BR112019013245A2 - Terapia gênica para tratamento da doença de wilson - Google Patents
Terapia gênica para tratamento da doença de wilson Download PDFInfo
- Publication number
- BR112019013245A2 BR112019013245A2 BR112019013245-7A BR112019013245A BR112019013245A2 BR 112019013245 A2 BR112019013245 A2 BR 112019013245A2 BR 112019013245 A BR112019013245 A BR 112019013245A BR 112019013245 A2 BR112019013245 A2 BR 112019013245A2
- Authority
- BR
- Brazil
- Prior art keywords
- raav
- atp7b
- copper
- mice
- fact
- Prior art date
Links
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
-
- 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
- A61K48/0075—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
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
-
- 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
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
-
- 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
- C12N2830/48—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)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Hematology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Environmental Sciences (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662440659P | 2016-12-30 | 2016-12-30 | |
| US62/440,659 | 2016-12-30 | ||
| US201762473656P | 2017-03-20 | 2017-03-20 | |
| US62/473,656 | 2017-03-20 | ||
| PCT/US2017/068919 WO2018126116A1 (en) | 2016-12-30 | 2017-12-29 | Gene therapy for treating wilson's disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112019013245A2 true BR112019013245A2 (pt) | 2020-02-11 |
Family
ID=62710046
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112019013245-7A BR112019013245A2 (pt) | 2016-12-30 | 2017-12-29 | Terapia gênica para tratamento da doença de wilson |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11473106B2 (enExample) |
| EP (2) | EP3562514A4 (enExample) |
| JP (1) | JP7128190B2 (enExample) |
| KR (2) | KR102863181B1 (enExample) |
| CN (1) | CN110290814A (enExample) |
| BR (1) | BR112019013245A2 (enExample) |
| CA (1) | CA3048044A1 (enExample) |
| MX (1) | MX2019007873A (enExample) |
| WO (1) | WO2018126116A1 (enExample) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN110290814A (zh) * | 2016-12-30 | 2019-09-27 | 宾夕法尼亚州立大学托管会 | 用于治疗威尔逊病的基因疗法 |
| BR112020010883A2 (pt) | 2017-12-01 | 2020-11-10 | Encoded Therapeutics, Inc. | proteínas de ligação a dna engenheiradas |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| EP3755795A4 (en) | 2018-02-19 | 2022-07-20 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof |
| WO2020072451A1 (en) * | 2018-10-01 | 2020-04-09 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
| 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 |
| JP7531495B2 (ja) * | 2019-01-04 | 2024-08-09 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | ウイルソン病を処置するための遺伝子治療構築物 |
| SG11202113048SA (en) | 2019-05-29 | 2021-12-30 | Encoded Therapeutics Inc | Compositions and methods for selective gene regulation |
| CN111088285B (zh) * | 2019-08-15 | 2021-12-28 | 北京锦篮基因科技有限公司 | 携带atp7b基因表达框及变异体的aav载体及应用 |
| JP7451705B2 (ja) * | 2019-11-26 | 2024-03-18 | コリア リサーチ インスティテュート オブ バイオサイエンス アンド バイオテクノロジー | タンパク質発現用ｍＲＮＡ構造体及びその用途 |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| BR112023015303A2 (pt) | 2021-02-01 | 2023-11-14 | Regenxbio Inc | Método para tratar doença cln2 devido a deficiência de tpp1 em um sujeito |
| WO2022208342A1 (en) * | 2021-04-01 | 2022-10-06 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| 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 |
| CN118434854A (zh) * | 2021-10-18 | 2024-08-02 | 逻辑生物治疗公司 | 用于治疗威尔森氏病的基因疗法 |
| WO2023230466A1 (en) * | 2022-05-23 | 2023-11-30 | Lutsenka Svetlana | Compositions and methods for treating wilson's disease |
