MX2015005328A - Composiciones y metodos para la administracion selectiva de moleculas de oligonucleotidos a tipos de celulas. - Google Patents
Composiciones y metodos para la administracion selectiva de moleculas de oligonucleotidos a tipos de celulas.Info
- Publication number
- MX2015005328A MX2015005328A MX2015005328A MX2015005328A MX2015005328A MX 2015005328 A MX2015005328 A MX 2015005328A MX 2015005328 A MX2015005328 A MX 2015005328A MX 2015005328 A MX2015005328 A MX 2015005328A MX 2015005328 A MX2015005328 A MX 2015005328A
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- nucleic acid
- compositions
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- target nucleic
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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
- C12N15/113—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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/16—Amides, e.g. hydroxamic acids
- A61K31/165—Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- 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
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- A—HUMAN NECESSITIES
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- 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
- A61K47/55—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 the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
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- 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
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- A—HUMAN NECESSITIES
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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
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- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- 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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- 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
- C12N15/1137—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 enzymes
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering N.A.
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3513—Protein; Peptide
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Abstract
La invención proporciona un conjugado que comprende (i) un ácido nucleico que es complementario a la secuencia de un ácido nucleico diana y cuya expresión previene o reduce la expresión del ácido nucleico diana y (ii) un agente de selectividad que es capaz de unirse con alta afinidad a un receptor que puede ser internalizado por la célula tras la unión a dicho agente de selectividad. Los conjugados de la presente invención son útiles para la administración de ácidos nucleicos a una célula de interés y de esta manera, para el tratamiento de enfermedades que requieren el descenso de la proteína codificada por el ácido nucleico diana así como para la administración de agentes de contraste a las células para fines diagnósticos.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
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US201261719297P | 2012-10-26 | 2012-10-26 | |
EP12382413 | 2012-10-26 | ||
PCT/EP2013/072411 WO2014064258A1 (en) | 2012-10-26 | 2013-10-25 | Compositions and methods for selective delivery of oligonucleotide molecules to cell types |
Publications (1)
Publication Number | Publication Date |
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MX2015005328A true MX2015005328A (es) | 2015-09-25 |
Family
ID=47148689
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2015005328A MX2015005328A (es) | 2012-10-26 | 2013-10-25 | Composiciones y metodos para la administracion selectiva de moleculas de oligonucleotidos a tipos de celulas. |
Country Status (7)
Country | Link |
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US (3) | US9938525B2 (es) |
EP (1) | EP2911697A1 (es) |
JP (1) | JP2016503394A (es) |
AU (1) | AU2013336582A1 (es) |
CA (1) | CA2889608A1 (es) |
MX (1) | MX2015005328A (es) |
WO (1) | WO2014064258A1 (es) |
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CA2472937C (en) | 2002-01-11 | 2014-06-17 | Biomarin Pharmaceutical, Inc. | Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes |
CA2796722C (en) | 2010-04-19 | 2021-06-01 | Nlife Therapeutics, S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
WO2012019168A2 (en) | 2010-08-06 | 2012-02-09 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
US20120237975A1 (en) | 2010-10-01 | 2012-09-20 | Jason Schrum | Engineered nucleic acids and methods of use thereof |
CA2831613A1 (en) | 2011-03-31 | 2012-10-04 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
BR112014002219A2 (pt) | 2011-07-05 | 2018-08-07 | Bioasis Technologies Inc | conjugados anticorpo p97 e métodos de sua utilização |
