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 schorzeń

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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
Application number
PL15817239T
Other languages
English (en)
Inventor
Oihana MURILLO SAUCA
Gloria GONZÁLEZ ASEGUINOLAZA
Rubén HERNÁNDEZ ALCOCEBA
Original Assignee
Fundacion Para La Investigacion Medica Aplicada
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Fundacion Para La Investigacion Medica Aplicada filed Critical Fundacion Para La Investigacion Medica Aplicada
Publication of PL3233129T3 publication Critical patent/PL3233129T3/pl

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    • 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)
PL15817239T 2014-12-17 2015-12-17 Konstrukty kwasu nukleinowego i wektory terapii genowej do zastosowania w leczeniu choroby Wilsona i innych schorzeń PL3233129T3 (pl)

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) EP3233129B1 (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 (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3233129T3 (da) 2014-12-17 2020-04-06 Fundacion Para La Investig Medica Aplicada Nukleinsyrekonstrukter og genterapivektorer til anvendelse i behandlingen af wilsons sygdom og andre lidelser
MX2017007965A (es) 2014-12-17 2018-01-18 Fundacion Para La Investig Medica Aplicada Construcciones de acido nucleico y vectores de terapia de genes para uso en el tratamiento de enfermedad de wilson.
GB201522416D0 (en) * 2015-12-18 2016-02-03 Ucl Business Plc Wilson's disease gene therapy
KR102604096B1 (ko) * 2016-12-30 2023-11-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 윌슨병을 치료하기 위한 유전자 치료
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
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
EP3813871A4 (en) 2018-06-29 2022-10-12 Icahn School of Medicine at Mount Sinai SPHINGOLIPID METABOLIZING PROTEINS ENCODING FOR ANC80
US20210369870A1 (en) 2018-11-16 2021-12-02 Encoded Therapeutics, Inc. Compositions and methods for treating wilson's disease
WO2021050064A1 (en) * 2019-09-11 2021-03-18 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
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
WO2022187571A1 (en) 2021-03-04 2022-09-09 Research Institute At Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography

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US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
JP3534749B2 (ja) 1991-08-20 2004-06-07 アメリカ合衆国 アデノウイルスが介在する胃腸管への遺伝子の輸送
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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
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Also Published As

Publication number Publication date
SI3233129T1 (sl) 2020-07-31
ES2784264T3 (es) 2020-09-23
HUE048551T2 (hu) 2020-08-28
JP2018505663A (ja) 2018-03-01
EP3233129B1 (en) 2020-01-15
DK3233129T3 (da) 2020-04-06
US11118238B2 (en) 2021-09-14
US20170356060A1 (en) 2017-12-14
PT3233129T (pt) 2020-04-15
JP6956635B2 (ja) 2021-11-02
EP3744351A1 (en) 2020-12-02
WO2016097218A1 (en) 2016-06-23
EP3233129A1 (en) 2017-10-25

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