JP2023537980A - メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療 - Google Patents
メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療 Download PDFInfo
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- JP2023537980A JP2023537980A JP2023509770A JP2023509770A JP2023537980A JP 2023537980 A JP2023537980 A JP 2023537980A JP 2023509770 A JP2023509770 A JP 2023509770A JP 2023509770 A JP2023509770 A JP 2023509770A JP 2023537980 A JP2023537980 A JP 2023537980A
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- nucleotide sequence
- seq
- nucleic acid
- sequence
- mecp2
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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
- 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
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- C—CHEMISTRY; METALLURGY
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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
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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
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Immunology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063064431P | 2020-08-12 | 2020-08-12 | |
| US63/064,431 | 2020-08-12 | ||
| PCT/EP2021/072365 WO2022034130A1 (en) | 2020-08-12 | 2021-08-11 | Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023537980A true JP2023537980A (ja) | 2023-09-06 |
| JP2023537980A5 JP2023537980A5 (https=) | 2024-07-25 |
Family
ID=77655525
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023509770A Pending JP2023537980A (ja) | 2020-08-12 | 2021-08-11 | メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療 |
Country Status (19)
| Country | Link |
|---|---|
| US (1) | US20230295657A1 (https=) |
| EP (1) | EP4196171A1 (https=) |
| JP (1) | JP2023537980A (https=) |
| KR (1) | KR20230044506A (https=) |
| CN (1) | CN116113441A (https=) |
| AR (1) | AR123206A1 (https=) |
| AU (1) | AU2021325717A1 (https=) |
| BR (1) | BR112023002374A2 (https=) |
| CA (1) | CA3188748A1 (https=) |
| CL (1) | CL2023000419A1 (https=) |
| CO (1) | CO2023000444A2 (https=) |
| EC (1) | ECSP23016688A (https=) |
| IL (1) | IL300294A (https=) |
| MX (1) | MX2023001701A (https=) |
| PE (1) | PE20230914A1 (https=) |
| PH (1) | PH12023550145A1 (https=) |
| TW (1) | TWI902867B (https=) |
| WO (1) | WO2022034130A1 (https=) |
| ZA (1) | ZA202300378B (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| GB202216168D0 (en) | 2022-10-31 | 2022-12-14 | UCB Biopharma SRL | Route of administration |
| WO2024163823A1 (en) * | 2023-02-02 | 2024-08-08 | Shape Therapeutics Inc. | Tissue-specific enhancers for regulating transcription |
| JP2026509301A (ja) * | 2023-03-14 | 2026-03-17 | ユーシービー バイオファルマ エスアールエル | 遺伝子治療 |
| CN121335978A (zh) * | 2023-06-16 | 2026-01-13 | 舒泰神(北京)生物制药股份有限公司 | 一种人源MeCp2启动子及其用途 |
| WO2025213097A1 (en) * | 2024-04-05 | 2025-10-09 | The Children's Hospital Of Philadelphia | Novel gene therapy constructs for stxbp1 haploinsufficiency |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2011505859A (ja) * | 2007-12-21 | 2011-03-03 | ノバルティス アーゲー | 哺乳類発現ベクター |
| JP2017535266A (ja) * | 2014-11-14 | 2017-11-30 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2017536116A (ja) * | 2014-11-21 | 2017-12-07 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 中枢神経系を標的化したaavベクター |
| JP2018500311A (ja) * | 2014-12-16 | 2018-01-11 | ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ | 若年型バッテン病のための遺伝子療法 |
