MA43570A - Thérapie génique pour traiter l'hypercholestérolémie familiale - Google Patents

Thérapie génique pour traiter l'hypercholestérolémie familiale

Info

Publication number
MA43570A
MA43570A MA043570A MA43570A MA43570A MA 43570 A MA43570 A MA 43570A MA 043570 A MA043570 A MA 043570A MA 43570 A MA43570 A MA 43570A MA 43570 A MA43570 A MA 43570A
Authority
MA
Morocco
Prior art keywords
treat
gene therapy
family hypercholesterolemia
hypercholesterolemia
family
Prior art date
Application number
MA043570A
Other languages
English (en)
Inventor
Daniel J Rader
James M Wilson
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of MA43570A publication Critical patent/MA43570A/fr

Links

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
    • C12N15/861—Adenoviral 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
    • A61K48/005—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
    • 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
    • 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
    • A61K48/0075—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
    • A61P3/06—Antihyperlipidemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Dermatology (AREA)
  • Manufacturing & Machinery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
MA043570A 2015-12-11 2016-12-09 Thérapie génique pour traiter l'hypercholestérolémie familiale MA43570A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562266383P 2015-12-11 2015-12-11
US201562269440P 2015-12-18 2015-12-18

Publications (1)

Publication Number Publication Date
MA43570A true MA43570A (fr) 2018-11-14

Family

ID=57794341

Family Applications (1)

Application Number Title Priority Date Filing Date
MA043570A MA43570A (fr) 2015-12-11 2016-12-09 Thérapie génique pour traiter l'hypercholestérolémie familiale

Country Status (14)

Country Link
US (2) US10889832B2 (fr)
EP (2) EP4085934A1 (fr)
JP (2) JP7328760B2 (fr)
KR (1) KR20180113990A (fr)
CN (2) CN109121421B (fr)
AU (2) AU2016366560A1 (fr)
BR (1) BR112018011687A2 (fr)
CA (1) CA3008142A1 (fr)
CO (1) CO2018007165A2 (fr)
ES (1) ES2921450T3 (fr)
IL (2) IL259850B2 (fr)
MA (1) MA43570A (fr)
MX (1) MX2018007080A (fr)
WO (1) WO2017100682A1 (fr)

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EP4085934A1 (fr) 2015-12-11 2022-11-09 The Trustees of The University of Pennsylvania Thérapie génique pour traiter l'hypercholestérolémie familiale
AU2017299779B2 (en) 2016-07-21 2021-12-02 Spark Therapeutics, Inc. Scalable high recovery methods for producing high yield recombinant adeno-associated viral (rAAV) vector and recombinant adeno-associated viral (rAAV) vectors produced thereby
WO2018022511A1 (fr) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprenant un variant de la lécithine-cholestérol-acyl-transférase et leurs utilisations
JP2020510648A (ja) 2017-02-20 2020-04-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 家族性高コレステロール血症を処置するための遺伝子治療
EA202091352A1 (ru) * 2017-11-30 2020-08-27 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Генотерапия мукополисахаридоза iiib
JP2022518354A (ja) * 2018-12-20 2022-03-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 家族性高コレステロール血症を治療するための遺伝子療法
WO2020150473A2 (fr) 2019-01-18 2020-07-23 Dogma Therapeutics, Inc. Inhibiteurs de pcsk9 et leurs procédés d'utilisation
BR112021013924A2 (pt) 2019-01-18 2021-09-21 Astrazeneca Ab Inibidores de pcsk9 e métodos de uso do mesmo
AU2020392243A1 (en) * 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
WO2021222653A1 (fr) * 2020-04-29 2021-11-04 Saliogen Therapeutics, Inc. Compositions et méthodes pour le traitement de l'hypercholestérolémie familiale et du cholestérol à lipoprotéines de basse densité élevée
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
CN117887723A (zh) * 2024-01-08 2024-04-16 苏州诺洁贝生物技术有限公司 密码子优化的ldlr基因及其应用

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AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
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DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
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CN1780910A (zh) * 2003-01-28 2006-05-31 拉塞有限公司 对与家族性高胆固醇血症有关的分离的低密度脂蛋白受体(ldl－r)的基因序列中的突变进行检测的方法和装置
EP1668143B1 (fr) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Variantes des virus associes aux adenovirus (aav), sequences, vecteurs les contenant, et leur utilisation
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WO2011126808A2 (fr) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Système d'ablation de transgène induit pharmacologiquement
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EP3795581A3 (fr) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University Nouvelles protéines de capside avv pour le transfert d'acides nucléiques
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WO2014124282A1 (fr) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Transfert génétique médié par aav amélioré pour thérapies rétiniennes
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BR112015021036A2 (pt) * 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
US11555059B2 (en) 2014-04-25 2023-01-17 The Trustees Of The University Of Pennsylvania LDLR variants and their use in compositions for reducing cholesterol levels
CR20160519A (es) * 2014-05-13 2017-01-27 The Trustees Of The Univ Of Pennysylvania Composiciones que comprenden avv que expresa constructos de anticuerpos duales y sus usos
EP4085934A1 (fr) 2015-12-11 2022-11-09 The Trustees of The University of Pennsylvania Thérapie génique pour traiter l'hypercholestérolémie familiale
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
JP2020510648A (ja) 2017-02-20 2020-04-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 家族性高コレステロール血症を処置するための遺伝子治療

Also Published As

Publication number Publication date
AU2023201657A1 (en) 2023-06-15
CN115957350A (zh) 2023-04-14
AU2016366560A1 (en) 2018-06-28
JP2022050575A (ja) 2022-03-30
CA3008142A1 (fr) 2017-06-15
IL259850B2 (en) 2023-11-01
CN109121421B (zh) 2022-11-01
WO2017100682A1 (fr) 2017-06-15
KR20180113990A (ko) 2018-10-17
EP3400304B1 (fr) 2022-04-06
US20190002917A1 (en) 2019-01-03
JP7328760B2 (ja) 2023-08-17
CO2018007165A2 (es) 2018-07-19
US10889832B2 (en) 2021-01-12
IL303850A (en) 2023-08-01
US20210285013A1 (en) 2021-09-16
EP3400304A1 (fr) 2018-11-14
BR112018011687A2 (pt) 2018-12-04
MX2018007080A (es) 2018-11-12
EP4085934A1 (fr) 2022-11-09
IL259850B1 (en) 2023-07-01
IL259850A (en) 2018-07-31
JP2019504003A (ja) 2019-02-14
ES2921450T3 (es) 2022-08-25
CN109121421A (zh) 2019-01-01

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