| CN115216533A (zh) * | 2022-06-30 | 2022-10-21 | 湖南家辉生物技术有限公司 | 一种用于诊断威尔逊病的生物标志物、扩增引物组、检测试剂及应用 |
| CN120457199A (zh) * | 2023-03-06 | 2025-08-08 | 凌意(杭州)生物科技有限公司 | 截短的atp7b及其用途 |
| AU2024239180A1 (en) * | 2023-03-22 | 2025-11-06 | Skyline Therapeutics Limited | Recombinant aav for the gene therapy of wilson disease |
| US20240360431A1 (en) * | 2023-04-30 | 2024-10-31 | Sichuan Real&Best Biotech Co., Ltd. | Engineered alpha-galactosidase a (a-gal a) peptides and functional variants thereof and associated methods of treating fabry disease |
| 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 |
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| CN117947035A (zh) * | 2024-03-25 | 2024-04-30 | 上海凌医生物科技有限公司 | 一种增强基因表达的启动子 |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2002350039A1 (en) | 2001-10-29 | 2003-05-12 | Renovis, Inc. | Method for isolating cell-type specific mrnas |
| 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 |
| EP1453547B1 (en) * | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| ES2874298T3 (es) | 2003-09-30 | 2021-11-04 | Univ Pennsylvania | Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | 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 |
| US20150045284A1 (en) | 2011-01-21 | 2015-02-12 | Aarhus Universitet | CRYSTAL STRUCTURE OF A TYPE IB P-TYPE ATPase |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| JP6373763B2 (ja) | 2012-02-17 | 2018-08-22 | 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 | Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法 |
| AU2013243954A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of cosmetic proteins and peptides |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| PL3233129T3 (pl) | 2014-12-17 | 2020-11-02 | Fundacion Para La Investigacion Medica Aplicada | Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeń |
| US11147887B2 (en) * | 2014-12-17 | 2021-10-19 | 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 |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| CN110290814A (zh) * | 2016-12-30 | 2019-09-27 | 宾夕法尼亚州立大学托管会 | 用于治疗威尔逊病的基因疗法 |
| 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 |
| JP7531495B2 (ja) | 2019-01-04 | 2024-08-09 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | ウイルソン病を処置するための遺伝子治療構築物 |
-
2017
- 2017-12-29 CN CN201780085376.9A patent/CN110290814A/zh active Pending
- 2017-12-29 CA CA3048044A patent/CA3048044A1/en active Pending
- 2017-12-29 US US16/474,958 patent/US11473106B2/en active Active
- 2017-12-29 BR BR112019013245-7A patent/BR112019013245A2/pt unknown
- 2017-12-29 EP EP17888104.1A patent/EP3562514A4/en active Pending
- 2017-12-29 MX MX2019007873A patent/MX2019007873A/es unknown
- 2017-12-29 EP EP24161028.6A patent/EP4361277A3/en active Pending
- 2017-12-29 KR KR1020237039367A patent/KR102863181B1/ko active Active
- 2017-12-29 WO PCT/US2017/068919 patent/WO2018126116A1/en not_active Ceased
- 2017-12-29 KR KR1020197021756A patent/KR102604096B1/ko active Active
- 2017-12-29 JP JP2019535891A patent/JP7128190B2/ja active Active
-
2022
- 2022-07-13 US US17/812,322 patent/US12460228B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| US20190338310A1 (en) | 2019-11-07 |
| WO2018126116A1 (en) | 2018-07-05 |
| KR102604096B1 (ko) | 2023-11-23 |
| EP3562514A1 (en) | 2019-11-06 |
| MX2019007873A (es) | 2019-11-18 |
| JP7128190B2 (ja) | 2022-08-30 |
| EP4361277A3 (en) | 2024-07-31 |
| US12460228B2 (en) | 2025-11-04 |
| KR20230160411A (ko) | 2023-11-23 |
| CN110290814A (zh) | 2019-09-27 |
| CA3048044A1 (en) | 2018-07-05 |
| EP3562514A4 (en) | 2020-08-05 |
| US20220389455A1 (en) | 2022-12-08 |
| KR102863181B1 (ko) | 2025-09-23 |
| EP4361277A2 (en) | 2024-05-01 |
| KR20190101410A (ko) | 2019-08-30 |
| JP2020514286A (ja) | 2020-05-21 |
| US11473106B2 (en) | 2022-10-18 |
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