WO2012092373A3 - In vivo polynucleotide delivery conjugates having enzyme sensitive linkages - Google Patents

In vivo polynucleotide delivery conjugates having enzyme sensitive linkages Download PDF

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Publication number
WO2012092373A3
WO2012092373A3 PCT/US2011/067588 US2011067588W WO2012092373A3 WO 2012092373 A3 WO2012092373 A3 WO 2012092373A3 US 2011067588 W US2011067588 W US 2011067588W WO 2012092373 A3 WO2012092373 A3 WO 2012092373A3
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WO
WIPO (PCT)
Prior art keywords
polynucleotide delivery
rnai
vivo
delivery conjugates
enzyme sensitive
Prior art date
Application number
PCT/US2011/067588
Other languages
French (fr)
Other versions
WO2012092373A2 (en
Inventor
David B. Rozema
David L. Lewis
Darren H. Wakefield
Eric A. Kitas
Philipp Hadwiger
Jon A. Wolff
Ingo Roehl
Peter Mohr
Torsten Hoffmann
Kerstin Jahn-Hofmann
Hans Martin Mueller
Guenther Ott
Andrei V. Blokhin
Jeffrey C. Carlson
Jonathan D. Benson
Original Assignee
Arrowhead Research Corporation
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
Priority to KR1020137017195A priority Critical patent/KR20130136494A/en
Priority to JP2013547648A priority patent/JP5941926B2/en
Priority to SG2013029830A priority patent/SG189942A1/en
Priority to EP11853858.6A priority patent/EP2658579A4/en
Priority to MX2013007316A priority patent/MX341118B/en
Priority to CN201180061179.6A priority patent/CN103491982B/en
Priority to RU2013117286A priority patent/RU2619453C2/en
Application filed by Arrowhead Research Corporation filed Critical Arrowhead Research Corporation
Priority to NZ611656A priority patent/NZ611656A/en
Priority to AU2011352204A priority patent/AU2011352204B2/en
Priority to CA2816041A priority patent/CA2816041C/en
Priority to MX2016006733A priority patent/MX347298B/en
Priority to BR112013014115A priority patent/BR112013014115A2/en
Publication of WO2012092373A2 publication Critical patent/WO2012092373A2/en
Priority to ZA2013/02896A priority patent/ZA201302896B/en
Publication of WO2012092373A3 publication Critical patent/WO2012092373A3/en

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00—Carboxylic acid amides
    • C07C233/01—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C233/02—Carboxylic 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
    • C07C233/04—Carboxylic 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
    • C07C233/05—Carboxylic 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
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/58—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 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
    • 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
    • A61K47/56—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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—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 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
    • A61K47/60—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 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C69/00—Esters of carboxylic acids; Esters of carbonic or haloformic acids
    • C07C69/96—Esters of carbonic or haloformic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/06—Dipeptides
    • C07K5/06104—Dipeptides with the first amino acid being acidic
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/06—Dipeptides
    • C07K5/06104—Dipeptides with the first amino acid being acidic
    • C07K5/06113—Asp- or Asn-amino acid
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08G—MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING UNSATURATED CARBON-TO-CARBON BONDS
    • C08G63/00—Macromolecular compounds obtained by reactions forming a carboxylic ester link in the main chain of the macromolecule
    • C08G63/91—Polymers modified by chemical after-treatment
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/06—Dipeptides
    • C07K5/06008—Dipeptides with the first amino acid being neutral
    • C07K5/06017—Dipeptides with the first amino acid being neutral and aliphatic
    • C07K5/06034—Dipeptides with the first amino acid being neutral and aliphatic the side chain containing 2 to 4 carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K5/00—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof
    • C07K5/04—Peptides containing up to four amino acids in a fully defined sequence; Derivatives thereof containing only normal peptide links
    • C07K5/06—Dipeptides
    • C07K5/06008—Dipeptides with the first amino acid being neutral
    • C07K5/06078—Dipeptides with the first amino acid being neutral and aromatic or cycloaliphatic
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12N2800/00—Nucleic acids vectors
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/10—Vectors comprising a non-peptidic targeting moiety

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • 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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)

Abstract

The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
PCT/US2011/067588 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages WO2012092373A2 (en)

Priority Applications (13)

