PL3233129T3 - Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeń - Google Patents
Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeńInfo
- Publication number
- PL3233129T3 PL3233129T3 PL15817239T PL15817239T PL3233129T3 PL 3233129 T3 PL3233129 T3 PL 3233129T3 PL 15817239 T PL15817239 T PL 15817239T PL 15817239 T PL15817239 T PL 15817239T PL 3233129 T3 PL3233129 T3 PL 3233129T3
- Authority
- PL
- Poland
- Prior art keywords
- wilson
- disease
- treatment
- conditions
- nucleic acid
- Prior art date
Links
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
- 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
- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
-
- 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)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
- C12Y306/03004—Cu2+-exporting ATPase (3.6.3.4)
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Psychiatry (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)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14382530 | 2014-12-17 | ||
| EP15817239.5A EP3233129B1 (en) | 2014-12-17 | 2015-12-17 | Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions |
| PCT/EP2015/080356 WO2016097218A1 (en) | 2014-12-17 | 2015-12-17 | Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3233129T3 true PL3233129T3 (pl) | 2020-11-02 |
Family
ID=52278488
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL15817239T PL3233129T3 (pl) | 2014-12-17 | 2015-12-17 | Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeń |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US11118238B2 (pl) |
| EP (2) | EP3744351A1 (pl) |
| JP (1) | JP6956635B2 (pl) |
| DK (1) | DK3233129T3 (pl) |
| ES (1) | ES2784264T3 (pl) |
| HU (1) | HUE048551T2 (pl) |
| PL (1) | PL3233129T3 (pl) |
| PT (1) | PT3233129T (pl) |
| SI (1) | SI3233129T1 (pl) |
| WO (1) | WO2016097218A1 (pl) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11118238B2 (en) | 2014-12-17 | 2021-09-14 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions |
| 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 |
| GB201522416D0 (en) * | 2015-12-18 | 2016-02-03 | Ucl Business Plc | Wilson's disease gene therapy |
| BR112019013245A2 (pt) | 2016-12-30 | 2020-02-11 | The Trustees Of The University Of Pennsylvania | Terapia gênica para tratamento da doença de wilson |
| WO2018206125A1 (en) | 2017-05-09 | 2018-11-15 | Fundacion Para La Investigacion Medica Aplicada | Human porphobilinogen deaminase derived proteins and polynucleotides and uses thereof |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| EP3813871A4 (en) | 2018-06-29 | 2022-10-12 | Icahn School of Medicine at Mount Sinai | SPHINGOLIPID METABOLIZING PROTEINS ENCODING FOR ANC80 |
| EP3880809A4 (en) | 2018-11-16 | 2023-01-11 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR THE TREATMENT OF WILSON'S DISEASE |
| DK3906066T5 (da) | 2019-01-04 | 2024-08-05 | Ultragenyx Pharmaceutical Inc | Geneterapikonstruktioner til behandling af wilsons sygdom |
| US20220347276A1 (en) * | 2019-09-11 | 2022-11-03 | Icahn School Of Medicine At Mount Sinai | Anc80 encoding sphingolipid-metabolizing proteins for mitigating disease-induced tissue damage |
| 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 |
| AU2021375404A1 (en) | 2020-11-03 | 2023-06-08 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| JP2024505251A (ja) * | 2021-02-01 | 2024-02-05 | バイオマリン ファーマシューティカル インコーポレイテッド | 感染性が改善されたａａｖウイルス粒子のａａｖ産生システム |
| US20240254509A1 (en) | 2021-03-04 | 2024-08-01 | Research Institute At Nationwide Children's Hospital | Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications |
| US20240307553A1 (en) | 2021-04-01 | 2024-09-19 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| TW202323528A (zh) * | 2021-10-18 | 2023-06-16 | 美商邏輯生物療法公司 | 用於治療威爾森氏病之基因療法 |
