DK3365438T3 - Codon-optimeret reduceret-størrelse atp7a cdna og anvendelser ved behandling af kobbertransportlidelser - Google Patents
Codon-optimeret reduceret-størrelse atp7a cdna og anvendelser ved behandling af kobbertransportlidelser Download PDFInfo
- Publication number
- DK3365438T3 DK3365438T3 DK16794111.1T DK16794111T DK3365438T3 DK 3365438 T3 DK3365438 T3 DK 3365438T3 DK 16794111 T DK16794111 T DK 16794111T DK 3365438 T3 DK3365438 T3 DK 3365438T3
- Authority
- DK
- Denmark
- Prior art keywords
- codon
- applications
- treatment
- reduced size
- copper transport
- Prior art date
Links
Classifications
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- 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)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K33/00—Medicinal preparations containing inorganic active ingredients
- A61K33/24—Heavy metals; Compounds thereof
- A61K33/34—Copper; Compounds thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
-
- 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
- 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
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Neurosurgery (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Inorganic Chemistry (AREA)
- Neurology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562244594P | 2015-10-21 | 2015-10-21 | |
| PCT/US2016/058124 WO2017070472A1 (en) | 2015-10-21 | 2016-10-21 | Codon-optimized reduced-size atp7a cdna and uses for treatment of copper transport disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3365438T3 true DK3365438T3 (da) | 2021-07-12 |
Family
ID=57256420
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK16794111.1T DK3365438T3 (da) | 2015-10-21 | 2016-10-21 | Codon-optimeret reduceret-størrelse atp7a cdna og anvendelser ved behandling af kobbertransportlidelser |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US10988778B2 (da) |
| EP (1) | EP3365438B1 (da) |
| JP (2) | JP6854286B2 (da) |
| CN (1) | CN108431216B (da) |
| AU (1) | AU2016341983B8 (da) |
| CA (1) | CA3001574A1 (da) |
| DK (1) | DK3365438T3 (da) |
| ES (1) | ES2879400T3 (da) |
| HU (1) | HUE054802T2 (da) |
| PL (1) | PL3365438T3 (da) |
| PT (1) | PT3365438T (da) |
| WO (1) | WO2017070472A1 (da) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3820462A4 (en) * | 2018-07-12 | 2022-04-27 | The Texas A&M University System | COMPOSITIONS FOR TREATMENT OF COPPER DEFICIENCY AND METHODS OF USE |
| CN117042764A (zh) * | 2021-03-18 | 2023-11-10 | 塞普鲁姆治疗公司 | 组氨酸铜组合物和其用途 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1993017321A1 (en) | 1992-02-25 | 1993-09-02 | Unisearch Limited | Electrothermal atomic absorption and preconcentration device |
| DE606516T1 (de) | 1993-01-13 | 1995-03-16 | Idemitsu Kosan Co | Gegen menschlichen Ceruloplasmin monoklonaler Antikörper. |
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| US6812339B1 (en) * | 2000-09-08 | 2004-11-02 | Applera Corporation | Polymorphisms in known genes associated with human disease, methods of detection and uses thereof |
| KR100459644B1 (ko) | 2001-03-31 | 2004-12-03 | 제노백(주) | 혈액 여과지에서의 효소면역측정법을 이용한셀룰로플라스민 농도 측정 방법 및 이 방법을 이용한윌슨병 스크리닝 검사용 키트와 진단 시약 |
| DE602004030327D1 (de) | 2003-05-21 | 2011-01-13 | Genzyme Corp | Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind |
| US20070161120A1 (en) | 2006-01-10 | 2007-07-12 | Viorica Lopez-Avila | Ceruloplasmin Assay |
| AU2007245181A1 (en) * | 2006-03-30 | 2007-11-08 | Novartis Ag | Compositions and methods of use for antibodies of c-Met |
| EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| DK2242840T3 (da) | 2008-01-29 | 2019-10-21 | Applied Genetic Tech Corporation | Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension |
| WO2010042102A1 (en) | 2008-10-06 | 2010-04-15 | The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Identification of subjects likely to benefit from copper treatment |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| 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 |
| 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 |
| CA2868391A1 (en) * | 2012-04-02 | 2013-10-10 | Stephane Bancel | Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same |
| US20140010861A1 (en) | 2012-04-02 | 2014-01-09 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
-
2016
- 2016-10-21 EP EP16794111.1A patent/EP3365438B1/en active Active
- 2016-10-21 AU AU2016341983A patent/AU2016341983B8/en active Active
- 2016-10-21 WO PCT/US2016/058124 patent/WO2017070472A1/en active Application Filing
- 2016-10-21 CN CN201680074686.6A patent/CN108431216B/zh active Active
- 2016-10-21 PT PT167941111T patent/PT3365438T/pt unknown
- 2016-10-21 US US15/769,294 patent/US10988778B2/en active Active
- 2016-10-21 JP JP2018520100A patent/JP6854286B2/ja active Active
- 2016-10-21 ES ES16794111T patent/ES2879400T3/es active Active
- 2016-10-21 CA CA3001574A patent/CA3001574A1/en active Pending
- 2016-10-21 PL PL16794111T patent/PL3365438T3/pl unknown
- 2016-10-21 DK DK16794111.1T patent/DK3365438T3/da active
- 2016-10-21 HU HUE16794111A patent/HUE054802T2/hu unknown
-
2020
- 2020-12-28 JP JP2020218556A patent/JP2021052796A/ja active Pending
-
2021
- 2021-03-30 US US17/217,961 patent/US20210214751A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2021052796A (ja) | 2021-04-08 |
| CN108431216A (zh) | 2018-08-21 |
| EP3365438A1 (en) | 2018-08-29 |
| AU2016341983B8 (en) | 2022-10-06 |
| AU2016341983A1 (en) | 2018-04-26 |
| WO2017070472A1 (en) | 2017-04-27 |
| AU2016341983B2 (en) | 2022-05-26 |
| HUE054802T2 (hu) | 2021-09-28 |
| PL3365438T3 (pl) | 2021-10-18 |
| PT3365438T (pt) | 2021-07-12 |
| EP3365438B1 (en) | 2021-04-28 |
| JP2018533941A (ja) | 2018-11-22 |
| AU2016341983A8 (en) | 2022-10-06 |
| US20180312871A1 (en) | 2018-11-01 |
| CN108431216B (zh) | 2022-08-05 |
| US10988778B2 (en) | 2021-04-27 |
| JP6854286B2 (ja) | 2021-04-07 |
| ES2879400T3 (es) | 2021-11-22 |
| US20210214751A1 (en) | 2021-07-15 |
| CA3001574A1 (en) | 2017-04-27 |
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