BR112018011450A2 - métodos e composições para o tratamento de um transtorno associado à serpinc1 - Google Patents
métodos e composições para o tratamento de um transtorno associado à serpinc1Info
- Publication number
- BR112018011450A2 BR112018011450A2 BR112018011450A BR112018011450A BR112018011450A2 BR 112018011450 A2 BR112018011450 A2 BR 112018011450A2 BR 112018011450 A BR112018011450 A BR 112018011450A BR 112018011450 A BR112018011450 A BR 112018011450A BR 112018011450 A2 BR112018011450 A2 BR 112018011450A2
- Authority
- BR
- Brazil
- Prior art keywords
- serpinc1
- compositions
- methods
- associated disorder
- treating
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 4
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 2
- 208000031220 Hemophilia Diseases 0.000 abstract 1
- 208000009292 Hemophilia A Diseases 0.000 abstract 1
- 101150026876 SERPINC1 gene Proteins 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- -1 for example Proteins 0.000 abstract 1
- 208000031169 hemorrhagic disease Diseases 0.000 abstract 1
- 229920002477 rna polymer Polymers 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- 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
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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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
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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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
- C07H21/04—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 deoxyribosyl 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
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
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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
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
Abstract
a presente invenção refere-se a rnai, por exemplo, ácido ribonucleico fita dupla (dsrna), composições que têm como alvo o gene de serpinc1 e métodos de uso de tais rnai, por exemplo, dsrna, composições para inibir a expressão de serpinc1 e para tratar indivíduos que têm um transtorno associado à serpinc1, por exemplo, um transtorno hemorrágico, tal como uma hemofilia.
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562264013P | 2015-12-07 | 2015-12-07 | |
US201662315228P | 2016-03-30 | 2016-03-30 | |
US201662366304P | 2016-07-25 | 2016-07-25 | |
US201662429241P | 2016-12-02 | 2016-12-02 | |
PCT/US2016/065245 WO2017100236A1 (en) | 2015-12-07 | 2016-12-07 | Methods and compositions for treating a serpinc1-associated disorder |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112018011450A2 true BR112018011450A2 (pt) | 2018-11-27 |
Family
ID=58800158
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112018011450A BR112018011450A2 (pt) | 2015-12-07 | 2016-12-07 | métodos e composições para o tratamento de um transtorno associado à serpinc1 |
Country Status (15)
Country | Link |
---|---|
US (2) | US11091759B2 (pt) |
EP (1) | EP3386518A1 (pt) |
JP (3) | JP6985283B2 (pt) |
KR (3) | KR20210157474A (pt) |
CN (1) | CN108601795A (pt) |
AU (2) | AU2016368289C1 (pt) |
BR (1) | BR112018011450A2 (pt) |
CA (1) | CA3007014A1 (pt) |
CO (1) | CO2018006590A2 (pt) |
IL (2) | IL259795B2 (pt) |
MX (1) | MX2018006989A (pt) |
RU (2) | RU2754188C2 (pt) |
SG (2) | SG10201913085TA (pt) |
TW (1) | TWI743069B (pt) |
WO (1) | WO2017100236A1 (pt) |
Families Citing this family (15)
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EP3212794B1 (en) | 2014-10-30 | 2021-04-07 | Genzyme Corporation | Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof |
