KR20180113990A - 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 - Google Patents

가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 Download PDF

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KR20180113990A
KR20180113990A KR1020187019657A KR20187019657A KR20180113990A KR 20180113990 A KR20180113990 A KR 20180113990A KR 1020187019657 A KR1020187019657 A KR 1020187019657A KR 20187019657 A KR20187019657 A KR 20187019657A KR 20180113990 A KR20180113990 A KR 20180113990A
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제임스 엠. 윌슨
다니엘 제이. 레이더
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더 트러스티스 오브 더 유니버시티 오브 펜실바니아
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    • 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
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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    • 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
    • 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

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  • General Engineering & Computer Science (AREA)
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  • Obesity (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Manufacturing & Machinery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020187019657A 2015-12-11 2016-12-09 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 Ceased KR20180113990A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562266383P 2015-12-11 2015-12-11
US62/266,383 2015-12-11
US201562269440P 2015-12-18 2015-12-18
US62/269,440 2015-12-18
PCT/US2016/065984 WO2017100682A1 (en) 2015-12-11 2016-12-09 Gene therapy for treating familial hypercholesterolemia

Publications (1)

Publication Number Publication Date
KR20180113990A true KR20180113990A (ko) 2018-10-17

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KR1020187019657A Ceased KR20180113990A (ko) 2015-12-11 2016-12-09 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법

Country Status (14)

Country Link
US (2) US10889832B2 (https=)
EP (2) EP3400304B1 (https=)
JP (2) JP7328760B2 (https=)
KR (1) KR20180113990A (https=)
CN (2) CN115957350A (https=)
AU (2) AU2016366560A1 (https=)
BR (1) BR112018011687A2 (https=)
CA (1) CA3008142A1 (https=)
CO (1) CO2018007165A2 (https=)
ES (1) ES2921450T3 (https=)
IL (2) IL303850A (https=)
MA (1) MA43570A (https=)
MX (1) MX2018007080A (https=)
WO (1) WO2017100682A1 (https=)

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IL303850A (en) 2015-12-11 2023-08-01 Univ Pennsylvania Gene therapy for treating familial hypercholesterolemia
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IL259850B1 (en) 2023-07-01
WO2017100682A1 (en) 2017-06-15
CO2018007165A2 (es) 2018-07-19
AU2023201657A1 (en) 2023-06-15
EP3400304A1 (en) 2018-11-14
IL303850A (en) 2023-08-01
JP7328760B2 (ja) 2023-08-17
US20210285013A1 (en) 2021-09-16
IL259850B2 (en) 2023-11-01
CN109121421A (zh) 2019-01-01
CA3008142A1 (en) 2017-06-15
IL259850A (en) 2018-07-31
MA43570A (fr) 2018-11-14
JP2022050575A (ja) 2022-03-30
EP3400304B1 (en) 2022-04-06
BR112018011687A2 (pt) 2018-12-04
JP2019504003A (ja) 2019-02-14
US20190002917A1 (en) 2019-01-03
ES2921450T3 (es) 2022-08-25
US10889832B2 (en) 2021-01-12
MX2018007080A (es) 2018-11-12
AU2016366560A1 (en) 2018-06-28
CN115957350A (zh) 2023-04-14
CN109121421B (zh) 2022-11-01
EP4085934A1 (en) 2022-11-09

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