AU2019220739B2 - Trialkyne linking agents and methods of use - Google Patents

Trialkyne linking agents and methods of use

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Publication number
AU2019220739B2
AU2019220739B2 AU2019220739A AU2019220739A AU2019220739B2 AU 2019220739 B2 AU2019220739 B2 AU 2019220739B2 AU 2019220739 A AU2019220739 A AU 2019220739A AU 2019220739 A AU2019220739 A AU 2019220739A AU 2019220739 B2 AU2019220739 B2 AU 2019220739B2
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compound
formula
mmol
rna
iii
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AU2019220739A
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AU2019220739A1 (en
Inventor
Erich ALTENHOFER
Jeffrey Carlson
Bo Chen
Matthew FOWLER-WATTERS
Zhen Li
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Arrowhead Pharmaceuticals Inc
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Arrowhead Pharmaceuticals Inc
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    • C07F9/00Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02Phosphorus compounds
    • C07F9/06Phosphorus compounds without P—C bonds
    • C07F9/22Amides of acids of phosphorus
    • C07F9/24Esteramides
    • C07F9/2404Esteramides the ester moiety containing a substituent or a structure which is considered as characteristic
    • C07F9/2416Esteramides the ester moiety containing a substituent or a structure which is considered as characteristic of cycloaliphatic alcohols
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
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    • A61K47/545Heterocyclic compounds
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    • A61K47/51Medicinal 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
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    • A61K47/548Phosphates or phosphonates, e.g. bone-seeking
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    • A61K47/65Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
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    • C07C205/42Compounds containing nitro groups bound to a carbon skeleton the carbon skeleton being further substituted by esterified hydroxy groups having nitro groups or esterified hydroxy groups bound to carbon atoms of six-membered aromatic rings of the carbon skeleton
    • C07C205/43Compounds containing nitro groups bound to a carbon skeleton the carbon skeleton being further substituted by esterified hydroxy groups having nitro groups or esterified hydroxy groups bound to carbon atoms of six-membered aromatic rings of the carbon skeleton to carbon atoms of the same non-condensed six-membered aromatic ring or to carbon atoms of six-membered aromatic rings being part of the same condensed ring system
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    • C07C233/18Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
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    • C07C233/16Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
    • C07C233/17Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/19Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to an acyclic carbon atom of a saturated carbon skeleton containing rings
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    • C07C233/63Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of rings other than six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups
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    • C07C233/82Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/83Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom of an acyclic saturated carbon skeleton
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    • C07C235/32Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton containing six-membered aromatic rings
    • C07C235/34Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton containing six-membered aromatic rings having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
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    • C07C237/22Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton having nitrogen atoms of amino groups bound to the carbon skeleton of the acid part, further acylated
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AU2019220739A 2018-02-17 2019-02-15 Trialkyne linking agents and methods of use Active AU2019220739B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025267360A AU2025267360A1 (en) 2018-02-17 2025-11-12 Trialkyne linking agents and methods of use

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862631683P 2018-02-17 2018-02-17
US62/631,683 2018-02-17
US201862646739P 2018-03-22 2018-03-22
US62/646,739 2018-03-22
US201862663763P 2018-04-27 2018-04-27
US62/663,763 2018-04-27
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Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020526192A (ja) 2017-07-06 2020-08-31 アローヘッド ファーマシューティカルズ インコーポレイテッド アルファ−ENaCの発現を阻害するためのRNAi剤、および使用方法
MX2021008261A (es) 2019-01-09 2021-08-16 Arrowhead Pharmaceuticals Inc Agentes de rnai para inhibir la expresion de hif-2 a (epas1), composiciones de estos y metodos de uso.
KR20220024153A (ko) 2019-05-24 2022-03-03 엠피리코 인크. 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료
KR20220058578A (ko) * 2019-09-03 2022-05-09 아크투루스 쎄라퓨틱스, 인크. 치료 활성 접합체의 아시알로당단백질 수용체 매개 전달
WO2022056266A2 (en) * 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
MX2023002854A (es) * 2020-09-11 2023-04-03 Arrowhead Pharmaceuticals Inc Plataformas de suministro al musculo esqueletico y metodos de uso.
KR20230167082A (ko) 2021-04-08 2023-12-07 애로우헤드 파마슈티컬스 인코포레이티드 진행성 당화 최종 산물에 대한 수용체의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
AU2022280863A1 (en) 2021-05-28 2023-11-30 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of mucin 5ac (muc5ac), compositions thereof, and methods of use
CN113956206B (zh) * 2021-10-25 2023-04-28 深圳湾实验室坪山生物医药研发转化中心 一种用于修饰蛋白质赖氨酸残基的探针及其制备方法
WO2023192828A2 (en) * 2022-03-28 2023-10-05 Empirico Inc. Compositions and methods for the treatment of angiopoietin like 7 (angptl7) related diseases
AU2023245603A1 (en) 2022-03-28 2024-11-07 Empirico Inc. Modified oligonucleotides
JP2025522433A (ja) 2022-06-15 2025-07-15 アローヘッド ファーマシューティカルズ インコーポレイテッド スーパーオキシドジスムターゼ1(SOD1)の発現を阻害するためのRNAi剤、その組成物、及び使用の方法
AU2023399039A1 (en) * 2022-12-14 2025-05-22 Alnylam Pharmaceuticals, Inc. ALPHA-V BETA-6(ανβ6) INTEGRIN LIGANDS FOR EXTRAHEPATIC DELIVERY
WO2025137384A1 (en) 2023-12-20 2025-06-26 Arrowhead Pharmaceuticals, Inc. Methods and reagents for improved oligonucleotide synthesis
WO2025152969A1 (zh) * 2024-01-15 2025-07-24 武汉人福创新药物研发中心有限公司 靶向化合物及其用途

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170202970A1 (en) * 2015-05-12 2017-07-20 Blinkbio, Inc. Silicon based drug conjugates and methods of using same
WO2019010274A1 (en) * 2017-07-06 2019-01-10 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS OF INHIBITION OF ALPHA-ENAC GENE EXPRESSION AND METHODS OF USE

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2650668A1 (en) * 2006-04-28 2007-11-08 Centre National De La Recherche Scientifique Method for the synthesis of triazole-containing oligonucleotide deratives
GB201313201D0 (en) * 2013-07-24 2013-09-04 Univ East Anglia Virus Detection
BR112016014162A2 (pt) * 2014-01-15 2018-01-09 Basf Se uso de um conjugado, conjugado e métodos para a fabricação de um conjugado e para transfectar uma célula
AU2017296195A1 (en) 2016-07-11 2019-01-24 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170202970A1 (en) * 2015-05-12 2017-07-20 Blinkbio, Inc. Silicon based drug conjugates and methods of using same
WO2019010274A1 (en) * 2017-07-06 2019-01-10 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS OF INHIBITION OF ALPHA-ENAC GENE EXPRESSION AND METHODS OF USE

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