Application Number Priority Date Filing Date Title
BR112013014115A BR112013014115A2 (en) 2010-12-29 2011-12-28 polynucleotide delivery conjugates in vivo showing sensitive enzyme linkages
RU2013117286A RU2619453C2 (en) 2010-12-29 2011-12-28 Conjugates for delivery of polynucleotides in vivo, containing bonds sensitive to enzymatic degradation
SG2013029830A SG189942A1 (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
EP11853858.6A EP2658579A4 (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
NZ611656A NZ611656A (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
CN201180061179.6A CN103491982B (en) 2010-12-29 2011-12-28 The polynucleotides delivering in vivo conjugate connected with enzyme sensitiveness
JP2013547648A JP5941926B2 (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme-sensitive linkages
KR1020137017195A KR20130136494A (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
MX2013007316A MX341118B (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages.
AU2011352204A AU2011352204B2 (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
CA2816041A CA2816041C (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
MX2016006733A MX347298B (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages.
ZA2013/02896A ZA201302896B (en) 2010-12-29 2013-04-22 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201061427936P 2010-12-29 2010-12-29
US61/427,936 2010-12-29
US13/336,028 2011-12-23
US13/336,028 US8426554B2 (en) 2010-12-29 2011-12-23 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages

Publications (2)

Publication Number Publication Date
WO2012092373A2 WO2012092373A2 (en) 2012-07-05
WO2012092373A3 true WO2012092373A3 (en) 2013-10-24

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PCT/US2011/067588 WO2012092373A2 (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages

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EP (1) EP2658579A4 (en)
JP (1) JP5941926B2 (en)
KR (1) KR20130136494A (en)
CN (1) CN103491982B (en)
AU (1) AU2011352204B2 (en)
BR (1) BR112013014115A2 (en)
CA (1) CA2816041C (en)
CL (2) CL2013001876A1 (en)
MX (2) MX341118B (en)
NZ (1) NZ611656A (en)
PE (1) PE20140198A1 (en)
RU (1) RU2619453C2 (en)
SG (1) SG189942A1 (en)
WO (1) WO2012092373A2 (en)
ZA (1) ZA201302896B (en)

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JOP20210043A1 (en) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc Compositions and Methods for Inhibiting Gene Expression of LPA
MA45328A (en) * 2016-04-01 2019-02-06 Avidity Biosciences Llc NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
US20200277647A1 (en) 2017-05-24 2020-09-03 Ramot At Tel-Aviv University Ltd. Chemiluminescent probes for imaging/detection of proteases
MX2020005860A (en) 2017-12-06 2020-09-09 Avidity Biosciences Inc Compositions and methods of treating muscle atrophy and myotonic dystrophy.
AU2019416109A1 (en) * 2018-12-28 2021-07-22 Sirnaomics, Inc. Targeted delivery of therapeutic molecules
JP2023537798A (en) 2020-03-19 2023-09-06 アビディティー バイオサイエンシーズ，インク． Compositions and methods for treating facioscapulohumeral muscular dystrophy
CA3198230A1 (en) * 2021-01-28 2022-08-04 Jian Chen Conjugate and use thereof
JPWO2022270607A1 (en) * 2021-06-24 2022-12-29
KR20230016999A (en) * 2021-07-27 2023-02-03 안동대학교 산학협력단 Pharmaceutical composition comprising the extract from vespa velutina nigrithorax as an effective component for prevention or treatment of thrombosis and health functional food comprising the same
CN113621003B (en) * 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) Method for preparing organic molecule covalent modification functionalized nucleic acid material and application thereof
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EP2658579A4 (en) 2015-07-22
MX2013007316A (en) 2013-07-29
NZ611656A (en) 2014-10-31
JP2014505685A (en) 2014-03-06
RU2013117286A (en) 2015-03-10
ZA201302896B (en) 2014-07-25
CN103491982A (en) 2014-01-01
AU2011352204A1 (en) 2013-05-02
RU2619453C2 (en) 2017-05-16
AU2011352204B2 (en) 2015-05-21
CN103491982B (en) 2017-09-12
KR20130136494A (en) 2013-12-12
BR112013014115A2 (en) 2019-09-24
WO2012092373A2 (en) 2012-07-05
MX347298B (en) 2017-04-21
CL2015003580A1 (en) 2016-07-15
CA2816041C (en) 2019-01-08
PE20140198A1 (en) 2014-02-21
CL2013001876A1 (en) 2014-04-11
JP5941926B2 (en) 2016-06-29
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