SI3233130T1 - Nukleinskokislinski konstrukti in vektorji za gensko terapijo za uporabo pri zdravljenju Wilsonove bolezni - Google Patents

Nukleinskokislinski konstrukti in vektorji za gensko terapijo za uporabo pri zdravljenju Wilsonove bolezni

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Publication number
SI3233130T1
SI3233130T1 SI201531622T SI201531622T SI3233130T1 SI 3233130 T1 SI3233130 T1 SI 3233130T1 SI 201531622 T SI201531622 T SI 201531622T SI 201531622 T SI201531622 T SI 201531622T SI 3233130 T1 SI3233130 T1 SI 3233130T1
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Slovenia
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treatment
nucleic acid
gene therapy
acid constructs
therapy vectors
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SI201531622T
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English (en)
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Sauca Oihana Murillo
Aseguinolaza Gloria Gonzalez
Alcoceba Ruben Hernandez
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Fundacion Para La Investigacion Medica Aplicada
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Publication of SI3233130T1 publication Critical patent/SI3233130T1/sl

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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • C12YENZYMES
    • C12Y306/00Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
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    • C12Y306/03004Cu2+-exporting ATPase (3.6.3.4)
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011Parvoviridae
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/80Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85Vector systems having a special element relevant for transcription from vertebrates mammalian

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SI201531622T 2014-12-17 2015-12-17 Nukleinskokislinski konstrukti in vektorji za gensko terapijo za uporabo pri zdravljenju Wilsonove bolezni SI3233130T1 (sl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP14382531 2014-12-17
PCT/EP2015/080357 WO2016097219A1 (en) 2014-12-17 2015-12-17 Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease
EP15817240.3A EP3233130B1 (en) 2014-12-17 2015-12-17 Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson disease

Publications (1)

Publication Number Publication Date
SI3233130T1 true SI3233130T1 (sl) 2021-08-31

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SI201531622T SI3233130T1 (sl) 2014-12-17 2015-12-17 Nukleinskokislinski konstrukti in vektorji za gensko terapijo za uporabo pri zdravljenju Wilsonove bolezni

Country Status (19)

Country Link
US (2) US11147887B2 (sl)
EP (2) EP3799889A1 (sl)
JP (1) JP6865167B2 (sl)
KR (1) KR102526616B1 (sl)
CN (1) CN107208105A (sl)
AU (1) AU2015367385B2 (sl)
BR (1) BR112017012895A2 (sl)
CA (1) CA2985624C (sl)
DK (1) DK3233130T3 (sl)
ES (1) ES2876412T3 (sl)
HU (1) HUE055002T2 (sl)
IL (1) IL252917B2 (sl)
MX (1) MX2017007965A (sl)
PL (1) PL3233130T3 (sl)
PT (1) PT3233130T (sl)
RU (1) RU2745567C2 (sl)
SG (1) SG11201704690TA (sl)
SI (1) SI3233130T1 (sl)
WO (1) WO2016097219A1 (sl)

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JP7315475B2 (ja) * 2017-05-19 2023-07-26 エンコーデッド セラピューティクス, インコーポレイテッド 高活性制御エレメント
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF
TW202039854A (zh) * 2018-11-16 2020-11-01 美商編碼製藥公司 治療威爾遜氏病的組合物和方法
EP4269579A3 (en) * 2019-01-04 2024-01-24 Ultragenyx Pharmaceutical Inc. Gene therapy constructs for treating wilson disease
KR102159877B1 (ko) * 2019-01-29 2020-09-24 (주) 넥셀 인간 전분화능 줄기세포를 이용한 생체 외 윌슨병 모델 제조방법 및 이를 통해 제조된 생체 외 윌슨병 모델
AU2020274200A1 (en) * 2019-05-14 2021-11-25 Duke University Compositions and methods for the treatment of ATPase-mediated diseases
CN111088285B (zh) * 2019-08-15 2021-12-28 北京锦篮基因科技有限公司 携带atp7b基因表达框及变异体的aav载体及应用
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
AU2021375404A1 (en) 2020-11-03 2023-06-08 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of AAV vectors by affinity chromatography
AU2022229489A1 (en) 2021-03-04 2023-08-31 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
CN113694218A (zh) * 2021-08-30 2021-11-26 昆明理工大学 一种atp7b基因p992l突变的基因修复治疗载体
CA3230084A1 (en) * 2021-09-07 2023-03-16 Eugene Scott SWENSON Methods of treating copper metabolism-associated diseases or disorders
AR127398A1 (es) * 2021-10-18 2024-01-17 Logicbio Therapeutics Inc Terapia génica para el tratamiento de la enfermedad de wilson
WO2023230466A1 (en) * 2022-05-23 2023-11-30 Lutsenka Svetlana Compositions and methods for treating wilson's disease
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography

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CA2985624A1 (en) 2016-06-23
RU2017125234A3 (sl) 2019-07-25
US20220008559A1 (en) 2022-01-13
BR112017012895A2 (pt) 2018-02-06
EP3233130B1 (en) 2021-03-31
PL3233130T3 (pl) 2021-11-22
IL252917A0 (en) 2017-08-31
US11147887B2 (en) 2021-10-19
CA2985624C (en) 2023-06-13
HUE055002T2 (hu) 2021-10-28
PT3233130T (pt) 2021-06-24
EP3799889A1 (en) 2021-04-07
KR20170108951A (ko) 2017-09-27
AU2015367385B2 (en) 2021-05-27
RU2745567C2 (ru) 2021-03-29
DK3233130T3 (da) 2021-06-21
AU2015367385A1 (en) 2017-06-29
WO2016097219A1 (en) 2016-06-23
ES2876412T3 (es) 2021-11-12
JP6865167B2 (ja) 2021-04-28
KR102526616B1 (ko) 2023-04-27
MX2017007965A (es) 2018-01-18
CN107208105A (zh) 2017-09-26
US20170348435A1 (en) 2017-12-07
IL252917B1 (en) 2023-06-01
IL252917B2 (en) 2023-10-01
SG11201704690TA (en) 2017-07-28
EP3233130A1 (en) 2017-10-25
RU2017125234A (ru) 2019-01-17
JP2018500904A (ja) 2018-01-18

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