ZA202104515B - Gene therapy constructs for treating wilson disease - Google Patents
Gene therapy constructs for treating wilson diseaseInfo
- Publication number
- ZA202104515B ZA202104515B ZA2021/04515A ZA202104515A ZA202104515B ZA 202104515 B ZA202104515 B ZA 202104515B ZA 2021/04515 A ZA2021/04515 A ZA 2021/04515A ZA 202104515 A ZA202104515 A ZA 202104515A ZA 202104515 B ZA202104515 B ZA 202104515B
- Authority
- ZA
- South Africa
- Prior art keywords
- gene therapy
- wilson disease
- atp7b
- treating wilson
- therapy constructs
- Prior art date
Links
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 title abstract 3
- 208000018839 Wilson disease Diseases 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 102000012437 Copper-Transporting ATPases Human genes 0.000 title 1
- 102100027591 Copper-transporting ATPase 2 Human genes 0.000 abstract 5
- 101000936280 Homo sapiens Copper-transporting ATPase 2 Proteins 0.000 abstract 3
- 238000004519 manufacturing process Methods 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
-
- 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 (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 |
|---|---|
| ZA202104515B true ZA202104515B (en) | 2024-12-18 |
Family
ID=69528954
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ZA2021/04515A ZA202104515B (en) | 2019-01-04 | 2021-06-29 | Gene therapy constructs for treating wilson 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 |
|---|---|---|---|---|
| MX2019007873A (es) | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| JP2021519581A (ja) | 2018-04-03 | 2021-08-12 | ストライドバイオ，インコーポレイテッド | 抗体を回避するウイルスベクター |
| SG11202009452WA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| US12338450B2 (en) | 2019-01-04 | 2025-06-24 | Ultragenyx Pharmaceutical Inc. | Gene therapy constructs for treating Wilson disease |
| AU2020241888A1 (en) | 2019-03-21 | 2021-09-30 | Ginkgo Bioworks, Inc. | Recombinant adeno-associated virus vectors |
| CN114072514A (zh) * | 2019-05-14 | 2022-02-18 | 杜克大学 | 用于治疗atp酶介导的疾病的组合物和方法 |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| AR123288A1 (es) | 2020-08-19 | 2022-11-16 | Stridebio Inc | Vectores de virus adenoasociados para el tratamiento del síndrome de rett |
| 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 |
| KR20240114769A (ko) * | 2021-12-10 | 2024-07-24 | 애반티바이오, 인크. | 증진된 발현을 위해 선택된 코작 서열을 포함하는 조성물 |
| US20250041453A1 (en) * | 2021-12-10 | 2025-02-06 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector |
| 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 |
| WO2025026198A1 (en) * | 2023-07-28 | 2025-02-06 | Lingyi Biotech Co., Ltd. | Polynucleotides for the treatment of wilson disease |
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 |
| US7985553B2 (en) * | 2001-10-29 | 2011-07-26 | Nathaniel Heintz | Method for isolating cell type-specific mRNAs |
| SG168422A1 (en) | 2001-11-13 | 2011-02-28 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| 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 |
| JP4559429B2 (ja) | 2003-05-21 | 2010-10-06 | ジェンザイム・コーポレーション | 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法 |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| EP3910063A1 (en) | 2003-09-30 | 2021-11-17 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| EP3409296A1 (en) | 2005-04-07 | 2018-12-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
| AU2009209408B2 (en) | 2008-01-29 | 2015-06-11 | Beacon Therapeutics Limited | Recombinant virus production using mammalian cells in suspension |