IL259795B2 (en) | 2015-12-07 | 2024-04-01 | Genzyme Corp | Methods and preparations for the treatment of diseases related to SERPINC1 |
US20200208150A1 (en) * | 2017-07-07 | 2020-07-02 | Alnylam Pharmaceuticals, Inc. | METHODS FOR TREATING OR PREVENTING CONTACT-ACTIVATION PATHWAY-ASSOCIATED DISEASES USING iRNA COMPOSITIONS TARGETING FACTOR XII (HAGEMAN FACTOR) (F12) |
AU2018301801A1 (en) * | 2017-07-10 | 2020-02-06 | Genzyme Corporation | Methods and compositions for treating a bleeding event in a subject having hemophilia |
US20210155926A1 (en) * | 2018-04-05 | 2021-05-27 | Silence Therapeutics Gmbh | siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS |
EP3550021A1 (en) * | 2018-04-05 | 2019-10-09 | Silence Therapeutics GmbH | Products and compositions for inhibiting expression of a target gene |
CN113631709A (zh) * | 2018-12-20 | 2021-11-09 | 普拉克西斯精密药物股份有限公司 | 用于治疗kcnt1相关病症的组合物和方法 |
SG11202107669WA (en) * | 2019-01-16 | 2021-08-30 | Genzyme Corp | Serpinc1 irna compositions and methods of use thereof |
CN115942940A (zh) | 2020-06-22 | 2023-04-07 | 建新公司 | 治疗血友病的方法及组合物 |
WO2022120291A1 (en) | 2020-12-06 | 2022-06-09 | Genzyme Corporation | Treatment of hemophilia with fitusiran |
JP2023552554A (ja) | 2020-12-06 | 2023-12-18 | ジェンザイム・コーポレーション | フィツシランを用いた血友病の処置 |
WO2023118150A1 (en) | 2021-12-22 | 2023-06-29 | Royal College Of Surgeons In Ireland | A conjugate for use in localising a molecule to the vascular endothelium. |
TW202342065A (zh) | 2021-12-22 | 2023-11-01 | 美商健臻公司 | 治療血友病的方法和組成物 |
WO2023240193A2 (en) | 2022-06-08 | 2023-12-14 | Genzyme Corporation | Treatment of hemophilia with fitusiran in pediatric patients |
WO2023240199A2 (en) | 2022-06-08 | 2023-12-14 | Genzyme Corporation | Treatment of hemophilia with fitusiran |
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2016
- 2016-12-07 IL IL259795A patent/IL259795B2/en unknown
- 2016-12-07 JP JP2018549141A patent/JP6985283B2/ja active Active
- 2016-12-07 KR KR1020217041486A patent/KR20210157474A/ko not_active IP Right Cessation
- 2016-12-07 KR KR1020237025828A patent/KR20230119027A/ko not_active Application Discontinuation
- 2016-12-07 IL IL308717A patent/IL308717A/en unknown
- 2016-12-07 TW TW105140410A patent/TWI743069B/zh active
- 2016-12-07 KR KR1020187019085A patent/KR20180095843A/ko not_active IP Right Cessation
- 2016-12-07 SG SG10201913085TA patent/SG10201913085TA/en unknown
- 2016-12-07 CN CN201680081199.2A patent/CN108601795A/zh active Pending
- 2016-12-07 CA CA3007014A patent/CA3007014A1/en active Pending
- 2016-12-07 RU RU2018123712A patent/RU2754188C2/ru active
- 2016-12-07 RU RU2021124592A patent/RU2021124592A/ru unknown
- 2016-12-07 AU AU2016368289A patent/AU2016368289C1/en active Active
- 2016-12-07 WO PCT/US2016/065245 patent/WO2017100236A1/en active Application Filing
- 2016-12-07 BR BR112018011450A patent/BR112018011450A2/pt active Search and Examination
- 2016-12-07 US US15/371,300 patent/US11091759B2/en active Active
- 2016-12-07 SG SG11201804729RA patent/SG11201804729RA/en unknown
- 2016-12-07 EP EP16822817.9A patent/EP3386518A1/en active Pending
- 2016-12-07 MX MX2018006989A patent/MX2018006989A/es unknown
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2018
- 2018-06-26 CO CONC2018/0006590A patent/CO2018006590A2/es unknown
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2021
- 2021-07-14 US US17/375,785 patent/US20220017902A1/en active Pending
- 2021-11-24 JP JP2021189823A patent/JP7422120B2/ja active Active
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2023
- 2023-02-14 AU AU2023200828A patent/AU2023200828A1/en active Pending
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