CL2014002742A1 - Agentes de iarn, composiciones y métodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr) (divisional de solicitud 1291-2014) - Google Patents
Agentes de iarn, composiciones y métodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr) (divisional de solicitud 1291-2014)Info
- Publication number
- CL2014002742A1 CL2014002742A1 CL2014002742A CL2014002742A CL2014002742A1 CL 2014002742 A1 CL2014002742 A1 CL 2014002742A1 CL 2014002742 A CL2014002742 A CL 2014002742A CL 2014002742 A CL2014002742 A CL 2014002742A CL 2014002742 A1 CL2014002742 A1 CL 2014002742A1
- Authority
- CL
- Chile
- Prior art keywords
- irid
- transthyretin
- ttr
- agents
- compositions
- Prior art date
Links
- 108010071690 Prealbumin Proteins 0.000 title 1
- 102000009190 Transthyretin Human genes 0.000 title 1
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
Classifications
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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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- 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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- 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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- 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
- 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
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 |
---|---|
CL2014002742A1 true CL2014002742A1 (es) | 2015-02-13 |
Family
ID=48430225
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2014001291A CL2014001291A1 (es) | 2011-11-18 | 2014-05-15 | Agentes de iarn de doble hebra; composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr). |
CL2014002742A CL2014002742A1 (es) | 2011-11-18 | 2014-10-10 | Agentes de iarn, composiciones y métodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr) (divisional de solicitud 1291-2014) |
CL2018002703A CL2018002703A1 (es) | 2011-11-18 | 2018-09-24 | Agentes de iarn, composiciones y métodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr). (divisional solicitud 201401291) |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2014001291A CL2014001291A1 (es) | 2011-11-18 | 2014-05-15 | Agentes de iarn de doble hebra; composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr). |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2018002703A CL2018002703A1 (es) | 2011-11-18 | 2018-09-24 | Agentes de iarn, composiciones y métodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr). (divisional solicitud 201401291) |
Country Status (37)
Country | Link |
---|---|
US (4) | US9399775B2 (es) |
EP (4) | EP3730618A1 (es) |
JP (4) | JP6165158B2 (es) |
KR (4) | KR102095699B1 (es) |
CN (2) | CN108186667B (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) | IL284530B (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 (179)
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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 |
US20050244869A1 (en) * | 2004-04-05 | 2005-11-03 | Brown-Driver Vickie L | Modulation of transthyretin expression |
MX343559B (es) | 2010-04-29 | 2016-11-10 | Ionis Pharmaceuticals Inc | Modulacion de la expresion de transtiretina. |
EP2751270B1 (en) | 2011-08-29 | 2018-08-22 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
ES2923787T3 (es) * | 2011-11-18 | 2022-09-30 | Alnylam Pharmaceuticals Inc | Agentes de iARN modificados |
HUE048622T2 (hu) * | 2011-11-18 | 2020-08-28 | Alnylam Pharmaceuticals Inc | RNSi szerek, készítmények és alkalmazási eljárások transztiretin (TTR) asszociált betegségek kezelésére |
ES2947520T3 (es) * | 2012-08-06 | 2023-08-10 | Alnylam Pharmaceuticals Inc | Proceso para la preparación de agentes de ARN conjugados con carbohidratos |
BR112015013105B1 (pt) | 2012-12-05 | 2022-02-08 | Alnylam Pharmaceuticals, Inc | Agente de rnai de fita dupla capaz de inibir a expressão de pcsk9, seus usos, composição farmacêutica e método de inibição da expressão de pcsk9 em uma célula in vitro |
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 |
RU2686080C2 (ru) | 2013-05-01 | 2019-04-24 | Ионис Фармасьютикалз, Инк. | Композиции и способы |
CA3177846A1 (en) * | 2013-07-11 | 2015-01-15 | 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) |
US10077444B2 (en) | 2013-10-02 | 2018-09-18 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of the LECT2 gene |
EP3060664B1 (en) * | 2013-10-25 | 2021-07-07 | Sanofi | 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 |
EP3798306A1 (en) * | 2013-12-12 | 2021-03-31 | Alnylam Pharmaceuticals, Inc. | Complement component irna compositions and methods of use thereof |
EP3581654B1 (en) | 2013-12-27 | 2021-03-31 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna |
KR20230152154A (ko) * | 2014-02-11 | 2023-11-02 | 알닐람 파마슈티칼스 인코포레이티드 | 케토헥소키나제(KHK) iRNA 조성물 및 그의 사용 방법 |
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 |
US9856475B2 (en) | 2014-03-25 | 2018-01-02 | Arcturus Therapeutics, Inc. | Formulations for treating amyloidosis |
WO2015148582A1 (en) * | 2014-03-25 | 2015-10-01 | Arcturus Therapeutics, Inc. | Transthyretin allele selective una oligomers for gene silencing |
EP3137476B1 (en) | 2014-04-28 | 2019-10-09 | Ionis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
PE20170010A1 (es) * | 2014-05-01 | 2017-03-04 | Ionis Pharmaceuticals Inc | Composiciones y metodos para modular la expresion del factor b del complemento |
WO2015168618A2 (en) | 2014-05-01 | 2015-11-05 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating growth hormone receptor expression |
MX2016014102A (es) | 2014-05-01 | 2017-05-03 | Ionis Pharmaceuticals Inc | Composiciones y metodos para modular la expresion de similar a la angiopoyetina tipo 3. |
KR102366078B1 (ko) | 2014-05-01 | 2022-02-21 | 아이오니스 파마수티컬즈, 인코포레이티드 | Pkk 발현을 조절하는 조성물 및 방법 |
SG10202104570TA (en) * | 2014-05-22 | 2021-06-29 | Alnylam Pharmaceuticals Inc | Angiotensinogen (agt) irna compositions and methods of use thereof |
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JOP20200115A1 (ar) * | 2014-10-10 | 2017-06-16 | Alnylam Pharmaceuticals Inc | تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات)) |
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EP3307316A1 (en) * | 2015-06-12 | 2018-04-18 | Alnylam Pharmaceuticals, Inc. | Complement component c5 irna compositions and methods of use thereof |
CN107849567A (zh) * | 2015-06-26 | 2018-03-27 | 苏州瑞博生物技术有限公司 | 一种siRNA、含有该siRNA的药物组合物和缀合物及它们的应用 |
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