CL2013001876A1 - Compuesto para la modificación reversible de una poliamina activa en membrana anfifática; polímero de suministro para suministrar un polinecleotido de interferencia de arn (iarn) a una célula in vivo; uso de un iarn con un polimero de suministro. - Google Patents

Compuesto para la modificación reversible de una poliamina activa en membrana anfifática; polímero de suministro para suministrar un polinecleotido de interferencia de arn (iarn) a una célula in vivo; uso de un iarn con un polimero de suministro.

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Publication number
CL2013001876A1
CL2013001876A1 CL2013001876A CL2013001876A CL2013001876A1 CL 2013001876 A1 CL2013001876 A1 CL 2013001876A1 CL 2013001876 A CL2013001876 A CL 2013001876A CL 2013001876 A CL2013001876 A CL 2013001876A CL 2013001876 A1 CL2013001876 A1 CL 2013001876A1
Authority
CL
Chile
Prior art keywords
iarn
supply polymer
arn
vivo
delivering
Prior art date
Application number
CL2013001876A
Other languages
English (en)
Spanish (es)
Inventor
David L Lewis
Philipp Hadwiger
Ingo Roehl
Peter Mohr
Torsten Hoffmann
Kerstin Jahn-Hofmann
Andrei V Blokhin
Jonathan D Benson
David B Rozema
Darren H Wakefield
Jon A Wolff
Hans Martin Mueller
Guenther Ott
Jeffrey C Carlson
Eric A Kitas
Original Assignee
Arrowhead Res Corp
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Priority claimed from US13/336,028 external-priority patent/US8426554B2/en
Application filed by Arrowhead Res Corp filed Critical Arrowhead Res Corp
Publication of CL2013001876A1 publication Critical patent/CL2013001876A1/es
Priority to CL2015003580A priority Critical patent/CL2015003580A1/es

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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K5/00Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/06Dipeptides
    • C07K5/06008Dipeptides with the first amino acid being neutral
    • C07K5/06017Dipeptides with the first amino acid being neutral and aliphatic
    • C07K5/06034Dipeptides with the first amino acid being neutral and aliphatic the side chain containing 2 to 4 carbon atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00Carboxylic acid amides
    • C07C233/01Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C233/02Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having nitrogen atoms of carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals
    • C07C233/04Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having nitrogen atoms of carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals with carbon atoms of carboxamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • C07C233/05Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having nitrogen atoms of carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals with carbon atoms of carboxamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • 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
    • 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
    • A61K47/56Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/58Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. poly[meth]acrylate, polyacrylamide, polystyrene, polyvinylpyrrolidone, polyvinylalcohol or polystyrene sulfonic acid resin
    • 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
    • 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
    • A61K47/56Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C69/00Esters of carboxylic acids; Esters of carbonic or haloformic acids
    • C07C69/96Esters of carbonic or haloformic acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K5/00Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/06Dipeptides
    • C07K5/06104Dipeptides with the first amino acid being acidic
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K5/00Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/06Dipeptides
    • C07K5/06104Dipeptides with the first amino acid being acidic
    • C07K5/06113Asp- or Asn-amino acid
    • CCHEMISTRY; METALLURGY
    • C08ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08GMACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G63/00Macromolecular compounds obtained by reactions forming a carboxylic ester link in the main chain of the macromolecule
    • C08G63/91Polymers modified by chemical after-treatment
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K5/00Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/06Dipeptides
    • C07K5/06008Dipeptides with the first amino acid being neutral
    • C07K5/06078Dipeptides with the first amino acid being neutral and aromatic or cycloaliphatic
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/95Protection of vectors from inactivation by agents such as antibodies or enzymes, e.g. using polymers
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    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/10Vectors comprising a non-peptidic targeting moiety

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  • Chemical & Material Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Polymers & Plastics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
CL2013001876A 2010-12-29 2013-06-25 Compuesto para la modificación reversible de una poliamina activa en membrana anfifática; polímero de suministro para suministrar un polinecleotido de interferencia de arn (iarn) a una célula in vivo; uso de un iarn con un polimero de suministro. CL2013001876A1 (es)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CL2015003580A CL2015003580A1 (es) 2010-12-29 2015-12-10 Conjugados de suministro de polinucleotidos in vivo que tienen acoplamientos sensibles a enzima.

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201061427936P 2010-12-29 2010-12-29
US13/336,028 US8426554B2 (en) 2010-12-29 2011-12-23 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages

Publications (1)

Publication Number Publication Date
CL2013001876A1 true CL2013001876A1 (es) 2014-04-11

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
CL2013001876A CL2013001876A1 (es) 2010-12-29 2013-06-25 Compuesto para la modificación reversible de una poliamina activa en membrana anfifática; polímero de suministro para suministrar un polinecleotido de interferencia de arn (iarn) a una célula in vivo; uso de un iarn con un polimero de suministro.
CL2015003580A CL2015003580A1 (es) 2010-12-29 2015-12-10 Conjugados de suministro de polinucleotidos in vivo que tienen acoplamientos sensibles a enzima.

