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) CN115957350A (fr)
AU (2) AU2016366560A1 (fr)
BR (1) BR112018011687A2 (fr)
CA (1) CA3008142A1 (fr)
CO (1) CO2018007165A2 (fr)
ES (1) ES2921450T3 (fr)
IL (2) IL303850A (fr)
MA (1) MA43570A (fr)
MX (1) MX2018007080A (fr)
WO (1) WO2017100682A1 (fr)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
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CA2946392A1 (fr) 2014-04-25 2015-10-29 James M. Wilson Variants de ldlr et leur utilisation dans des compositions permettant de reduire les taux de cholesterol
CA3008142A1 (fr) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Therapie genique pour traiter l'hypercholesterolemie familiale
KR20190045174A (ko) 2016-07-21 2019-05-02 스파크 테라퓨틱스, 인코포레이티드 재조합 아데노신 관련 바이러스(rAAV) 벡터를 고수율로 생산하기 위한 확장 가능한 고회수 방법 및 이에 의해 생산된 재조합 아데노-관련 바이러스(rAAV) 벡터
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
KR20190118163A (ko) * 2017-02-20 2019-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법
EA202091352A1 (ru) * 2017-11-30 2020-08-27 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Генотерапия мукополисахаридоза iiib
AU2019401638A1 (en) * 2018-12-20 2021-06-24 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
EA202191890A1 (ru) 2019-01-18 2022-02-03 Астразенека Аб Ингибиторы pcsk9 и способы их применения
US11248001B2 (en) 2019-01-18 2022-02-15 Astrazeneca Ab PCSK9 inhibitors and methods of use thereof
US20230270886A1 (en) * 2019-11-28 2023-08-31 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
IL297756A (en) * 2020-04-29 2022-12-01 Saliogen Therapeutics Inc Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol
EP4284335A1 (fr) 2021-02-01 2023-12-06 RegenxBio Inc. Thérapie génique de céroïdes-lipofuscinoses neuronales
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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ES2741502T3 (es) 2013-02-08 2020-02-11 Univ Pennsylvania Cápside de AAV8 modificada para la transferencia génica para terapias de retina
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WO2015012924A2 (fr) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Cassettes d'expression préférentielle pour tissus modifiées par un codon, vecteurs les contenant, et utilisation
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
CA2946392A1 (fr) * 2014-04-25 2015-10-29 James M. Wilson Variants de ldlr et leur utilisation dans des compositions permettant de reduire les taux de cholesterol
MY186389A (en) * 2014-05-13 2021-07-22 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
WO2017100676A1 (fr) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Procédé de purification évolutif d'aav8
CA3008142A1 (fr) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Therapie genique pour traiter l'hypercholesterolemie familiale
KR20190118163A (ko) 2017-02-20 2019-10-17 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법

Also Published As

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

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