IL284554A - Structures for gene therapy to treat Wilson's disease - Google Patents
Structures for gene therapy to treat Wilson's diseaseInfo
- Publication number
- IL284554A IL284554A IL284554A IL28455421A IL284554A IL 284554 A IL284554 A IL 284554A IL 284554 A IL284554 A IL 284554A IL 28455421 A IL28455421 A IL 28455421A IL 284554 A IL284554 A IL 284554A
- Authority
- IL
- Israel
- Prior art keywords
- gene therapy
- wilson disease
- therapy constructs
- treating wilson
- treating
- Prior art date
Links
- 102000012437 Copper-Transporting ATPases Human genes 0.000 title 1
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 title 1
- 208000018839 Wilson disease Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
Classifications
-
- 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
-
- 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
- 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962788324P | 2019-01-04 | 2019-01-04 | |
| US201962834830P | 2019-04-16 | 2019-04-16 | |
| PCT/US2020/012131 WO2020142653A1 (en) | 2019-01-04 | 2020-01-03 | Gene therapy constructs for treating wilson disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL284554A true IL284554A (en) | 2021-08-31 |
Family
ID=69528954
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL284554A IL284554A (en) | 2019-01-04 | 2021-07-01 | Structures for gene therapy to treat Wilson's disease |
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 |
Family Cites Families (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US8679837B2 (en) | 2009-04-02 | 2014-03-25 | University Of Florida Research Foundation, Inc. | Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| CN102947453A (zh) | 2010-01-28 | 2013-02-27 | 费城儿童医院 | 用于病毒载体纯化的可扩缩生产平台和用于基因治疗中的如此纯化的病毒载体 |
| 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 |
| CA3048044A1 (en) | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating wilson's disease |
| 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 |
-
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
-
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
-
2024
- 2024-03-01 JP JP2024031460A patent/JP7746437B2/ja active Active
-
2025
- 2025-05-20 US US19/213,623 patent/US20250333763A1/en active Pending
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 |
| TWI848038B (zh) | 2024-07-11 |
| 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 |
| 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 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| IL284554A (en) | Structures for gene therapy to treat Wilson's disease | |
| IL277190A (en) | Methods of treating HPV-related diseases | |
| SG11202110789VA (en) | Gene therapy for eye pathologies | |
| SG11202100022SA (en) | Gene therapy vectors for treatment of danon disease | |
| GB202003618D0 (en) | Gene Therapy | |
| SG11202107744SA (en) | Gene therapy vectors for treatment of danon disease | |
| EP3796891A4 (en) | THERAPEUTIC CONSTRUCTS FOR TREATMENT OF CANCER | |
| IL289667A (en) | Cardiac aav gene therapy for cardiomyopathy in humans | |
| GB201905301D0 (en) | Gene therapy | |
| IL261292A (en) | Gene therapy for the treatment of retinal degeneration | |
| GB202114972D0 (en) | Gene therapy | |
| IL279685A (en) | Gene therapy | |
| GB201522416D0 (en) | Wilson's disease gene therapy | |
| IL276701A (en) | Oligonucleotide therapy for Wilson's disease | |
| IL290770A (en) | Compounds and methods for treating oxalate-related diseases | |
| GB201802326D0 (en) | Gene therapy | |
| GB201817470D0 (en) | Gene therapy | |
| IL306119A (en) | Gene therapy for the treatment of beta-hemoglobinopathies | |
| GB201817469D0 (en) | Gene therapy for retinal disease | |
| IL286099A (en) | Methods of treating the disease using levoketoconazole | |
| GB202010894D0 (en) | Gene therapy | |
| GB202003109D0 (en) | Gene therapy | |
| GB202002202D0 (en) | Gene therapy | |
| GB201911728D0 (en) | Bacterial composistions for the treatment of disease | |
| GB202104611D0 (en) | Gene therapy for retinal disease |