Family Applications After (1)

Application Number Title Priority Date Filing Date
CL2015003580A CL2015003580A1 (es) 2010-12-29 2015-12-10 Conjugados de suministro de polinucleotidos in vivo que tienen acoplamientos sensibles a enzima.

Country Status (15)

Country Link
EP (1) EP2658579A4 (fr)
JP (1) JP5941926B2 (fr)
KR (1) KR20130136494A (fr)
CN (1) CN103491982B (fr)
AU (1) AU2011352204B2 (fr)
BR (1) BR112013014115A2 (fr)
CA (1) CA2816041C (fr)
CL (2) CL2013001876A1 (fr)
MX (2) MX347298B (fr)
NZ (1) NZ611656A (fr)
PE (1) PE20140198A1 (fr)
RU (1) RU2619453C2 (fr)
SG (1) SG189942A1 (fr)
WO (1) WO2012092373A2 (fr)
ZA (1) ZA201302896B (fr)

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US20150291958A1 (en) 2012-11-15 2015-10-15 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
CN105451743B (zh) * 2013-08-07 2019-11-19 箭头研究公司 用于在活体中递送RNAi触发子至肿瘤细胞的聚偶联物
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
JP2020520981A (ja) 2017-05-24 2020-07-16 ラモット・アット・テル・アビブ・ユニバーシテイ・リミテッドRamot At Tel Aviv University Ltd. プロテアーゼのイメージング/検出のための化学発光プローブ
KR102623786B1 (ko) 2017-12-06 2024-01-11 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
WO2020139788A2 (fr) * 2018-12-28 2020-07-02 Sirnaomics, Inc. Administration ciblée de molécules thérapeutiques
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
EP4285937A1 (fr) * 2021-01-28 2023-12-06 Nanjing Chempion Biotechnology Co., Ltd. Conjugué et son utilisation
CN117480175A (zh) * 2021-06-24 2024-01-30 东北泰克诺亚奇股份有限公司 荧光探针
KR102712976B1 (ko) * 2021-07-27 2024-10-07 국립안동대학교 산학협력단 등검은말벌 추출물을 유효성분으로 함유하는 혈전증의 예방 또는 치료용 약학적 조성물 및 건강 기능 식품
CN113621003B (zh) * 2021-09-10 2023-02-03 国科温州研究院(温州生物材料与工程研究所) 一种制备有机分子共价修饰的功能化核酸材料的方法及其应用
WO2023043953A1 (fr) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions et méthodes de traitement d'une dystrophie musculaire facio-scapulo-humérale

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US6214345B1 (en) * 1993-05-14 2001-04-10 Bristol-Myers Squibb Co. Lysosomal enzyme-cleavable antitumor drug conjugates
US6180095B1 (en) * 1997-12-17 2001-01-30 Enzon, Inc. Polymeric prodrugs of amino- and hydroxyl-containing bioactive agents
CN1433478A (zh) * 1999-12-30 2003-07-30 诺瓦提斯公司 用于基因治疗的新的胶体合成载体
NZ520458A (en) * 2000-02-24 2005-02-25 Genentech Inc Caspase activated prodrugs therapy
EP1243276A1 (fr) * 2001-03-23 2002-09-25 Franciscus Marinus Hendrikus De Groot Prodrogues activables à séparateurs allongés et multiples
US7091186B2 (en) * 2001-09-24 2006-08-15 Seattle Genetics, Inc. p-Amidobenzylethers in drug delivery agents
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WO2007059404A2 (fr) * 2005-11-10 2007-05-24 Medarex, Inc. Composes et conjugues cytotoxiques
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JP5274461B2 (ja) * 2006-08-18 2013-08-28 アローヘッド リサーチ コーポレイション ポリヌクレオチドのインビボ送達のためのポリ結合体
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Also Published As

Publication number Publication date
EP2658579A2 (fr) 2013-11-06
CN103491982B (zh) 2017-09-12
CA2816041C (fr) 2019-01-08
KR20130136494A (ko) 2013-12-12
MX341118B (es) 2016-08-09
CL2015003580A1 (es) 2016-07-15
EP2658579A4 (fr) 2015-07-22
ZA201302896B (en) 2014-07-25
SG189942A1 (en) 2013-06-28
AU2011352204A1 (en) 2013-05-02
JP2014505685A (ja) 2014-03-06
CN103491982A (zh) 2014-01-01
CA2816041A1 (fr) 2012-07-05
MX2013007316A (es) 2013-07-29
PE20140198A1 (es) 2014-02-21
AU2011352204B2 (en) 2015-05-21
WO2012092373A3 (fr) 2013-10-24
WO2012092373A2 (fr) 2012-07-05
MX347298B (es) 2017-04-21
NZ611656A (en) 2014-10-31
RU2619453C2 (ru) 2017-05-16
BR112013014115A2 (pt) 2019-09-24
JP5941926B2 (ja) 2016-06-29
RU2013117286A (ru) 2015-03-10

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