AU2016341983B8 - Codon-optimized reduced-size ATP7A cDNA and uses for treatment of copper transport disorders - Google Patents

Codon-optimized reduced-size ATP7A cDNA and uses for treatment of copper transport disorders Download PDF

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Publication number
AU2016341983B8
AU2016341983B8 AU2016341983A AU2016341983A AU2016341983B8 AU 2016341983 B8 AU2016341983 B8 AU 2016341983B8 AU 2016341983 A AU2016341983 A AU 2016341983A AU 2016341983 A AU2016341983 A AU 2016341983A AU 2016341983 B8 AU2016341983 B8 AU 2016341983B8
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copper
nucleic acid
atp7a
vector
codon
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AU2016341983B2 (en
AU2016341983A8 (en
AU2016341983A1 (en
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Stephen G. Kaler
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US Department of Health and Human Services
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Government of the United States of America
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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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Inorganic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2016341983A 2015-10-21 2016-10-21 Codon-optimized reduced-size ATP7A cDNA and uses for treatment of copper transport disorders Active AU2016341983B8 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562244594P 2015-10-21 2015-10-21
US62/244,594 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 (4)

Publication Number Publication Date
AU2016341983A1 AU2016341983A1 (en) 2018-04-26
AU2016341983B2 AU2016341983B2 (en) 2022-05-26
AU2016341983A8 AU2016341983A8 (en) 2022-10-06
AU2016341983B8 true AU2016341983B8 (en) 2022-10-06

Family

ID=57256420

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2016341983A Active AU2016341983B8 (en) 2015-10-21 2016-10-21 Codon-optimized reduced-size ATP7A cDNA and uses for treatment of copper transport disorders

Country Status (12)

Country Link
US (3) US10988778B2 (enExample)
EP (1) EP3365438B1 (enExample)
JP (2) JP6854286B2 (enExample)
CN (1) CN108431216B (enExample)
AU (1) AU2016341983B8 (enExample)
CA (1) CA3001574A1 (enExample)
DK (1) DK3365438T3 (enExample)
ES (1) ES2879400T3 (enExample)
HU (1) HUE054802T2 (enExample)
PL (1) PL3365438T3 (enExample)
PT (1) PT3365438T (enExample)
WO (1) WO2017070472A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020014594A1 (en) * 2018-07-12 2020-01-16 The Taxas A&M University System Compositions for the treatment of copper deficiency and methods of use
AU2022240726A1 (en) * 2021-03-18 2023-09-21 Cyprium Therapeutics, Inc. Copper histidinate compositions and uses thereof

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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
EP1625210B1 (en) 2003-05-21 2010-12-01 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
MX2008012485A (es) * 2006-03-30 2008-10-10 Novartis Ag Composiciones y metodos de uso para anticuerpos de c-met.
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009097129A1 (en) 2008-01-29 2009-08-06 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in 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
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
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
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
AU2013243949A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
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Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DONSANTE ANTHONY ET AL, "ATP7A Gene Addition to the Choroid Plexus Results in Long-term Rescue of the Lethal Copper Transport Defect in a Menkes Disease Mouse Model", MOLECULAR THERAPY, (2011-12), vol. 19, no. 12, ISSN 1525-0016 pp2114-2123 *

Also Published As

Publication number Publication date
CN108431216A (zh) 2018-08-21
DK3365438T3 (da) 2021-07-12
JP6854286B2 (ja) 2021-04-07
EP3365438B1 (en) 2021-04-28
WO2017070472A1 (en) 2017-04-27
CA3001574A1 (en) 2017-04-27
ES2879400T3 (es) 2021-11-22
AU2016341983B2 (en) 2022-05-26
US20250129382A1 (en) 2025-04-24
JP2021052796A (ja) 2021-04-08
HUE054802T2 (hu) 2021-09-28
JP2018533941A (ja) 2018-11-22
US10988778B2 (en) 2021-04-27
US20180312871A1 (en) 2018-11-01
AU2016341983A8 (en) 2022-10-06
US20210214751A1 (en) 2021-07-15
EP3365438A1 (en) 2018-08-29
AU2016341983A1 (en) 2018-04-26
PT3365438T (pt) 2021-07-12
US12173306B2 (en) 2024-12-24
CN108431216B (zh) 2022-08-05
PL3365438T3 (pl) 2021-10-18

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Free format text: IN VOL 36 , NO 21 , PAGE(S) 3014 UNDER THE HEADING APPLICATIONS ACCEPTED - NAME INDEX UNDER THE NAME THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES, APPLICATION NO. 2016341983, UNDER INID (54) CORRECT THE TITLE TO READ CODON-OPTIMIZED REDUCED-SIZE ATP7A CDNA AND USES FOR TREATMENT OF COPPER TRANSPORT DISORDERS

Free format text: IN VOL 32 , NO 16 , PAGE(S) 2278 UNDER THE HEADING PCT APPLICATIONS THAT HAVE ENTERED THE NATIONAL PHASE - NAME INDEX UNDER THE NAME THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES, APPLICATION NO. 2016341983, UNDER INID (54) CORRECT THE TITLE TO READ CODON-OPTIMIZED REDUCED-SIZE ATP7A CDNA AND USES FOR TREATMENT OF COPPER TRANSPORT DISORDERS