BR112022002905A2 - Composições e métodos para modular splicing e expressão de proteína - Google Patents
Composições e métodos para modular splicing e expressão de proteínaInfo
- Publication number
- BR112022002905A2 BR112022002905A2 BR112022002905A BR112022002905A BR112022002905A2 BR 112022002905 A2 BR112022002905 A2 BR 112022002905A2 BR 112022002905 A BR112022002905 A BR 112022002905A BR 112022002905 A BR112022002905 A BR 112022002905A BR 112022002905 A2 BR112022002905 A2 BR 112022002905A2
- Authority
- BR
- Brazil
- Prior art keywords
- protein expression
- methods
- compositions
- modulate splicing
- alternative splicing
- Prior art date
Links
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
-
- 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
- C12N15/1138—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 receptors or cell surface proteins
-
- 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
-
- 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
- C12N2310/314—Phosphoramidates
-
- 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
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
-
- 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
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962888887P | 2019-08-19 | 2019-08-19 | |
| US202063049262P | 2020-07-08 | 2020-07-08 | |
| PCT/US2020/047081 WO2021034985A1 (en) | 2019-08-19 | 2020-08-19 | Compositions and methods for modulating splicing and protein expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022002905A2 true BR112022002905A2 (pt) | 2022-07-12 |
Family
ID=74659533
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022002905A BR112022002905A2 (pt) | 2019-08-19 | 2020-08-19 | Composições e métodos para modular splicing e expressão de proteína |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20220290142A1 (https=) |
| EP (1) | EP4017979A4 (https=) |
| JP (1) | JP2022544702A (https=) |
| KR (1) | KR20220104677A (https=) |
| CN (1) | CN114746550A (https=) |
| AU (1) | AU2020334067A1 (https=) |
| BR (1) | BR112022002905A2 (https=) |
| CA (1) | CA3147970A1 (https=) |
| IL (1) | IL290595A (https=) |
| MX (1) | MX2022002198A (https=) |
| WO (1) | WO2021034985A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7652713B2 (ja) * | 2019-05-27 | 2025-03-27 | ビジョン ファーマ ピーティーワイ リミテッド | 新規の網膜色素変性処置 |
| KR20220054401A (ko) * | 2019-09-03 | 2022-05-02 | 더 리전츠 오브 더 유니버시티 오브 콜로라도, 어 바디 코퍼레이트 | 감염의 숙주 rna 바이오마커의 신속한 조기-검출 및 인간의 covid-19 코로나바이러스 감염의 조기 식별을 위한 시스템, 방법 및 조성물 |
| CN115916977A (zh) * | 2020-05-11 | 2023-04-04 | 弗洛里神经科学与心理健康研究所 | 用于治疗与syngap1中功能失去突变相关的障碍的组合物和方法 |
| CN117980479A (zh) * | 2021-06-21 | 2024-05-03 | 斯托克制药公司 | 用于治疗基于无义介导的rna衰变的病状和疾病的反义寡聚体 |
| WO2023004021A2 (en) * | 2021-07-23 | 2023-01-26 | The Children's Medical Center Corporation | Zinc finger ccch-type containing 14 (zc3h14) mutants and methods of use |
| WO2023163801A1 (en) * | 2022-02-24 | 2023-08-31 | Q-State Biosciences, Inc. | Therapeutics for syngap haploinsufficiency |
| EP4504951A2 (en) * | 2022-04-05 | 2025-02-12 | The Johns Hopkins University | Methods and materials for treating syngap1-associated neurodevelopmental disorders |
| WO2023212625A1 (en) * | 2022-04-28 | 2023-11-02 | AcuraStem Incorporated | Syf2 antisense oligonucleotides |
| US20250313832A1 (en) * | 2022-05-13 | 2025-10-09 | The Trustees Of The University Of Pennsylvania | Compositions for treating syngap-1 related neurodevelopmental disorders |
