JP2011505846A - ポリペプチド−核酸コンジュゲートおよびその使用 - Google Patents

ポリペプチド−核酸コンジュゲートおよびその使用 Download PDF

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JP2011505846A
JP2011505846A JP2010538304A JP2010538304A JP2011505846A JP 2011505846 A JP2011505846 A JP 2011505846A JP 2010538304 A JP2010538304 A JP 2010538304A JP 2010538304 A JP2010538304 A JP 2010538304A JP 2011505846 A JP2011505846 A JP 2011505846A
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compound
polypeptide
disease
sirna
seq
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JP2011505846A5 (enExample
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ベリヴォ，リシャール
ドゥムール，ミシェル
シェ，クリスチャン
レジーナ，アンソニー
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アンジオケム，インコーポレーテッド
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    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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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 nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/40—Vectors comprising a peptide as targeting moiety, e.g. a synthetic peptide, from undefined source
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein

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  • Chemical Kinetics & Catalysis (AREA)
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  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
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JP2010538304A 2007-12-20 2008-12-19 ポリペプチド−核酸コンジュゲートおよびその使用 Pending JP2011505846A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US882507P 2007-12-20 2007-12-20
PCT/CA2008/002269 WO2009079790A1 (en) 2007-12-20 2008-12-19 Polypeptide-nucleic acid conjugates and uses thereof

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JP2011505846A true JP2011505846A (ja) 2011-03-03
JP2011505846A5 JP2011505846A5 (enExample) 2012-02-09

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US (1) US20110039785A1 (enExample)
EP (1) EP2235175A4 (enExample)
JP (1) JP2011505846A (enExample)
CN (1) CN101946001A (enExample)
AU (1) AU2008340943A1 (enExample)
BR (1) BRPI0821310A2 (enExample)
CA (1) CA2709635A1 (enExample)
MX (1) MX2010006925A (enExample)
RU (1) RU2010129761A (enExample)
WO (1) WO2009079790A1 (enExample)
ZA (1) ZA201004609B (enExample)

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JP2013530993A (ja) * 2010-07-02 2013-08-01 アンジオケム インコーポレーテッド 治療用コンジュゲートのための短く且つｄ−アミノ酸を含有するポリペプチドおよびその使用
JP2014515762A (ja) * 2011-05-05 2014-07-03 サレプタ セラピューティクス， インコーポレイテッド ペプチドオリゴヌクレオチドコンジュゲート
JP2015504968A (ja) * 2012-01-27 2015-02-16 ザ、リージェンツ、オブ、ザ、ユニバーシティ、オブ、カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ 糖ポリマーを用いた生体分子の安定化
US9499583B2 (en) 2005-07-13 2016-11-22 Sarepta Therapeutics, Inc. Antibacterial antisense oligonucleotide and method
US9572899B2 (en) 2003-04-29 2017-02-21 Avi Biopharma, Inc. Compositions for enhancing transport of molecules into cells
US9862946B2 (en) 2011-05-05 2018-01-09 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
US11236329B2 (en) 2007-06-29 2022-02-01 Sarepta Therapeutics, Inc. Compound and method for treating myotonic dystrophy
JP2023071635A (ja) * 2021-11-11 2023-05-23 杭州痴創生物科技有限公司 分子標的薬ｎＣｏＶｓｈＲＮＡ・２ＡＣＥ２の合成方法

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ATE512674T1 (de) 2003-01-06 2011-07-15 Angiochem Inc Angiopep-1, verwandte verbindungen, und deren verwnedungen
MX2007010113A (es) 2005-02-18 2007-12-07 Angiochem Inc Polipeptidos de aprotinina para transportar un compuesto a traves de la barrera hematoencefalica.
PL2233156T3 (pl) 2005-07-15 2013-09-30 Angiochem Inc Zastosowanie polipeptydów aprotyninowych jako nośników w koniugatach farmaceutycznych
JP2010506860A (ja) 2006-10-19 2010-03-04 アンジオケム，インコーポレーテッド Ｐ−糖タンパク質機能を刺激するための化合物およびその使用
US9365634B2 (en) 2007-05-29 2016-06-14 Angiochem Inc. Aprotinin-like polypeptides for delivering agents conjugated thereto to tissues
WO2010043047A1 (en) 2008-10-15 2010-04-22 Angiochem Inc. Conjugates of glp-1 agonists and uses thereof
WO2010063122A1 (en) 2008-12-05 2010-06-10 Angiochem Inc. Conjugates of neurotensin or neurotensin analogs and uses thereof
EP2373789A4 (en) 2008-12-17 2012-08-29 Angiochem Inc MEMBRANTYP-1 MATRIX METALLOPROTEINASE INHIBITORS AND THEIR USE
RU2011146654A (ru) 2009-04-20 2013-05-27 Ангиокем Инк. Способы лечения рака яичников с применением конъюгированного средства
WO2011000095A1 (en) * 2009-07-02 2011-01-06 Angiochem Inc. Multimeric peptide conjugates and uses thereof
WO2011041897A1 (en) * 2009-10-06 2011-04-14 Angiochem Inc. Compositions and methods for the transport of therapeutic agents
RU2012147589A (ru) * 2010-04-09 2014-05-20 Мерк Шарп Энд Домэ Корп. Новые отдельные химические структурные единицы и способы доставки олигонуклеотидов
KR20130103662A (ko) * 2010-04-19 2013-09-24 엔라이프 떼라퓨틱스, 에스.엘. 올리고뉴클레오티드 분자들의 특정 뉴런 유형들로의 선택적인 전달을 위한 조성물들 및 방법
EP2380595A1 (en) 2010-04-19 2011-10-26 Nlife Therapeutics S.L. Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
US20140259192A1 (en) 2011-07-12 2014-09-11 Sanofi Transgenic animal comprising a deletion or functional deletion of the 3'utr of an endogenous gene
ES2632212T3 (es) 2011-08-04 2017-09-11 Yeda Research And Development Co. Ltd. miR-135 y composiciones que lo comprenden para el tratamiento de afecciones médicas asociadas con la serotonina
CN104145015A (zh) * 2011-12-01 2014-11-12 安吉奥开米公司 靶向的溶酶体酶化合物
US20140335163A1 (en) * 2011-12-01 2014-11-13 Angiochem Inc. Targeted iduronate-2-sulfatase compounds
WO2013185235A1 (en) * 2012-06-15 2013-12-19 Angiochem Inc. Targeted iduronidase compounds
CN104781276A (zh) 2012-08-14 2015-07-15 安吉奥开米公司 肽-树枝状聚合物结合物及其用途
KR101445265B1 (ko) * 2012-09-18 2014-09-30 포항공과대학교 산학협력단 히알루론산-핵산 접합체 및 이를 포함하는 핵산 전달용 조성물
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