2006-04-28 | 2007-11-08 | Centre National De La Recherche Scientifique | Procede de synthese de derives oligonucleotidiques |
| US8252755B2 (en) | 2006-09-22 | 2012-08-28 | Dharmacon, Inc. | Duplex oligonucleotide complexes and methods for gene silencing by RNA interference |
| CA2930393C (fr) * | 2007-12-04 | 2022-11-29 | Alnylam Pharmaceuticals, Inc. | Conjugues glucidiques utilises en tant qu'agents d'administration pour des oligonucleotides |
| EP3604533A1 (fr) | 2008-04-11 | 2020-02-05 | Arbutus Biopharma Corporation | Délivrance spécifique à un site d'acides nucléiques en combinant des ligands de ciblage avec des composants endosomolytiques |
| AU2009298802A1 (en) * | 2008-09-23 | 2010-04-08 | Alnylam Pharmaceuticals, Inc. | Chemical modifications of monomers and oligonucleotides with cycloaddition |
| US8247827B2 (en) | 2008-09-30 | 2012-08-21 | Bridgelux, Inc. | LED phosphor deposition |
| SG183374A1 (en) * | 2010-02-24 | 2012-09-27 | Arrowhead Res Corp | Compositions for targeted delivery of sirna |
| WO2011126974A1 (fr) * | 2010-04-09 | 2011-10-13 | Merck Sharp & Dohme Corp. | Entités chimiques isolées inédites et procédés d'administration d'oligonucléotides |
| AR090905A1 (es) * | 2012-05-02 | 2014-12-17 | Merck Sharp & Dohme | Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica |
-
2013
- 2013-04-30 AR ARP130101481A patent/AR090906A1/es unknown
- 2013-04-30 TW TW102115516A patent/TWI595885B/zh active
- 2013-04-30 TW TW106117248A patent/TW201808342A/zh unknown
- 2013-05-01 WO PCT/US2013/039025 patent/WO2013166121A1/fr not_active Ceased
- 2013-05-01 CA CA2872100A patent/CA2872100A1/fr not_active Abandoned
- 2013-05-01 HK HK15108687.4A patent/HK1208227A1/xx unknown
- 2013-05-01 AU AU2013256400A patent/AU2013256400B2/en active Active
- 2013-05-01 US US14/398,317 patent/US9540639B2/en active Active
- 2013-05-01 JP JP2015510418A patent/JP6239592B2/ja active Active
- 2013-05-01 EP EP13784779.4A patent/EP2844663B1/fr active Active
-
2016
- 2016-11-29 US US15/363,490 patent/US10214742B2/en active Active
-
2017
- 2017-11-01 JP JP2017211436A patent/JP2018048186A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2015520742A (ja) | 2015-07-23 |
| EP2844663A1 (fr) | 2015-03-11 |
| TW201350131A (zh) | 2013-12-16 |
| EP2844663A4 (fr) | 2015-09-30 |
| CA2872100A1 (fr) | 2013-11-07 |
| TWI595885B (zh) | 2017-08-21 |
| JP2018048186A (ja) | 2018-03-29 |
| AU2013256400B2 (en) | 2017-09-21 |
| US9540639B2 (en) | 2017-01-10 |
| US10214742B2 (en) | 2019-02-26 |
| US20170233731A1 (en) | 2017-08-17 |
| AR090906A1 (es) | 2014-12-17 |
| JP6239592B2 (ja) | 2017-11-29 |
| TW201808342A (zh) | 2018-03-16 |
| AU2013256400A1 (en) | 2014-11-06 |
| WO2013166121A1 (fr) | 2013-11-07 |
| EP2844663B1 (fr) | 2020-03-25 |
| US20150203843A1 (en) | 2015-07-23 |
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