TWI848038B - 用於治療威爾森氏症之基因療法構築體 - Google Patents
用於治療威爾森氏症之基因療法構築體 Download PDFInfo
- Publication number
- TWI848038B TWI848038B TW109100147A TW109100147A TWI848038B TW I848038 B TWI848038 B TW I848038B TW 109100147 A TW109100147 A TW 109100147A TW 109100147 A TW109100147 A TW 109100147A TW I848038 B TWI848038 B TW I848038B
- Authority
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- Prior art keywords
- sequence
- raav
- atp7b
- aav
- seq
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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
- 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
-
- 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
- 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
- C12N15/63—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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
-
- 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
-
- 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)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962788324P | 2019-01-04 | 2019-01-04 | |
| US62/788,324 | 2019-01-04 | ||
| US201962834830P | 2019-04-16 | 2019-04-16 | |
| US62/834,830 | 2019-04-16 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| TW202043479A TW202043479A (zh) | 2020-12-01 |
| TWI848038B true TWI848038B (zh) | 2024-07-11 |
Family
ID=69528954
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW109100147A TWI848038B (zh) | 2019-01-04 | 2020-01-03 | 用於治療威爾森氏症之基因療法構築體 |
Country Status (22)
| Country | Link |
|---|---|
| US (2) | US12338450B2 (enExample) |
| EP (2) | EP3906066B1 (enExample) |
| JP (2) | JP7531495B2 (enExample) |
| KR (1) | KR20210112339A (enExample) |
| CN (1) | CN113518628B (enExample) |
| AU (1) | AU2020204679A1 (enExample) |
| BR (1) | BR112021013140A2 (enExample) |
| CA (1) | CA3124880A1 (enExample) |
| CO (1) | CO2021010088A2 (enExample) |
| DK (1) | DK3906066T5 (enExample) |
| ES (1) | ES2970216T3 (enExample) |
| FI (1) | FI3906066T3 (enExample) |
| HR (1) | HRP20231750T1 (enExample) |
| HU (1) | HUE065782T2 (enExample) |
| IL (1) | IL284554A (enExample) |
| MX (1) | MX2021008135A (enExample) |
| PL (1) | PL3906066T3 (enExample) |
| PT (1) | PT3906066T (enExample) |
| SG (1) | SG11202106855YA (enExample) |
| TW (1) | TWI848038B (enExample) |
| WO (1) | WO2020142653A1 (enExample) |
| ZA (1) | ZA202104515B (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3048044A1 (en) | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating wilson's disease |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| EA202092362A1 (ru) | 2018-04-03 | 2021-01-11 | Страйдбайо, Инк. | Вирусные векторы для нацеливания на ткани глаза |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| SG11202106855YA (en) | 2019-01-04 | 2021-07-29 | Ultragenyx Pharmaceutical Inc | Gene therapy constructs for treating wilson disease |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| WO2020232297A1 (en) * | 2019-05-14 | 2020-11-19 | Duke University | Compositions and methods for the treatment of atpase-mediated diseases |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| US12104163B2 (en) | 2020-08-19 | 2024-10-01 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| CN115216481A (zh) * | 2021-04-21 | 2022-10-21 | 四川至善唯新生物科技有限公司 | 一种表达量提高的截短型atp7b基因及其应用 |
| CN113694218A (zh) * | 2021-08-30 | 2021-11-26 | 昆明理工大学 | 一种atp7b基因p992l突变的基因修复治疗载体 |
| IL313304A (en) * | 2021-12-10 | 2024-08-01 | Aavantibio Inc | Compositions comprising KOZAK sequences selected for enhanced expression |
| JP2024545183A (ja) * | 2021-12-10 | 2024-12-05 | ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． | ウイルスベクターによりｂａｇ－３関連心筋症を処置するための方法および組成物 |
| WO2024183769A1 (en) * | 2023-03-06 | 2024-09-12 | Lingyi Biotech Co., Ltd. | A truncated atp7b and the use thereof |
| WO2024193638A1 (en) * | 2023-03-22 | 2024-09-26 | Skyline Therapeutics (Shanghai) Co., Ltd. | Recombinant aav for the gene therapy of wilson disease |
| WO2025002226A1 (zh) * | 2023-06-28 | 2025-01-02 | 迈威(上海)生物科技股份有限公司 | 编码atp7b的核酸、腺相关病毒载体及其在制备防治肝豆状核变性的药物中的应用 |
| CN120882865A (zh) * | 2023-07-28 | 2025-10-31 | 凌意(杭州)生物科技有限公司 | 用于治疗威尔逊病的多核苷酸 |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018126116A1 (en) * | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating wilson's disease |
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| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| AU2002350039A1 (en) * | 2001-10-29 | 2003-05-12 | Renovis, Inc. | Method for isolating cell-type specific mrnas |
| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| DE10156121A1 (de) | 2001-11-16 | 2003-05-28 | Wolfgang Stremmel | Somatische Gentherapie mit Vektoren, die einen verkürzten metallsensitiven Promotor besitzen, zur Behandlung des Morbus Wilson sowie anderer Erkrankungen, die mit Veränderungen im Metallstoffwechsel einhergehen, wie z.B. Alzheimer-Erkrankung |
| JP4810062B2 (ja) | 2001-12-17 | 2011-11-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）血清型８の配列 |
| ES2521682T3 (es) | 2003-05-21 | 2014-11-13 | Genzyme Corporation | Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías |
| US20080050357A1 (en) | 2003-08-01 | 2008-02-28 | Claes Gustafsson | Systems and Methods for Antibody Engineering |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| EP2383346B1 (en) | 2005-04-07 | 2014-10-08 | The Trustees of the University of Pennsylvania | Modified AAVrh.64 capsids, compositions containing same and uses thereof |
| AU2009209408B2 (en) | 2008-01-29 | 2015-06-11 | Beacon Therapeutics Limited | Recombinant virus production using mammalian cells in suspension |
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| EP2553106A2 (en) | 2010-03-29 | 2013-02-06 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| BR112014020325A2 (pt) * | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| WO2013151663A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| SG11201702073XA (en) | 2014-09-24 | 2017-04-27 | Hope City | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3744351A1 (en) | 2014-12-17 | 2020-12-02 | Fundacion para la Investigacion Medica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions |
| SG11201704690TA (en) * | 2014-12-17 | 2017-07-28 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| JP7532360B2 (ja) | 2018-11-16 | 2024-08-13 | エンコーデッド セラピューティクス， インコーポレイテッド | ウィルソン病を処置するための組成物および方法 |
| SG11202106855YA (en) | 2019-01-04 | 2021-07-29 | Ultragenyx Pharmaceutical Inc | Gene therapy constructs for treating wilson disease |
| US20250320509A1 (en) * | 2022-05-23 | 2025-10-16 | The Johns Hopkins University | Compositions and methods for treating wilson's disease |
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2020
- 2020-01-03 SG SG11202106855YA patent/SG11202106855YA/en unknown
- 2020-01-03 FI FIEP20704633.5T patent/FI3906066T3/fi active
- 2020-01-03 ES ES20704633T patent/ES2970216T3/es active Active
- 2020-01-03 CN CN202080007863.5A patent/CN113518628B/zh active Active
- 2020-01-03 HR HRP20231750TT patent/HRP20231750T1/hr unknown
- 2020-01-03 MX MX2021008135A patent/MX2021008135A/es unknown
- 2020-01-03 AU AU2020204679A patent/AU2020204679A1/en active Pending
- 2020-01-03 CA CA3124880A patent/CA3124880A1/en active Pending
- 2020-01-03 HU HUE20704633A patent/HUE065782T2/hu unknown
- 2020-01-03 EP EP20704633.5A patent/EP3906066B1/en active Active
- 2020-01-03 US US17/417,619 patent/US12338450B2/en active Active
- 2020-01-03 JP JP2021538754A patent/JP7531495B2/ja active Active
- 2020-01-03 EP EP23196931.2A patent/EP4269579A3/en active Pending
- 2020-01-03 BR BR112021013140A patent/BR112021013140A2/pt unknown
- 2020-01-03 PT PT207046335T patent/PT3906066T/pt unknown
- 2020-01-03 PL PL20704633.5T patent/PL3906066T3/pl unknown
- 2020-01-03 TW TW109100147A patent/TWI848038B/zh active
- 2020-01-03 WO PCT/US2020/012131 patent/WO2020142653A1/en not_active Ceased
- 2020-01-03 DK DK20704633.5T patent/DK3906066T5/da active
- 2020-01-03 KR KR1020217024152A patent/KR20210112339A/ko active Pending
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2021
- 2021-06-29 ZA ZA2021/04515A patent/ZA202104515B/en unknown
- 2021-07-01 IL IL284554A patent/IL284554A/en unknown
- 2021-07-29 CO CONC2021/0010088A patent/CO2021010088A2/es unknown
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2024
- 2024-03-01 JP JP2024031460A patent/JP7746437B2/ja active Active
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2025
- 2025-05-20 US US19/213,623 patent/US20250333763A1/en active Pending
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2018126116A1 (en) * | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating wilson's disease |
Non-Patent Citations (1)
| Title |
|---|
| 期刊 Hasan, Nesrin M et al. Molecular events initiating exit of a copper-transporting ATPase ATP7B from the trans-Golgi network. The Journal of biological chemistry vol. 287 The American Society for Biochemistry and Molecular Biology 2012 36041-50 * |
Also Published As
| Publication number | Publication date |
|---|---|
| ES2970216T3 (es) | 2024-05-27 |
| DK3906066T5 (da) | 2024-08-05 |
| US12338450B2 (en) | 2025-06-24 |
| ZA202104515B (en) | 2024-12-18 |
| KR20210112339A (ko) | 2021-09-14 |
| EP4269579A2 (en) | 2023-11-01 |
| WO2020142653A1 (en) | 2020-07-09 |
| EP3906066A1 (en) | 2021-11-10 |
| DK3906066T3 (da) | 2024-02-05 |
| PT3906066T (pt) | 2024-02-06 |
| SG11202106855YA (en) | 2021-07-29 |
| EP3906066B1 (en) | 2023-11-01 |
| US20220090131A1 (en) | 2022-03-24 |
| AU2020204679A1 (en) | 2021-07-15 |
| FI3906066T3 (fi) | 2024-01-30 |
| MX2021008135A (es) | 2021-08-11 |
| CA3124880A1 (en) | 2020-07-09 |
| HUE065782T2 (hu) | 2024-06-28 |
| JP2022516283A (ja) | 2022-02-25 |
| CN113518628A (zh) | 2021-10-19 |
| IL284554A (en) | 2021-08-31 |
| HRP20231750T1 (hr) | 2024-05-24 |
| EP4269579A3 (en) | 2024-01-24 |
| JP7531495B2 (ja) | 2024-08-09 |
| US20250333763A1 (en) | 2025-10-30 |
| CO2021010088A2 (es) | 2021-09-20 |
| JP2024054426A (ja) | 2024-04-16 |
| BR112021013140A2 (pt) | 2021-11-30 |
| PL3906066T3 (pl) | 2024-09-02 |
| CN113518628B (zh) | 2025-04-29 |
| TW202043479A (zh) | 2020-12-01 |
| JP7746437B2 (ja) | 2025-09-30 |
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