SG11201804729RA - Methods and compositions for treating a serpinc1-associated disorder - Google Patents
Methods and compositions for treating a serpinc1-associated disorderInfo
- Publication number
- SG11201804729RA SG11201804729RA SG11201804729RA SG11201804729RA SG11201804729RA SG 11201804729R A SG11201804729R A SG 11201804729RA SG 11201804729R A SG11201804729R A SG 11201804729RA SG 11201804729R A SG11201804729R A SG 11201804729RA SG 11201804729R A SG11201804729R A SG 11201804729RA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- street
- cambridge
- floor
- compositions
- Prior art date
Links
- 239000000203 mixture Substances 0.000 title abstract 4
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 208000031220 Hemophilia Diseases 0.000 abstract 1
- 208000009292 Hemophilia A Diseases 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 208000031169 hemorrhagic disease Diseases 0.000 abstract 1
- 230000008520 organization Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 229920002477 rna polymer Polymers 0.000 abstract 1
- 102220240796 rs553605556 Human genes 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
Classifications
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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
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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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
-
- 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
-
- 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
-
- 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.
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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 |
|---|---|
| SG11201804729RA true SG11201804729RA (en) | 2018-07-30 |
Family
ID=58800158
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201804729RA SG11201804729RA (en) | 2015-12-07 | 2016-12-07 | Methods and compositions for treating a serpinc1-associated disorder |
| SG10201913085TA SG10201913085TA (en) | 2015-12-07 | 2016-12-07 | Methods and compositions for treating a serpinc1-associated disorder |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG10201913085TA SG10201913085TA (en) | 2015-12-07 | 2016-12-07 | Methods and compositions for treating a serpinc1-associated disorder |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US11091759B2 (ru) |
| EP (1) | EP3386518A1 (ru) |
| JP (3) | JP6985283B2 (ru) |
| KR (3) | KR20210157474A (ru) |
| CN (1) | CN108601795A (ru) |
| AU (2) | AU2016368289C1 (ru) |
| BR (1) | BR112018011450A2 (ru) |
| CA (1) | CA3007014A1 (ru) |
| CO (1) | CO2018006590A2 (ru) |
| IL (2) | IL259795B2 (ru) |
| MX (1) | MX2018006989A (ru) |
| RU (2) | RU2754188C2 (ru) |
| SG (2) | SG11201804729RA (ru) |
| TW (1) | TWI743069B (ru) |
| WO (1) | WO2017100236A1 (ru) |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3212794B1 (en) | 2014-10-30 | 2021-04-07 | Genzyme Corporation | Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof |
| US11091759B2 (en) | 2015-12-07 | 2021-08-17 | Genzyme Corporation | Methods and compositions for treating a Serpinc1-associated disorder |
| EP3649243A1 (en) * | 2017-07-07 | 2020-05-13 | Alnylam Pharmaceuticals, Inc. | Methods for treating or preventing contact-activation pathway-associated diseases using irna compositions targeting factor xii (hageman factor) (f12) |
| WO2019014187A1 (en) * | 2017-07-10 | 2019-01-17 | Genzyme Corporation | METHODS AND COMPOSITIONS FOR TREATING A BLEEDING EVENT IN A SUBJECT WITH HEMOPHILIA |
| EP3550021A1 (en) * | 2018-04-05 | 2019-10-09 | Silence Therapeutics GmbH | Products and compositions for inhibiting expression of a target gene |
| US20210155926A1 (en) * | 2018-04-05 | 2021-05-27 | Silence Therapeutics Gmbh | siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS |
| EP3898977A1 (en) * | 2018-12-20 | 2021-10-27 | Praxis Precision Medicines, Inc. | Compositions and methods for the treatment of kcnt1 related disorders |
| JP2022517270A (ja) * | 2019-01-16 | 2022-03-07 | ジェンザイム・コーポレーション | Ｓｅｒｐｉｎｃ１ ｉＲＮＡ組成物およびその使用方法 |
| CA3188137A1 (en) | 2020-06-22 | 2021-12-30 | Genzyme Corporation | Methods and compositions for treating hemophilia |
| KR20230117403A (ko) | 2020-12-06 | 2023-08-08 | 젠자임 코포레이션 | 피투시란을 이용한 혈우병의 치료 |
| WO2022120291A1 (en) | 2020-12-06 | 2022-06-09 | Genzyme Corporation | Treatment of hemophilia with fitusiran |
| 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 |
| US20240000744A1 (en) | 2022-06-08 | 2024-01-04 | Genzyme Corporation | Treatment of Hemophilia with Fitusiran |
| WO2023240193A2 (en) | 2022-06-08 | 2023-12-14 | Genzyme Corporation | Treatment of hemophilia with fitusiran in pediatric patients |
| WO2024094699A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Container holder and drug delivery device comprising the container holder |
| WO2024094702A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Device body for a drug delivery device, assembly for a drug delivery device and drug delivery device |
| WO2024094698A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Front sub-assembly for a drug delivery device |
| WO2024094707A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Drug delivery device with feedback element and method for providing a user of a drug delivery device feedback regarding a dose dispensing process |
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| WO2024094701A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Assembly for a drug delivery device |
| WO2024094705A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Drug delivery device having a two-part user indicator |
| WO2024094703A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Plunger for expelling a drug, drug delivery device, rear sub-assembly and corresponding methods |
| WO2024094700A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Method for assembling an assembly for a drug delivery device and drug delivery device. |
| WO2024094704A1 (en) | 2022-10-31 | 2024-05-10 | Sanofi | Arrangement for a drug delivery device, drug delivery device and method for assembly |
| WO2024097190A1 (en) | 2022-11-02 | 2024-05-10 | Genzyme Corporation | Systems and methods for modeling thrombin-antithrombin |
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| WO2015175510A1 (en) * | 2014-05-12 | 2015-11-19 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating a serpinc1-associated disorder |
| US10689647B2 (en) | 2015-05-08 | 2020-06-23 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of antithrombin 3 (AT3) by double-stranded RNA |
| US11091759B2 (en) | 2015-12-07 | 2021-08-17 | Genzyme Corporation | Methods and compositions for treating a Serpinc1-associated disorder |
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