| WO2024038365A1 (en) | 2022-08-16 | 2024-02-22 | Pfizer Inc. | Methods for purification of aav vectors by anion exchange chromatography |
| CN117925624B (zh) * | 2024-03-22 | 2024-05-31 | 上海凌医生物科技有限公司 | 一种金属响应调控元件 |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| AU8200191A (en) | 1990-07-09 | 1992-02-04 | United States of America, as represented by the Secretary, U.S. Department of Commerce, The | High efficiency packaging of mutant adeno-associated virus using amber suppressions |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| DE69233013T2 (de) | 1991-08-20 | 2004-03-04 | The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer | Adenovirus vermittelter gentransfer in den gastrointestinaltrakt |
| JPH10146191A (ja) | 1996-11-15 | 1998-06-02 | Sumitomo Pharmaceut Co Ltd | ウィルソン病治療用の組換えアデノウイルスベクター |
| US6200560B1 (en) * | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| EP1103610A1 (en) | 1999-11-26 | 2001-05-30 | Introgene B.V. | Production of vaccines from immortalised mammalian cell lines |
| FR2808803B1 (fr) | 2000-05-11 | 2004-12-10 | Agronomique Inst Nat Rech | Cellules es modifiees et gene specifique de cellules es |
| 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 |
| FR2836924B1 (fr) | 2002-03-08 | 2005-01-14 | Vivalis | Lignees de cellules aviaires utiles pour la production de substances d'interet |
| EP1528101A1 (en) | 2003-11-03 | 2005-05-04 | ProBioGen AG | Immortalized avian cell lines for virus production |
| FR2884255B1 (fr) | 2005-04-11 | 2010-11-05 | Vivalis | Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe |
| US8048999B2 (en) | 2005-12-13 | 2011-11-01 | Kyoto University | Nuclear reprogramming factor |
| JP5813321B2 (ja) | 2007-03-23 | 2015-11-17 | ウィスコンシン アラムニ リサーチ ファンデーション | 体細胞の再プログラム化 |
| EP1985305A1 (en) | 2007-04-24 | 2008-10-29 | Vivalis | Duck embryonic derived stem cell lines for the production of viral vaccines |
| EP1995309A1 (en) | 2007-05-21 | 2008-11-26 | Vivalis | Recombinant protein production in avian EBx® cells |
| ES2337973B8 (es) | 2008-05-16 | 2011-07-21 | Proyecto De Biomedicina Cima, S.L. | Adenovirus auxiliares auto-inactivantes para la produccion de adenovirus recombinantes de alta capacidad. |
| EP2394667A1 (en) * | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| CN104411338A (zh) * | 2012-04-02 | 2015-03-11 | 现代治疗公司 | 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 |
| ES2926774T3 (es) | 2013-03-15 | 2022-10-28 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores AAV de doble unión de glicano |
| US11118238B2 (en) | 2014-12-17 | 2021-09-14 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions |
-
2015
- 2015-12-17 US US15/537,801 patent/US11118238B2/en active Active
- 2015-12-17 DK DK15817239.5T patent/DK3233129T3/da active
- 2015-12-17 PT PT158172395T patent/PT3233129T/pt unknown
- 2015-12-17 PL PL15817239T patent/PL3233129T3/pl unknown
- 2015-12-17 WO PCT/EP2015/080356 patent/WO2016097218A1/en not_active Ceased
- 2015-12-17 JP JP2017532750A patent/JP6956635B2/ja active Active
- 2015-12-17 ES ES15817239T patent/ES2784264T3/es active Active
- 2015-12-17 SI SI201531159T patent/SI3233129T1/sl unknown
- 2015-12-17 HU HUE15817239A patent/HUE048551T2/hu unknown
- 2015-12-17 EP EP19219208.6A patent/EP3744351A1/en not_active Withdrawn
- 2015-12-17 EP EP15817239.5A patent/EP3233129B1/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| SI3233129T1 (sl) | 2020-07-31 |
| WO2016097218A1 (en) | 2016-06-23 |
| EP3233129B1 (en) | 2020-01-15 |
| EP3744351A1 (en) | 2020-12-02 |
| DK3233129T3 (da) | 2020-04-06 |
| EP3233129A1 (en) | 2017-10-25 |
| JP6956635B2 (ja) | 2021-11-02 |
| PT3233129T (pt) | 2020-04-15 |
| US11118238B2 (en) | 2021-09-14 |
| HUE048551T2 (hu) | 2020-08-28 |
| JP2018505663A (ja) | 2018-03-01 |
| US20170356060A1 (en) | 2017-12-14 |
| ES2784264T3 (es) | 2020-09-23 |
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