ES2879400T3 - ADNc de ATP7A de tamaño reducido con codones optimizados y usos para el tratamiento de trastornos del transporte de cobre - Google Patents

ADNc de ATP7A de tamaño reducido con codones optimizados y usos para el tratamiento de trastornos del transporte de cobre Download PDF

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Publication number
ES2879400T3
ES2879400T3 ES16794111T ES16794111T ES2879400T3 ES 2879400 T3 ES2879400 T3 ES 2879400T3 ES 16794111 T ES16794111 T ES 16794111T ES 16794111 T ES16794111 T ES 16794111T ES 2879400 T3 ES2879400 T3 ES 2879400T3
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Spain
Prior art keywords
copper
atp7a
vector
nucleic acid
composition
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Active
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ES16794111T
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English (en)
Spanish (es)
Inventor
Stephen Kaler
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US Department of Health and Human Services
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US Department of Health and Human Services
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Publication of ES2879400T3 publication Critical patent/ES2879400T3/es
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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
    • 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

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  • 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)
ES16794111T 2015-10-21 2016-10-21 ADNc de ATP7A de tamaño reducido con codones optimizados y usos para el tratamiento de trastornos del transporte de cobre Active ES2879400T3 (es)

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
ES2879400T3 true ES2879400T3 (es) 2021-11-22

Family

ID=57256420

Family Applications (1)

Application Number Title Priority Date Filing Date
ES16794111T Active ES2879400T3 (es) 2015-10-21 2016-10-21 ADNc de ATP7A de tamaño reducido con codones optimizados y usos para el tratamiento de trastornos del transporte de cobre

Country Status (11)

Country Link
US (3) US10988778B2 (https=)
EP (1) EP3365438B1 (https=)
JP (2) JP6854286B2 (https=)
CN (1) CN108431216B (https=)
AU (1) AU2016341983B8 (https=)
DK (1) DK3365438T3 (https=)
ES (1) ES2879400T3 (https=)
HU (1) HUE054802T2 (https=)
PL (1) PL3365438T3 (https=)
PT (1) PT3365438T (https=)
WO (1) WO2017070472A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210290571A1 (en) * 2018-07-12 2021-09-23 The Texas A&M University System Compositions for the treatment of copper deficiency and methods of use
US20240139236A1 (en) * 2021-03-18 2024-05-02 Cyprium Therapeutics, Inc. Copper histidinate compositions and uses thereof

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993017321A1 (en) 1992-02-25 1993-09-02 Unisearch Limited Electrothermal atomic absorption and preconcentration device
EP0606516B1 (en) 1993-01-13 1998-12-30 Idemitsu Kosan Company Limited Anti-human ceruloplasmin monoclonal antibody
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
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
US20070161120A1 (en) 2006-01-10 2007-07-12 Viorica Lopez-Avila Ceruloplasmin Assay
US8101727B2 (en) * 2006-03-30 2012-01-24 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
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
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
WO2010071832A1 (en) * 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
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
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
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

Also Published As

Publication number Publication date
US12173306B2 (en) 2024-12-24
WO2017070472A1 (en) 2017-04-27
PL3365438T3 (pl) 2021-10-18
JP2021052796A (ja) 2021-04-08
HUE054802T2 (hu) 2021-09-28
CN108431216B (zh) 2022-08-05
US10988778B2 (en) 2021-04-27
US20210214751A1 (en) 2021-07-15
AU2016341983B8 (en) 2022-10-06
PT3365438T (pt) 2021-07-12
DK3365438T3 (da) 2021-07-12
JP2018533941A (ja) 2018-11-22
CN108431216A (zh) 2018-08-21
US20250129382A1 (en) 2025-04-24
EP3365438A1 (en) 2018-08-29
JP6854286B2 (ja) 2021-04-07
US20180312871A1 (en) 2018-11-01
AU2016341983A8 (en) 2022-10-06
AU2016341983B2 (en) 2022-05-26
EP3365438B1 (en) 2021-04-28
CA3001574A1 (en) 2017-04-27
AU2016341983A1 (en) 2018-04-26

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