CL2021000747A1 - Métodos y composiciones para aumentar la expresión de proteína y / o tratar un trastorno de haploinsuficiencia - Google Patents
Métodos y composiciones para aumentar la expresión de proteína y / o tratar un trastorno de haploinsuficienciaInfo
- Publication number
- CL2021000747A1 CL2021000747A1 CL2021000747A CL2021000747A CL2021000747A1 CL 2021000747 A1 CL2021000747 A1 CL 2021000747A1 CL 2021000747 A CL2021000747 A CL 2021000747A CL 2021000747 A CL2021000747 A CL 2021000747A CL 2021000747 A1 CL2021000747 A1 CL 2021000747A1
- Authority
- CL
- Chile
- Prior art keywords
- compositions
- treating
- methods
- protein expression
- increasing protein
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 4
- 239000000203 mixture Substances 0.000 title 1
- 102000004169 proteins and genes Human genes 0.000 title 1
- 108020004705 Codon Proteins 0.000 abstract 4
- 210000004962 mammalian cell Anatomy 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/67—General methods for enhancing the expression
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
-
- 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
-
- 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
- C12N15/63—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
- C12N15/86—Viral vectors
-
- 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
- C12N2320/34—Allele or polymorphism specific uses
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Virology (AREA)
- Pain & Pain Management (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862736847P | 2018-09-26 | 2018-09-26 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2021000747A1 true CL2021000747A1 (es) | 2021-10-22 |
Family
ID=69949913
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2021000747A CL2021000747A1 (es) | 2018-09-26 | 2021-03-25 | Métodos y composiciones para aumentar la expresión de proteína y / o tratar un trastorno de haploinsuficiencia |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US12281314B2 (enExample) |
| EP (1) | EP3856905A4 (enExample) |
| JP (2) | JP2022502044A (enExample) |
| KR (1) | KR102814622B1 (enExample) |
| CN (1) | CN113195719A (enExample) |
| AU (1) | AU2019350871B2 (enExample) |
| BR (1) | BR112021005788A2 (enExample) |
| CA (1) | CA3114109A1 (enExample) |
| CL (1) | CL2021000747A1 (enExample) |
| CO (1) | CO2021005040A2 (enExample) |
| EA (1) | EA202190736A1 (enExample) |
| IL (1) | IL281848A (enExample) |
| MX (1) | MX2021003567A (enExample) |
| WO (1) | WO2020069199A1 (enExample) |
| ZA (1) | ZA202102302B (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7712270B2 (ja) | 2019-11-01 | 2025-07-23 | テバード バイオサイエンシズ インコーポレイテッド | 未成熟終止コドン媒介障害を処置するための方法および組成物 |
| CA3160097A1 (en) * | 2019-11-04 | 2021-05-14 | Flagship Pioneering, Inc. | Trem compositions for con-rare codons and related uses |
| US20230203510A1 (en) * | 2020-05-29 | 2023-06-29 | Flagship Pioneering Innovations Vi, Llc | Trem compositions and methods relating thereto |
| EP4158032A2 (en) * | 2020-05-29 | 2023-04-05 | Flagship Pioneering Innovations VI, LLC | Trem compositions and methods relating thereto |
| EP4334450A4 (en) * | 2021-05-05 | 2025-06-18 | Tevard Biosciences, Inc. | Methods and compositions for treating a premature termination codon-mediated disorder |
| CA3249261A1 (en) | 2022-04-18 | 2023-10-26 | Vertex Pharmaceuticals Incorporated | COMPOSITIONS AND PROCESSES TO IMPROVE ADENE-ASSOCIATED VIRUS (AAV) THERAPY AND REDUCE AAV TROPISM TO THE LIVER |
| CN117257958B (zh) * | 2023-11-21 | 2024-02-09 | 四川大学华西医院 | Trps1抑制剂的新用途及用于治疗和/或预防雄激素性秃发的药物 |
| WO2025176166A1 (en) * | 2024-02-21 | 2025-08-28 | Zhuhai Codone Biotechnology Co., Ltd. | Engineered trna expression cassette |
| WO2026001270A1 (en) * | 2024-06-24 | 2026-01-02 | Zhuhai Codone Biotechnology Co., Ltd. | Engineered trna molecules and methods |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1114149A1 (en) | 1998-09-17 | 2001-07-11 | SmithKline Beecham Corporation | Rnase p polypeptides |