| JP2019511570A (ja) * | 2016-03-02 | 2019-04-25 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 前頭側頭型認知症の治療 |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EA201492116A1 (ru) * | 2012-05-16 | 2015-05-29 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии mecp2 |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| TWI601821B (zh) * | 2016-04-15 | 2017-10-11 | 中央研究院 | 治療神經發展性疾病 |
| CN113166781A (zh) * | 2018-10-15 | 2021-07-23 | 沃雅戈治疗公司 | 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体 |
-
2021
- 2021-08-11 BR BR112023002374A patent/BR112023002374A2/pt unknown
- 2021-08-11 PH PH1/2023/550145A patent/PH12023550145A1/en unknown
- 2021-08-11 MX MX2023001701A patent/MX2023001701A/es unknown
- 2021-08-11 AU AU2021325717A patent/AU2021325717A1/en active Pending
- 2021-08-11 IL IL300294A patent/IL300294A/en unknown
- 2021-08-11 KR KR1020237007287A patent/KR20230044506A/ko active Pending
- 2021-08-11 CN CN202180056525.5A patent/CN116113441A/zh active Pending
- 2021-08-11 PE PE2023000142A patent/PE20230914A1/es unknown
- 2021-08-11 US US18/021,005 patent/US20230295657A1/en active Pending
- 2021-08-11 WO PCT/EP2021/072365 patent/WO2022034130A1/en not_active Ceased
- 2021-08-11 AR ARP210102238A patent/AR123206A1/es unknown
- 2021-08-11 CA CA3188748A patent/CA3188748A1/en active Pending
- 2021-08-11 TW TW110129603A patent/TWI902867B/zh active
- 2021-08-11 EP EP21766127.1A patent/EP4196171A1/en active Pending
- 2021-08-11 JP JP2023509770A patent/JP2023537980A/ja active Pending
-
2023
- 2023-01-09 ZA ZA2023/00378A patent/ZA202300378B/en unknown
- 2023-01-16 CO CONC2023/0000444A patent/CO2023000444A2/es unknown
- 2023-02-10 CL CL2023000419A patent/CL2023000419A1/es unknown
- 2023-03-08 EC ECSENADI202316688A patent/ECSP23016688A/es unknown
Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2011505859A (ja) * | 2007-12-21 | 2011-03-03 | ノバルティス アーゲー | 哺乳類発現ベクター |
| JP2017535266A (ja) * | 2014-11-14 | 2017-11-30 | ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. | 筋萎縮性側索硬化症(als)を治療する組成物および方法 |
| JP2017536116A (ja) * | 2014-11-21 | 2017-12-07 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 中枢神経系を標的化したaavベクター |
| JP2018500311A (ja) * | 2014-12-16 | 2018-01-11 | ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ | 若年型バッテン病のための遺伝子療法 |
| JP2019511570A (ja) * | 2016-03-02 | 2019-04-25 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 前頭側頭型認知症の治療 |
Non-Patent Citations (2)
| Title |
|---|
| HUMAN MOLECULAR GENETICS, vol. 14, no. 23, JPN7025002646, 2005, pages 3709 - 3722, ISSN: 0005767187 * |
| RNA, vol. 9, JPN6025024400, 2003, pages 607 - 617, ISSN: 0005767188 * |
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230044506A (ko) | 2023-04-04 |
| TW202221018A (zh) | 2022-06-01 |
| CL2023000419A1 (es) | 2023-07-21 |
| US20230295657A1 (en) | 2023-09-21 |
| CO2023000444A2 (es) | 2023-01-26 |
| EP4196171A1 (en) | 2023-06-21 |
| IL300294A (en) | 2023-04-01 |
| BR112023002374A2 (pt) | 2023-03-21 |
| AR123206A1 (es) | 2022-11-09 |
| TWI902867B (zh) | 2025-11-01 |
| MX2023001701A (es) | 2023-03-09 |
| CN116113441A (zh) | 2023-05-12 |
| PH12023550145A1 (en) | 2024-03-18 |
| WO2022034130A1 (en) | 2022-02-17 |
| AU2021325717A1 (en) | 2023-03-02 |
| ZA202300378B (en) | 2024-04-24 |
| CA3188748A1 (en) | 2022-02-17 |
| PE20230914A1 (es) | 2023-06-02 |
| ECSP23016688A (es) | 2023-04-28 |
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