HUE033746T2 (en) | 2011-08-04 | 2018-01-29 | Yeda Res & Dev | MIR-135 and compositions thereof for the treatment of therapeutic conditions associated with serotonin |
US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
EP4015005A1 (en) | 2011-10-03 | 2022-06-22 | ModernaTX, Inc. | Modified nucleosides, nucleotides, and nucleic acids, and uses thereof |
SG11201402666WA (en) | 2011-12-16 | 2014-10-30 | Moderna Therapeutics Inc | Modified nucleoside, nucleotide, and nucleic acid compositions |
AU2013243955B2 (en) | 2012-04-02 | 2018-02-22 | Modernatx, Inc. | Modified polynucleotides for the production of oncology-related proteins and peptides |
US9572897B2 (en) | 2012-04-02 | 2017-02-21 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
US9283287B2 (en) | 2012-04-02 | 2016-03-15 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of nuclear proteins |
US9303079B2 (en) | 2012-04-02 | 2016-04-05 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
JP6433424B2 (ja) | 2012-07-31 | 2018-12-05 | バイオアシス テクノロジーズ インコーポレイテッド | 脱リン酸化されたリソソーム蓄積症タンパク質およびその使用方法 |
EP2911695B1 (en) | 2012-10-26 | 2023-11-29 | Palomo Limited | Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
PT2922554T (pt) | 2012-11-26 | 2022-06-28 | Modernatx Inc | Arn modificado nas porções terminais |
BR112015022416A2 (pt) | 2013-03-13 | 2017-10-24 | Bioasis Technologies Inc | fragmentos de p97 e seus usos |
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WO2015048744A2 (en) | 2013-09-30 | 2015-04-02 | Moderna Therapeutics, Inc. | Polynucleotides encoding immune modulating polypeptides |
CN105980401A (zh) | 2013-10-03 | 2016-09-28 | 现代治疗公司 | 编码低密度脂蛋白受体的多核苷酸 |
ES2764973T3 (es) | 2014-02-03 | 2020-06-05 | Bioasis Technologies Inc | Proteínas de fusión de P97 |
CA2936158C (en) | 2014-02-05 | 2023-06-13 | Yeda Research And Development Co. Ltd. | Use of mir-135 or precursor thereof for the treatment and diagnosis of a bipolar disease |
US10392605B2 (en) | 2014-02-19 | 2019-08-27 | Bioasis Technologies Inc. | P97-IDS fusion proteins |
US10058619B2 (en) * | 2014-05-01 | 2018-08-28 | Bioasis Technologies, Inc. | P97-polynucleotide conjugates |
US20170151339A1 (en) * | 2014-06-30 | 2017-06-01 | Tarveda Therapeutics, Inc. | Targeted conjugates and particles and formulations thereof |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
US10733023B1 (en) * | 2015-08-06 | 2020-08-04 | D2Iq, Inc. | Oversubscription scheduling |
GB2545898B (en) | 2015-12-21 | 2019-10-09 | Sutura Therapeutics Ltd | Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides |
GB2574525B (en) * | 2015-12-21 | 2020-09-02 | Sutura Therapeutics Ltd | Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides |
KR20180112060A (ko) | 2016-02-23 | 2018-10-11 | 타베다 세라퓨틱스, 인코포레이티드 | Hsp90 표적화된 접합체 및 이의 입자 및 제형 |
MA49421A (fr) | 2017-06-15 | 2020-04-22 | Modernatx Inc | Formulations d'arn |
AU2018326799A1 (en) | 2017-08-31 | 2020-02-27 | Modernatx, Inc. | Methods of making lipid nanoparticles |
EP3852728B1 (en) | 2018-09-20 | 2024-09-18 | ModernaTX, Inc. | Preparation of lipid nanoparticles and methods of administration thereof |
CN109085709B (zh) * | 2018-09-29 | 2021-07-30 | 昆山龙腾光电股份有限公司 | 一种贴附组件及贴附系统 |
JP2022545555A (ja) * | 2019-08-28 | 2022-10-27 | ザ・リージエンツ・オブ・ザ・ユニバーシテイー・オブ・カリフオルニア | 概日リズムの調節因子およびそれらの使用 |
AU2022335128A1 (en) * | 2021-08-24 | 2024-03-07 | Jcr Pharmaceuticals Co., Ltd. | Human transferrin receptor-binding antibody-peptide conjugate |
WO2024069229A2 (en) | 2022-08-03 | 2024-04-04 | Sutura Therapeutics Ltd | Biologically active compounds |
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-
2013
- 2013-10-25 MX MX2015005328A patent/MX2015005328A/es unknown
- 2013-10-25 EP EP13783538.5A patent/EP2911697A1/en active Pending
- 2013-10-25 JP JP2015538468A patent/JP2016503394A/ja active Pending
- 2013-10-25 AU AU2013336582A patent/AU2013336582A1/en not_active Abandoned
- 2013-10-25 WO PCT/EP2013/072411 patent/WO2014064258A1/en active Application Filing
- 2013-10-25 US US14/438,869 patent/US9938525B2/en active Active - Reinstated
- 2013-10-25 CA CA2889608A patent/CA2889608A1/en not_active Abandoned
- 2013-10-25 US US14/064,047 patent/US20140315795A1/en not_active Abandoned
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2016
- 2016-11-23 US US15/360,195 patent/US20170073679A1/en not_active Abandoned
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US20150315575A1 (en) | 2015-11-05 |
EP2911697A1 (en) | 2015-09-02 |
JP2016503394A (ja) | 2016-02-04 |
US20140315795A1 (en) | 2014-10-23 |
CA2889608A1 (en) | 2014-05-01 |
WO2014064258A1 (en) | 2014-05-01 |
US20170073679A1 (en) | 2017-03-16 |
AU2013336582A1 (en) | 2015-06-11 |
US9938525B2 (en) | 2018-04-10 |
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