| US20040142325A1 (en) | 2001-09-14 | 2004-07-22 | Liat Mintz | Methods and systems for annotating biomolecular sequences |
| JP4406731B2 (ja) * | 2005-03-31 | 2010-02-03 | 国立大学法人 岡山大学 | 部位特異的アミノ酸導入法のための新規な直交化ｔＲＮＡ |
| WO2008127900A1 (en) * | 2007-04-13 | 2008-10-23 | The Salk Institute For Biological Studies | Methods of genetically encoding unnatural amino acids in eukaryotic cells using orthogonal trna/synthetase pairs |
| US20120283116A1 (en) * | 2009-09-11 | 2012-11-08 | Leppert Mark F | Mutant Sodium Channel Nav1.7 and Methods Related Thereto |
| US9676810B2 (en) * | 2010-07-08 | 2017-06-13 | The Brigham And Women's Hospital, Inc. | Neuroprotective molecules and methods of treating neurological disorders and inducing stress granules |
| US20130129668A1 (en) * | 2011-09-01 | 2013-05-23 | The Regents Of The University Of California | Diagnosis and treatment of arthritis using epigenetics |
| WO2014052855A1 (en) | 2012-09-27 | 2014-04-03 | Population Diagnostics, Inc. | Methods and compositions for screening and treating developmental disorders |
| EP3052624A1 (en) | 2013-10-02 | 2016-08-10 | Wageningen Universiteit | Systematic optimization of coding sequence for functional protein expression |
| EP3362087A1 (en) | 2015-10-15 | 2018-08-22 | The Scripps Research Institute | BINDING ACTIVITY OF AMINOACYL-tRNA SYNTHETASE IN CHARCOT-MARIE-TOOTH (CMT) NEUROPATHY AND CMT-RELATED NEUROLOGICAL DISEASES |
| CN116059236A (zh) * | 2015-12-14 | 2023-05-05 | 冷泉港实验室 | 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体 |
| WO2017193051A1 (en) | 2016-05-06 | 2017-11-09 | The Trustees Of Princeton University | Methods of monitoring rnase l activity |
| PL3458074T3 (pl) * | 2016-05-16 | 2024-11-12 | Board Of Regents Of The University Of Texas System | KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA |
| WO2018104385A1 (en) | 2016-12-07 | 2018-06-14 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Codon optimization |
| JP2022534988A (ja) | 2019-05-31 | 2022-08-04 | フラッグシップ パイオニアリング， インコーポレイテッド | ｔＲＮＡプールを調節するためのＴＲＥＭの使用 |
-
2019
- 2019-09-26 EA EA202190736A patent/EA202190736A1/ru unknown
- 2019-09-26 JP JP2021517022A patent/JP2022502044A/ja active Pending
- 2019-09-26 EP EP19866182.9A patent/EP3856905A4/en active Pending
- 2019-09-26 WO PCT/US2019/053268 patent/WO2020069199A1/en not_active Ceased
- 2019-09-26 US US17/280,369 patent/US12281314B2/en active Active
- 2019-09-26 MX MX2021003567A patent/MX2021003567A/es unknown
- 2019-09-26 BR BR112021005788-9A patent/BR112021005788A2/pt not_active Application Discontinuation
- 2019-09-26 KR KR1020217012452A patent/KR102814622B1/ko active Active
- 2019-09-26 CN CN201980077435.7A patent/CN113195719A/zh active Pending
- 2019-09-26 CA CA3114109A patent/CA3114109A1/en active Pending
- 2019-09-26 AU AU2019350871A patent/AU2019350871B2/en active Active
-
2021
- 2021-03-25 IL IL281848A patent/IL281848A/en unknown
- 2021-03-25 CL CL2021000747A patent/CL2021000747A1/es unknown
- 2021-04-07 ZA ZA2021/02302A patent/ZA202102302B/en unknown
- 2021-04-20 CO CONC2021/0005040A patent/CO2021005040A2/es unknown
-
2024
- 2024-09-30 JP JP2024170988A patent/JP2025010148A/ja active Pending
-
2025
- 2025-04-22 US US19/185,430 patent/US20260009041A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20220073933A1 (en) | 2022-03-10 |
| KR20210091699A (ko) | 2021-07-22 |
| JP2022502044A (ja) | 2022-01-11 |
| ZA202102302B (en) | 2022-04-28 |
| KR102814622B1 (ko) | 2025-05-29 |
| IL281848A (en) | 2021-05-31 |
| AU2019350871A1 (en) | 2021-05-20 |
| EP3856905A4 (en) | 2023-06-21 |
| CO2021005040A2 (es) | 2021-09-20 |
| CA3114109A1 (en) | 2020-04-02 |
| AU2019350871B2 (en) | 2026-02-26 |
| BR112021005788A2 (pt) | 2021-10-26 |
| JP2025010148A (ja) | 2025-01-20 |
| US12281314B2 (en) | 2025-04-22 |
| CN113195719A (zh) | 2021-07-30 |
| WO2020069199A1 (en) | 2020-04-02 |
| EA202190736A1 (ru) | 2021-09-16 |
| US20260009041A1 (en) | 2026-01-08 |
| EP3856905A1 (en) | 2021-08-04 |
| MX2021003567A (es) | 2021-08-24 |
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