| US20250368991A1 (en) * | 2022-06-28 | 2025-12-04 | Agency For Science, Technology And Research | Oligonucleotides |
| WO2025090633A1 (en) * | 2023-10-23 | 2025-05-01 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies |
| WO2025111249A2 (en) * | 2023-11-20 | 2025-05-30 | Dana-Farber Cancer Institute, Inc. | Methods, kits and systems for determining sarcomatoid differentiation of renal cell carcinoma and methods for treating based on the same |
| GB202401412D0 (en) * | 2024-02-02 | 2024-03-20 | Harness Therapeutics Ltd | Functional nucleic acid |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009064920A2 (en) * | 2007-11-13 | 2009-05-22 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating protein expression |
| EP2364325A4 (en) * | 2008-11-07 | 2012-07-25 | Ct Hospitalier Universitaire Sainte Justine | SYNGAP1 DYSFUNCTIONS AND THEIR USE IN DIAGNOSTIC AND THERAPEUTIC APPLICATIONS FOR INTELLECTUAL SENTENCING |
| AU2015327836B2 (en) * | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| CN108603230A (zh) * | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| WO2017106377A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
| EP3390666A4 (en) * | 2015-12-14 | 2019-08-07 | Cold Spring Harbor Laboratory | COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES |
| EP3390635A4 (en) * | 2015-12-14 | 2019-05-01 | Cold Spring Harbor Laboratory | Antisense oligomers for the treatment of a tuberous sclerosis complex |
| 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 |
| JP2018538287A (ja) * | 2015-12-14 | 2018-12-27 | コールド スプリング ハーバー ラボラトリー | 多発性嚢胞腎の処置のためのアンチセンスオリゴマー |
| JP7036723B2 (ja) * | 2015-12-14 | 2022-03-15 | コールド スプリング ハーバー ラボラトリー | 眼疾患の処置のための組成物および方法 |
| EA201991309A1 (ru) * | 2016-11-28 | 2019-11-29 | Способы модуляции сплайсинга рнк | |
| KR102318434B1 (ko) * | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| HUE070436T2 (hu) * | 2017-10-23 | 2025-06-28 | Stoke Therapeutics Inc | Antiszensz oligomerek nonszensz-mediált RNS-lebomláson alapuló állapotok és betegségek kezelésére |
-
2020
- 2020-08-19 EP EP20855637.3A patent/EP4017979A4/en active Pending
- 2020-08-19 WO PCT/US2020/047081 patent/WO2021034985A1/en not_active Ceased
- 2020-08-19 JP JP2022511203A patent/JP2022544702A/ja active Pending
- 2020-08-19 CN CN202080073358.0A patent/CN114746550A/zh active Pending
- 2020-08-19 AU AU2020334067A patent/AU2020334067A1/en active Pending
- 2020-08-19 MX MX2022002198A patent/MX2022002198A/es unknown
- 2020-08-19 CA CA3147970A patent/CA3147970A1/en active Pending
- 2020-08-19 KR KR1020227008965A patent/KR20220104677A/ko active Pending
- 2020-08-19 BR BR112022002905A patent/BR112022002905A2/pt unknown
-
2022
- 2022-02-13 IL IL290595A patent/IL290595A/en unknown
- 2022-02-16 US US17/673,226 patent/US20220290142A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| KR20220104677A (ko) | 2022-07-26 |
| IL290595A (en) | 2022-04-01 |
| MX2022002198A (es) | 2022-05-24 |
| US20220290142A1 (en) | 2022-09-15 |
| JP2022544702A (ja) | 2022-10-20 |
| EP4017979A4 (en) | 2024-03-27 |
| CN114746550A (zh) | 2022-07-12 |
| AU2020334067A1 (en) | 2022-03-17 |
| CA3147970A1 (en) | 2021-02-25 |
| EP4017979A1 (en) | 2022-06-29 |
| WO2021034985A1 (en) | 2021-02-25 |
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