| WO2010114948A2 (en) | 2009-04-02 | 2010-10-07 | University Of Florida Research Foundation, Inc. | An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| WO2011094198A1 (en) | 2010-01-28 | 2011-08-04 | The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | 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 |
| CN104520428B (zh) * | 2012-02-17 | 2018-09-21 | 费城儿童医院 | 将基因转移到细胞、器官和组织的aav载体组合物和方法 |
| AU2013243952A1 (en) | 2012-04-02 | 2014-10-30 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal 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 |
| LT3137497T (lt) * | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| EP3800260A1 (en) | 2014-09-24 | 2021-04-07 | City of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| JP6865167B2 (ja) | 2014-12-17 | 2021-04-28 | フンダシオン パラ ラ インベスティガシオン メディカ アプリカダ | ウィルソン病の処置に使用するための核酸構築物及び遺伝子治療ベクター |
| PT3233129T (pt) | 2014-12-17 | 2020-04-15 | Fundacion Para La Investig Medica Aplicada | Construções de ácido nucleico e vetores de terapia génica para utilização no tratamento de doença de wilson e outras condições |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| MX2019007873A (es) * | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| CN113302290A (zh) * | 2018-11-16 | 2021-08-24 | 编码治疗公司 | 治疗威尔逊氏病的组合物和方法 |
| US12338450B2 (en) | 2019-01-04 | 2025-06-24 | 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 US US17/417,619 patent/US12338450B2/en active Active
- 2020-01-03 MX MX2021008135A patent/MX2021008135A/es unknown
- 2020-01-03 JP JP2021538754A patent/JP7531495B2/ja active Active
- 2020-01-03 CA CA3124880A patent/CA3124880A1/en active Pending
- 2020-01-03 KR KR1020217024152A patent/KR20210112339A/ko active Pending
- 2020-01-03 FI FIEP20704633.5T patent/FI3906066T3/fi active
- 2020-01-03 CN CN202080007863.5A patent/CN113518628B/zh active Active
- 2020-01-03 PL PL20704633.5T patent/PL3906066T3/pl unknown
- 2020-01-03 PT PT207046335T patent/PT3906066T/pt unknown
- 2020-01-03 HU HUE20704633A patent/HUE065782T2/hu unknown
- 2020-01-03 HR HRP20231750TT patent/HRP20231750T1/hr unknown
- 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 TW TW109100147A patent/TWI848038B/zh active
- 2020-01-03 BR BR112021013140A patent/BR112021013140A2/pt unknown
- 2020-01-03 SG SG11202106855YA patent/SG11202106855YA/en unknown
- 2020-01-03 EP EP20704633.5A patent/EP3906066B1/en active Active
- 2020-01-03 AU AU2020204679A patent/AU2020204679A1/en active Pending
- 2020-01-03 ES ES20704633T patent/ES2970216T3/es active Active
- 2020-01-03 EP EP23196931.2A patent/EP4269579A3/en 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 |
|---|---|
| US20220090131A1 (en) | 2022-03-24 |
| CO2021010088A2 (es) | 2021-09-20 |
| TWI848038B (zh) | 2024-07-11 |
| DK3906066T5 (da) | 2024-08-05 |
| EP4269579A2 (en) | 2023-11-01 |
| DK3906066T3 (da) | 2024-02-05 |
| FI3906066T3 (fi) | 2024-01-30 |
| HUE065782T2 (hu) | 2024-06-28 |
| MX2021008135A (es) | 2021-08-11 |
| CN113518628B (zh) | 2025-04-29 |
| JP7531495B2 (ja) | 2024-08-09 |
| EP3906066B1 (en) | 2023-11-01 |
| EP4269579A3 (en) | 2024-01-24 |
| PL3906066T3 (pl) | 2024-09-02 |
| CA3124880A1 (en) | 2020-07-09 |
| TW202043479A (zh) | 2020-12-01 |
| SG11202106855YA (en) | 2021-07-29 |
| WO2020142653A1 (en) | 2020-07-09 |
| EP3906066A1 (en) | 2021-11-10 |
| HRP20231750T1 (hr) | 2024-05-24 |
| US20250333763A1 (en) | 2025-10-30 |
| CN113518628A (zh) | 2021-10-19 |
| JP2024054426A (ja) | 2024-04-16 |
| JP2022516283A (ja) | 2022-02-25 |
| IL284554A (en) | 2021-08-31 |
| PT3906066T (pt) | 2024-02-06 |
| ES2970216T3 (es) | 2024-05-27 |
| AU2020204679A1 (en) | 2021-07-15 |
| KR20210112339A (ko) | 2021-09-14 |
| JP7746437B2 (ja) | 2025-09-30 |
| US12338450B2 (en) | 2025-06-24 |
| BR112021013140A2 (pt) | 2021-11-30 |
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