NI201400046A - AGENTES iARN, COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR) - Google Patents
AGENTES iARN, COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR)Info
- Publication number
- NI201400046A NI201400046A NI201400046A NI201400046A NI201400046A NI 201400046 A NI201400046 A NI 201400046A NI 201400046 A NI201400046 A NI 201400046A NI 201400046 A NI201400046 A NI 201400046A NI 201400046 A NI201400046 A NI 201400046A
- Authority
- NI
- Nicaragua
- Prior art keywords
- ttr
- treat
- methods
- agents
- transtiretin
- Prior art date
Links
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- 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
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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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- 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
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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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- 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
- C12N15/1136—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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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- 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
- 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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- 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
- 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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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- 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
- 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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- 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
- 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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- 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
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- 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
- 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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- 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
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
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- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- 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)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Endocrinology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (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
La presente invención proporciona agentes de iARN, por ejemplo, agentes de iARN de doble hebra, que se dirigen al gen de la transtiretina (TTR) y métodos para utilizar dichos agentes de iARN para tratar o prevenir enfermedades asociadas.
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 |
---|---|
NI201400046A true NI201400046A (es) | 2014-12-23 |
Family
ID=48430225
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
NI201400046A NI201400046A (es) | 2011-11-18 | 2014-05-15 | AGENTES iARN, COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR) |
Country Status (38)
Country | Link |
---|---|
US (5) | US9399775B2 (es) |
EP (4) | EP3301177B1 (es) |
JP (4) | JP6165158B2 (es) |
KR (4) | KR102095699B1 (es) |
CN (2) | CN104080794B (es) |
AR (1) | AR088911A1 (es) |
AU (5) | AU2012340159B2 (es) |
BR (1) | BR112014011896B1 (es) |
CA (1) | CA2856243A1 (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) | IL293434B2 (es) |
IN (1) | IN2014CN03463A (es) |
LT (1) | LT3301177T (es) |
MX (2) | 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 (203)
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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 |
MX2011004268A (es) | 2008-10-20 | 2011-06-01 | Alnylam Pharmaceuticals Inc | Composiciones y metodos para inhibir la expresion de transtiretina. |
RU2592669C2 (ru) | 2010-04-29 | 2016-07-27 | Айсис Фармасьютикалс, Инк. | Модуляция экспрессии транстиретина |
DK2751270T3 (en) | 2011-08-29 | 2018-10-29 | Ionis Pharmaceuticals Inc | OLIGOMER-CONJUGATE COMPLEXES AND THEIR USE |
KR20150021489A (ko) | 2011-11-18 | 2015-03-02 | 알닐람 파마슈티칼스 인코포레이티드 | 변형된 RNAi 제제 |
CA2856243A1 (en) * | 2011-11-18 | 2013-05-23 | Alnylam Pharmaceuticals, Inc. | Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases |
WO2014025805A1 (en) | 2012-08-06 | 2014-02-13 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated rna agents and process for their preparation |
EP2929031B9 (en) | 2012-12-05 | 2020-08-12 | Alnylam Pharmaceuticals, Inc. | Pcsk9 irna compositions and methods of use thereof |
LT2970974T (lt) * | 2013-03-14 | 2017-12-11 | Alnylam Pharmaceuticals, Inc. | Komplemento komponento c5 irnr kompozicijos ir jų panaudojimas |
BR112015027369B1 (pt) | 2013-05-01 | 2021-06-08 | Ionis Pharmaceuticals, Inc | compostos compreendendo um oligonucleotídeo modificado e um grupo de conjugado, composição compreendendo os referidos compostos e usos dos mesmos |
EP4039278A1 (en) | 2013-07-11 | 2022-08-10 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
WO2015042564A1 (en) | 2013-09-23 | 2015-03-26 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing transthyretin (ttr) associated diseases |
MX2016004230A (es) * | 2013-10-02 | 2016-10-21 | Alnylam Pharmaceuticals Inc | Composiciones y metodos para inhibir la expresion del gen lect2. |
WO2015061536A1 (en) * | 2013-10-25 | 2015-04-30 | Regulus Therapeutics Inc. | Microrna compounds and methods for modulating mir-21 activity |
CA2932904A1 (en) * | 2013-12-06 | 2015-06-11 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of transthyretin (ttr) by double-stranded rna |
KR102736538B1 (ko) * | 2013-12-12 | 2024-12-03 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 성분 iRNA 조성물 및 이의 이용 방법 |
WO2015100436A1 (en) | 2013-12-27 | 2015-07-02 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna |
CN106103718B (zh) * | 2014-02-11 | 2021-04-02 | 阿尔尼拉姆医药品有限公司 | 己酮糖激酶(KHK)iRNA组合物及其使用方法 |
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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 |
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 |
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EA201692370A1 (ru) * | 2014-05-22 | 2017-03-31 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ иРНК АНГИОТЕНЗИНОГЕНА (AGT) И СПОСОБЫ ИХ ИСПОЛЬЗОВАНИЯ |
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- 2017-06-20 JP JP2017120295A patent/JP6513135B2/ja active Active
- 2017-09-07 AU AU2017225076A patent/AU2017225076C1/en active Active
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2018
- 2018-01-25 IL IL257166A patent/IL257166A/en unknown
- 2018-09-24 CL CL2018002703A patent/CL2018002703A1/es unknown
- 2018-10-04 HK HK18112708.8A patent/HK1253410A1/zh unknown
- 2018-12-03 IL IL263458A patent/IL263458B/en unknown
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2019
- 2019-04-09 JP JP2019073911A patent/JP2019131595A/ja not_active Withdrawn
- 2019-10-03 AU AU2019240658A patent/AU2019240658B2/en active Active
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2020
- 2020-01-09 US US16/738,014 patent/US20200318111A1/en not_active Abandoned
- 2020-04-28 HR HRP20200672TT patent/HRP20200672T1/hr unknown
- 2020-06-17 CY CY20201100553T patent/CY1123693T1/el unknown
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2021
- 2021-07-09 US US17/371,182 patent/US20230010288A1/en not_active Abandoned
- 2021-07-12 JP JP2021114868A patent/JP2021168677A/ja active Pending
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2022
- 2022-09-15 AU AU2022231749A patent/AU2022231749B2/en active Active
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2023
- 2023-11-15 US US18/509,440 patent/US20240279652A1/en active Pending
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2024
- 2024-12-12 AU AU2024278344A patent/AU2024278344A1/en active Pending
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