PE20142362A1 - Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr) - Google Patents
Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr)Info
- Publication number
- PE20142362A1 PE20142362A1 PE2014000707A PE2014000707A PE20142362A1 PE 20142362 A1 PE20142362 A1 PE 20142362A1 PE 2014000707 A PE2014000707 A PE 2014000707A PE 2014000707 A PE2014000707 A PE 2014000707A PE 20142362 A1 PE20142362 A1 PE 20142362A1
- Authority
- PE
- Peru
- Prior art keywords
- ttr
- iarn
- transtiretin
- treat
- associated diseases
- Prior art date
Links
- 201000010099 disease Diseases 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title 1
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 230000000295 complement effect Effects 0.000 abstract 2
- 239000002773 nucleotide Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
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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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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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
- A61K31/70—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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- 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
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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
- A61P25/28—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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P5/00—Drugs for disorders of the endocrine system
- A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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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
- C12N15/113—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
- C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
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- 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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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Endocrinology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
- Heterocyclic Carbon Compounds Containing A Hetero Ring Having Oxygen Or Sulfur (AREA)
Abstract
REFERIDO A UN AGENTE DE IARN DE DOBLE HEBRA QUE COMPRENDE UNA HEBRA SENTIDO COMPLEMENTARIA A UNA HEBRA ANTISENTIDO, EN DONDE DICHA HEBRA ANTISENTIDO COMPRENDE UNA REGION COMPLEMENTARIA A UNA PARTE DE UNA TRANSTIRETINA QUE CODIFICA ARNM (TTR), EN DONDE CADA HEBRA TIENE APROXIMADAMENTE 14 A 30 NUCLEOTIDOS. REFERIDO TAMBIEN A UNA COMPOSICION FARMACEUTICA EN DONDE EL AGENTE DE IARN DE DOBLE HEBRA SON CARACTERIZADOS PORQUE SE DIRIGEN AL GEN DE LA TRASTIRETINA (TTR) PARA TRATAR O PREVENIR ENFERMEDADES ASOCIADAS A TTR.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161561710P | 2011-11-18 | 2011-11-18 | |
| US201261615618P | 2012-03-26 | 2012-03-26 | |
| US201261680098P | 2012-08-06 | 2012-08-06 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20142362A1 true PE20142362A1 (es) | 2015-01-30 |
Family
ID=48430225
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2014000707A PE20142362A1 (es) | 2011-11-18 | 2012-11-16 | Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr) |
Country Status (38)
| Country | Link |
|---|---|
| US (5) | US9399775B2 (es) |
| EP (4) | EP2780353B1 (es) |
| JP (4) | JP6165158B2 (es) |
| KR (4) | KR20220045091A (es) |
| CN (2) | CN108186667B (es) |
| AR (1) | AR088911A1 (es) |
| AU (5) | AU2012340159B2 (es) |
| BR (1) | BR112014011896B1 (es) |
| CA (2) | CA2856243C (es) |
| CL (3) | CL2014001291A1 (es) |
| CO (1) | CO7020872A2 (es) |
| CR (1) | CR20140232A (es) |
| CY (1) | CY1123693T1 (es) |
| DK (1) | DK3301177T3 (es) |
| DO (1) | DOP2014000107A (es) |
| EC (1) | ECSP14005606A (es) |
| ES (1) | ES2800065T3 (es) |
| GT (1) | GT201400097A (es) |
| HK (2) | HK1200171A1 (es) |
| HR (1) | HRP20200672T1 (es) |
| HU (1) | HUE048622T2 (es) |
| IL (6) | IL284530B (es) |
| IN (1) | IN2014CN03463A (es) |
| LT (1) | LT3301177T (es) |
| MX (3) | MX348575B (es) |
| MY (1) | MY167390A (es) |
| NI (1) | NI201400046A (es) |
| PE (1) | PE20142362A1 (es) |
| PH (1) | PH12014501106B1 (es) |
| PL (1) | PL3301177T3 (es) |
| PT (1) | PT3301177T (es) |
| RS (1) | RS60414B1 (es) |
| RU (1) | RU2678807C2 (es) |
| SG (2) | SG10201912170WA (es) |
| SI (1) | SI3301177T1 (es) |
| SM (1) | SMT202000308T1 (es) |
| UA (1) | UA118649C2 (es) |
| WO (1) | WO2013075035A1 (es) |
