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JOP20200250A1 - تركيبات aav، وطرق تحضيرها وطرق الاستخدام - Google Patents

تركيبات aav، وطرق تحضيرها وطرق الاستخدام

Info

Publication number
JOP20200250A1
JOP20200250A1 JOP/2020/0250A JOP20200250A JOP20200250A1 JO P20200250 A1 JOP20200250 A1 JO P20200250A1 JO P20200250 A JOP20200250 A JO P20200250A JO P20200250 A1 JOP20200250 A1 JO P20200250A1
Authority
JO
Jordan
Prior art keywords
methods
making
aav compositions
pharmaceutical compositions
aav
Prior art date
Application number
JOP/2020/0250A
Other languages
English (en)
Inventor
Tuyen Ong
Richard Truran
Valerie Girard
Original Assignee
Nightstarx Ltd
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 Nightstarx Ltd filed Critical Nightstarx Ltd
Publication of JOP20200250A1 publication Critical patent/JOP20200250A1/ar

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/0018—Culture media for cell or tissue culture
    • C12N5/0031—Serum-free culture media
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/0018—Culture media for cell or tissue culture
    • C12N5/005—Protein-free medium
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y205/00—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
    • C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
    • C12Y205/0106—Protein geranylgeranyltransferase type II (2.5.1.60)
    • 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
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    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C—CHEMISTRY; METALLURGY
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Manufacturing & Machinery (AREA)
  • Cell Biology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

يكشف الاختراع الحالي عن طرق تنقية جسيم AAV ناتج عن عودة الاتحاد الجيني (rAAV) من مزرعة خلية عائلة ثديية. كما يوفر الكشف تركيبة صيدلية منتجة بواسطة طرق الكشف، وتركيبات صيدلية وطريقة استخدام التركيبات الصيدلية التي تم وصفها هنا لعلاج المرض.
JOP/2020/0250A 2018-04-05 2019-04-05 تركيبات aav، وطرق تحضيرها وطرق الاستخدام JOP20200250A1 (ar)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862653139P 2018-04-05 2018-04-05
US201862746980P 2018-10-17 2018-10-17
US201862773975P 2018-11-30 2018-11-30
PCT/US2019/026064 WO2019195729A1 (en) 2018-04-05 2019-04-05 Aav compositions, methods of making and methods of use

Publications (1)

Publication Number Publication Date
JOP20200250A1 true JOP20200250A1 (ar) 2020-10-04

Family

ID=66223873

Family Applications (1)

Application Number Title Priority Date Filing Date
JOP/2020/0250A JOP20200250A1 (ar) 2018-04-05 2019-04-05 تركيبات aav، وطرق تحضيرها وطرق الاستخدام

Country Status (19)

Country Link
US (1) US20210145929A1 (ar)
EP (1) EP3775234A1 (ar)
JP (1) JP2021520233A (ar)
KR (1) KR20210005045A (ar)
CN (1) CN112384626A (ar)
AU (1) AU2019247866A1 (ar)
BR (1) BR112020020174A2 (ar)
CA (1) CA3096051A1 (ar)
CL (1) CL2020002566A1 (ar)
CO (1) CO2020013699A2 (ar)
CR (1) CR20200507A (ar)
IL (1) IL277786A (ar)
JO (1) JOP20200250A1 (ar)
MA (1) MA52200A (ar)
MX (1) MX2020010191A (ar)
PH (1) PH12020551642A1 (ar)
SG (1) SG11202009698PA (ar)
TW (1) TW202000905A (ar)
WO (1) WO2019195729A1 (ar)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
US20230128412A1 (en) * 2020-03-16 2023-04-27 Ultragenyx Pharmaceutical Inc. Methods for enhancing recombinant adeno-associated virus yield
US11357870B2 (en) * 2020-09-02 2022-06-14 4D Molecular Therapeutics Inc. Codon optimized REP1 genes and uses thereof
WO2022109247A1 (en) * 2020-11-20 2022-05-27 Biogen Ma Inc. Viral vector potency assay
WO2023165969A1 (en) 2022-03-03 2023-09-07 Evonik Operations Gmbh Process to enhance viral vector production in cell-culture using glycyl-glutamine
WO2025076729A1 (zh) * 2023-10-11 2025-04-17 朗信启昇(苏州)生物制药有限公司 包含aav的药物组合物及其制备方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IN2012DN06629A (ar) * 2010-01-28 2015-10-23 Philadelphia Children Hospital
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
EP3054007A1 (en) * 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step

Also Published As

Publication number Publication date
CN112384626A (zh) 2021-02-19
SG11202009698PA (en) 2020-10-29
CR20200507A (es) 2021-03-02
US20210145929A1 (en) 2021-05-20
MX2020010191A (es) 2021-02-26
IL277786A (en) 2020-11-30
WO2019195729A1 (en) 2019-10-10
CL2020002566A1 (es) 2021-04-16
BR112020020174A2 (pt) 2021-01-19
MA52200A (fr) 2021-02-17
JP2021520233A (ja) 2021-08-19
CA3096051A1 (en) 2019-10-10
AU2019247866A1 (en) 2020-10-22
PH12020551642A1 (en) 2021-07-26
KR20210005045A (ko) 2021-01-13
TW202000905A (zh) 2020-01-01
EP3775234A1 (en) 2021-02-17
CO2020013699A2 (es) 2021-04-08

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