BR112022005028A2 - Molécula de ácido nucleico para tratamento de trombocitopenia e uso da mesma - Google Patents
Molécula de ácido nucleico para tratamento de trombocitopenia e uso da mesmaInfo
- Publication number
- BR112022005028A2 BR112022005028A2 BR112022005028A BR112022005028A BR112022005028A2 BR 112022005028 A2 BR112022005028 A2 BR 112022005028A2 BR 112022005028 A BR112022005028 A BR 112022005028A BR 112022005028 A BR112022005028 A BR 112022005028A BR 112022005028 A2 BR112022005028 A2 BR 112022005028A2
- Authority
- BR
- Brazil
- Prior art keywords
- nucleic acid
- thpo
- small activating
- gene
- strand
- Prior art date
Links
- 108020004707 nucleic acids Proteins 0.000 title abstract 10
- 102000039446 nucleic acids Human genes 0.000 title abstract 10
- 150000007523 nucleic acids Chemical class 0.000 title abstract 10
- 206010043554 thrombocytopenia Diseases 0.000 title abstract 3
- 101150116986 THPO gene Proteins 0.000 abstract 8
- 230000003213 activating effect Effects 0.000 abstract 4
- 239000008194 pharmaceutical composition Substances 0.000 abstract 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 3
- 108700039691 Genetic Promoter Regions Proteins 0.000 abstract 3
- SXXLKZCNJHJYFL-UHFFFAOYSA-N 4,5,6,7-tetrahydro-[1,2]oxazolo[4,5-c]pyridin-5-ium-3-olate Chemical compound C1CNCC2=C1ONC2=O SXXLKZCNJHJYFL-UHFFFAOYSA-N 0.000 abstract 2
- 101000799461 Homo sapiens Thrombopoietin Proteins 0.000 abstract 2
- 208000008425 Protein deficiency Diseases 0.000 abstract 2
- 102100034195 Thrombopoietin Human genes 0.000 abstract 2
- 201000010099 disease Diseases 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 238000009472 formulation Methods 0.000 abstract 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 1
- 230000004913 activation Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
Classifications
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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
-
- 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
-
- 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
-
- 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
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- 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
- C12N2310/11—Antisense
-
- 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
-
- 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
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- 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
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
-
- 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
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN201910891508 | 2019-09-20 | ||
| PCT/CN2020/116227 WO2021052470A1 (zh) | 2019-09-20 | 2020-09-18 | 用于治疗血小板减少症的核酸分子及其应用 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022005028A2 true BR112022005028A2 (pt) | 2022-07-05 |
Family
ID=74883124
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022005028A BR112022005028A2 (pt) | 2019-09-20 | 2020-09-18 | Molécula de ácido nucleico para tratamento de trombocitopenia e uso da mesma |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20230212565A1 (https=) |
| EP (1) | EP4036232A4 (https=) |
| JP (1) | JP2022552940A (https=) |
| KR (1) | KR20220070206A (https=) |
| CN (1) | CN114585738A (https=) |
| AU (1) | AU2020349991A1 (https=) |
| BR (1) | BR112022005028A2 (https=) |
| CA (1) | CA3148158A1 (https=) |
| CO (1) | CO2022004857A2 (https=) |
| PE (1) | PE20220604A1 (https=) |
| TW (1) | TW202126808A (https=) |
| WO (1) | WO2021052470A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111849968A (zh) | 2019-04-30 | 2020-10-30 | 中美瑞康核酸技术(南通)研究院有限公司 | 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用 |
| WO2024216109A1 (en) * | 2023-04-12 | 2024-10-17 | Alnylam Pharmaceuticals, Inc. | Formulations for oligonucleotide delivery |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6270989B1 (en) * | 1991-11-05 | 2001-08-07 | Transkaryotic Therapies, Inc. | Protein production and delivery |
| GB2285446B (en) * | 1994-01-03 | 1999-07-28 | Genentech Inc | Thrombopoietin |
| EP1871426B1 (en) * | 2005-04-15 | 2017-06-07 | The Regents of The University of California | Small activating rna molecules and their use |
| WO2007086990A2 (en) * | 2005-11-17 | 2007-08-02 | Board Of Regents, The University Of Texas System | Modulation of gene expression by oligomers targeted to chromosomal dna |
| KR101766408B1 (ko) | 2009-06-10 | 2017-08-10 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| GB201010557D0 (en) * | 2010-06-23 | 2010-08-11 | Mina Therapeutics Ltd | RNA molecules and uses thereof |
| KR102195514B1 (ko) * | 2011-06-21 | 2020-12-29 | 미나 테라퓨틱스 리미티드 | 알부민 생산 및 세포 증식 |
| CN106032532A (zh) * | 2015-03-17 | 2016-10-19 | 中国医学科学院北京协和医院 | 一种小激活rna及其制备方法和应用 |
| KR102599712B1 (ko) * | 2015-04-22 | 2023-11-09 | 미나 테라퓨틱스 리미티드 | C/EBP 알파 saRNA 조성물 및 사용 방법 |
| US20180305689A1 (en) * | 2015-04-22 | 2018-10-25 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| JP2019500345A (ja) * | 2015-12-14 | 2019-01-10 | コールド スプリング ハーバー ラボラトリー | 肝臓病の処置のための組成物および方法 |
| CN106480028B (zh) * | 2016-10-12 | 2019-04-05 | 上海市第七人民医院 | TPO基因的saRNA分子及其应用 |
-
2020
- 2020-09-18 PE PE2022000448A patent/PE20220604A1/es unknown
- 2020-09-18 AU AU2020349991A patent/AU2020349991A1/en not_active Abandoned
- 2020-09-18 US US17/753,927 patent/US20230212565A1/en not_active Abandoned
- 2020-09-18 KR KR1020227008451A patent/KR20220070206A/ko not_active Withdrawn
- 2020-09-18 CA CA3148158A patent/CA3148158A1/en active Pending
- 2020-09-18 BR BR112022005028A patent/BR112022005028A2/pt not_active Application Discontinuation
- 2020-09-18 JP JP2022517729A patent/JP2022552940A/ja active Pending
- 2020-09-18 WO PCT/CN2020/116227 patent/WO2021052470A1/zh not_active Ceased
- 2020-09-18 CN CN202080065027.2A patent/CN114585738A/zh active Pending
- 2020-09-18 EP EP20866111.6A patent/EP4036232A4/en not_active Withdrawn
- 2020-09-21 TW TW109132616A patent/TW202126808A/zh unknown
-
2022
- 2022-04-18 CO CONC2022/0004857A patent/CO2022004857A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20220070206A (ko) | 2022-05-30 |
| AU2020349991A1 (en) | 2022-04-07 |
| WO2021052470A1 (zh) | 2021-03-25 |
| EP4036232A1 (en) | 2022-08-03 |
| TW202126808A (zh) | 2021-07-16 |
| CN114585738A (zh) | 2022-06-03 |
| EP4036232A4 (en) | 2023-01-11 |
| CO2022004857A2 (es) | 2022-07-08 |
| CA3148158A1 (en) | 2021-03-25 |
| PE20220604A1 (es) | 2022-04-25 |
| US20230212565A1 (en) | 2023-07-06 |
| JP2022552940A (ja) | 2022-12-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B11A | Dismissal acc. art.33 of ipl - examination not requested within 36 months of filing | ||
| B11Y | Definitive dismissal - extension of time limit for request of examination expired [chapter 11.1.1 patent gazette] |