IN2014CN03463A - - Google Patents
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- Publication number
- IN2014CN03463A IN2014CN03463A IN3463CHN2014A IN2014CN03463A IN 2014CN03463 A IN2014CN03463 A IN 2014CN03463A IN 3463CHN2014 A IN3463CHN2014 A IN 3463CHN2014A IN 2014CN03463 A IN2014CN03463 A IN 2014CN03463A
- Authority
- IN
- India
- Prior art keywords
- rnai agents
- ttr
- transthyretin
- gene
- treating
- Prior art date
Links
- 108010071690 Prealbumin Proteins 0.000 abstract 3
- 108091030071 RNAI Proteins 0.000 abstract 3
- 102000009190 Transthyretin Human genes 0.000 abstract 3
- 230000009368 gene silencing by RNA Effects 0.000 abstract 3
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
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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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- 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
- C12N2310/346—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
- 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
- C12N2310/351—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
- 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
- C12N2310/353—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)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Neurosurgery (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)
Applications Claiming Priority (4)
| 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 | |
| PCT/US2012/065691 WO2013075035A1 (en) | 2011-11-18 | 2012-11-16 | Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IN2014CN03463A true IN2014CN03463A (es) | 2015-10-09 |
Family
ID=48430225
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IN3463CHN2014 IN2014CN03463A (es) | 2011-11-18 | 2012-11-16 |
Country Status (37)
| Country | Link |
|---|---|
| US (4) | US9399775B2 (es) |
| EP (4) | EP3913056A1 (es) |
| JP (4) | JP6165158B2 (es) |
| KR (4) | KR102263352B1 (es) |
| CN (2) | CN104080794B (es) |
| AR (1) | AR088911A1 (es) |
| AU (4) | 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) | PH12014501106A1 (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) |
| UA (1) | UA118649C2 (es) |
| WO (1) | WO2013075035A1 (es) |
Families Citing this family (180)
| 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 |
| PT2563920T (pt) | 2010-04-29 | 2017-05-26 | Ionis Pharmaceuticals Inc | Modulação da expressão de transtirretina |
| WO2013033230A1 (en) | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| IL293434B2 (en) * | 2011-11-18 | 2024-05-01 | Alnylam Pharmaceuticals Inc | RNAI factors, preparations and methods of using them for the treatment of transthyretin-related diseases |
| MX2018012038A (es) | 2011-11-18 | 2021-09-06 | Alnylam Pharmaceuticals Inc | Agentes de iarn modificados. |
| AU2013299717B2 (en) * | 2012-08-06 | 2018-06-28 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugated RNA agents and process for their preparation |
| LT2929031T (lt) | 2012-12-05 | 2018-02-12 | Alnylam Pharmaceuticals, Inc. | Pcsk9 irna kompozicijos ir jų naudojimo būdai |
| CA2904654C (en) * | 2013-03-14 | 2023-12-05 | Alnylam Pharmaceuticals, Inc. | Complement component c5 irna compositions and methods of use thereof |
| JP6995478B2 (ja) | 2013-05-01 | 2022-01-14 | 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 | Ｈｂｖおよびｔｔｒ発現を調節するための組成物および方法 |
| US10808246B2 (en) * | 2013-07-11 | 2020-10-20 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide-ligand conjugates and process for their preparation |
| AR097738A1 (es) | 2013-09-23 | 2016-04-13 | Alnylam Pharmaceuticals Inc | Métodos para tratar o prevenir enfermedades asociadas con la transtiretina (ttr) |
| TWI669393B (zh) | 2013-10-02 | 2019-08-21 | 艾爾妮蘭製藥公司 | 抑制ｌｅｃｔ２基因表現之組合物及方法 |
| EP3060664B1 (en) * | 2013-10-25 | 2021-07-07 | Sanofi | 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 |
| KR20210158880A (ko) * | 2013-12-12 | 2021-12-31 | 알닐람 파마슈티칼스 인코포레이티드 | 보체 성분 ｉＲＮＡ 조성물 및 이의 이용 방법 |
| DK3087184T3 (da) | 2013-12-27 | 2019-07-29 | Dicerna Pharmaceuticals Inc | Fremgangsmåder og sammensætninger til specifik inhibering af glycolatoxidase (hao1) med dobbeltstrenget rna |
| EA201691587A1 (ru) * | 2014-02-11 | 2017-01-30 | Элнилэм Фармасьютикалз, Инк. | КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ КЕТОГЕКСОКИНАЗЫ (KHK) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ |
| US9856475B2 (en) | 2014-03-25 | 2018-01-02 | Arcturus Therapeutics, Inc. | Formulations for treating amyloidosis |
| 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 |
| CN110846316B (zh) * | 2014-03-25 | 2023-10-31 | 阿克丘勒斯治疗公司 | 在基因沉默中具有降低的脱靶效应的una寡聚物 |
| EP3137476B1 (en) | 2014-04-28 | 2019-10-09 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
| ES2849600T3 (es) | 2014-05-01 | 2021-08-19 | Ionis Pharmaceuticals Inc | Conjugados de oligonucleótidos antisentido modificados y su uso para modular la expresión de PKK |
| BR112016024850B1 (pt) | 2014-05-01 | 2021-07-06 | Ionis Pharmaceuticals, Inc | compostos e composições para modulação da expressão de angptl3 e seus usos |
| SG11201608502TA (en) * | 2014-05-01 | 2016-11-29 | Ionis Pharmaceuticals Inc | Compositions and methods for modulating complement factor b expression |
| CA2942570A1 (en) | 2014-05-01 | 2015-11-05 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating growth hormone receptor expression |
| CA3215908A1 (en) * | 2014-05-22 | 2015-11-26 | Alnylam Pharmaceuticals, Inc. | Angiotensinogen (agt) irna compositions and methods of use thereof |
| WO2015179693A1 (en) | 2014-05-22 | 2015-11-26 | Isis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| NZ730296A (en) | 2014-08-20 | 2023-09-29 | Alnylam Pharmaceuticals Inc | Modified double-stranded rna agents |
| US10060921B2 (en) | 2014-08-29 | 2018-08-28 | Alnylam Pharmaceuticals, Inc. | Methods of treating transthyretin (TTR) mediated amyloidosis |
| KR20170045344A (ko) | 2014-09-07 | 2017-04-26 | 셀렉타 바이오사이언시즈, 인크. | 항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물 |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| JOP20200115A1 (ar) | 2014-10-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) |
| JOP20200092A1 (ar) | 2014-11-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها |
| AU2015350120B2 (en) | 2014-11-17 | 2021-05-27 | Alnylam Pharmaceuticals, Inc. | Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof |
| JP7105065B2 (ja) | 2014-12-15 | 2022-07-22 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | リガンド修飾二本鎖核酸 |
| WO2016097212A1 (en) | 2014-12-17 | 2016-06-23 | Proqr Therapeutics Ii B.V. | Targeted rna editing |
| AU2015364508A1 (en) * | 2014-12-18 | 2017-07-06 | Alnylam Pharmaceuticals, Inc. | ReversirTM compounds |
| CA2976445A1 (en) | 2015-02-13 | 2016-08-18 | Alnylam Pharmaceuticals, Inc. | Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof |
| CA2979703A1 (en) * | 2015-03-17 | 2016-09-22 | Arrowhead Pharmaceuticals, Inc. | Compositions and methods for inhibiting gene expression of factor xii |
| WO2016161299A1 (en) | 2015-04-01 | 2016-10-06 | Arcturus Therapeutics, Inc. | Therapeutic una oligomers and uses thereof |
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