Families Citing this family (220)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9879266B2 (en) | 2002-11-14 | 2018-01-30 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
| US9228186B2 (en) | 2002-11-14 | 2016-01-05 | Thermo Fisher Scientific Inc. | Methods and compositions for selecting siRNA of improved functionality |
| US20050244869A1 (en) * | 2004-04-05 | 2005-11-03 | Brown-Driver Vickie L | Modulation of transthyretin expression |
| KR102354558B1 (ko) | 2008-10-20 | 2022-01-25 | 알닐람 파마슈티칼스 인코포레이티드 | 트랜스티레틴의 발현을 억제하기 위한 조성물 및 방법 |
| SI2563920T1 (sl) | 2010-04-29 | 2017-05-31 | Ionis Pharmaceuticals, Inc. | Modulacija izražanja transtiretina |
| EP3453761A1 (en) | 2011-08-29 | 2019-03-13 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| BR112014011896B1 (pt) * | 2011-11-18 | 2021-01-19 | Alnylam Pharmaceuticals, Inc. | Agentes de rnai, composições, método de inibição de expressão de uma transtirretina(ttr), uso destes agentes, e kits |
| CN108977446A (zh) * | 2011-11-18 | 2018-12-11 | 阿尔尼拉姆医药品有限公司 | 修饰的RNAi试剂 |
| WO2014025805A1 (en) * | 2012-08-06 | 2014-02-13 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated rna agents and process for their preparation |
| ES2657608T3 (es) | 2012-12-05 | 2018-03-06 | Alnylam Pharmaceuticals, Inc. | Composiciones de arni de pcsk9 y métodos de uso de las mismas |
| PT2970974T (pt) * | 2013-03-14 | 2017-11-29 | Alnylam Pharmaceuticals Inc | Composições de irna de componente do complemento c5 e métodos para a sua utilização |
| SG10201906382QA (en) | 2013-05-01 | 2019-08-27 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating hbv and ttr expression |
| JP6702862B2 (ja) * | 2013-07-11 | 2020-06-03 | アルニラム ファーマスーティカルズ インコーポレイテッドAlnylam Pharmaceuticals, Inc. | オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法 |
| WO2015042564A1 (en) | 2013-09-23 | 2015-03-26 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing transthyretin (ttr) associated diseases |
| CN109536493A (zh) * | 2013-10-02 | 2019-03-29 | 阿尔尼拉姆医药品有限公司 | 用于抑制lect2基因表达的组合物和方法 |
| WO2015061536A1 (en) * | 2013-10-25 | 2015-04-30 | Regulus Therapeutics Inc. | Microrna compounds and methods for modulating mir-21 activity |
| US10150965B2 (en) | 2013-12-06 | 2018-12-11 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of transthyretin (TTR) by double-stranded RNA |
| EA201691215A1 (ru) * | 2013-12-12 | 2016-11-30 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ iRNA К КОМПОНЕНТУ КОМПЛЕМЕНТА И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| DK3087184T3 (da) | 2013-12-27 | 2019-07-29 | Dicerna Pharmaceuticals Inc | Fremgangsmåder og sammensætninger til specifik inhibering af glycolatoxidase (hao1) med dobbeltstrenget rna |
| EP3960860A3 (en) * | 2014-02-11 | 2022-06-08 | Alnylam Pharmaceuticals, Inc. | Ketohexokinase (khk) irna compositions and methods of use thereof |
| JP6771387B2 (ja) * | 2014-03-25 | 2020-10-21 | アークトゥラス・セラピューティクス・インコーポレイテッドArcturus Therapeutics,Inc. | 遺伝子サイレンシング用トランスサイレチン対立遺伝子選択的unaオリゴマー |
| US9856475B2 (en) | 2014-03-25 | 2018-01-02 | Arcturus Therapeutics, Inc. | Formulations for treating amyloidosis |
| JP6752151B2 (ja) * | 2014-03-25 | 2020-09-09 | アークトゥラス・セラピューティクス・インコーポレイテッドArcturus Therapeutics,Inc. | 遺伝子サイレンシングにおいて低下したoff−target効果を有するunaオリゴマー |
| US9926556B2 (en) | 2014-04-28 | 2018-03-27 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
| SG10201809953QA (en) | 2014-05-01 | 2018-12-28 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating complement factor b expression |
| CN110903337A (zh) | 2014-05-01 | 2020-03-24 | Ionis制药公司 | 用于调节生长激素受体表达的组合物和方法 |
| WO2015168532A2 (en) | 2014-05-01 | 2015-11-05 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating pkk expression |
| JP2017521045A (ja) | 2014-05-01 | 2017-08-03 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | アンジオポエチン様因子3発現を調節するための組成物及び方法 |
| US10570169B2 (en) | 2014-05-22 | 2020-02-25 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| SG11201609376SA (en) * | 2014-05-22 | 2016-12-29 | Alnylam Pharmaceuticals Inc | Angiotensinogen (agt) irna compositions and methods of use thereof |
| IL316808A (en) | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| PL3185957T3 (pl) | 2014-08-29 | 2022-11-14 | Alnylam Pharmaceuticals, Inc. | Patisiran do zastosowania w leczeniu amyloidozy związanej z transtyretyną |
| KR102692773B1 (ko) | 2014-09-07 | 2024-08-08 | 셀렉타 바이오사이언시즈, 인크. | 유전자 편집 항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물 |
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