[go: up one dir, main page]

CR20200507A - Composiciones aav, métodos de fabricación y métodos de uso - Google Patents

Composiciones aav, métodos de fabricación y métodos de uso

Info

Publication number
CR20200507A
CR20200507A CR20200507A CR20200507A CR20200507A CR 20200507 A CR20200507 A CR 20200507A CR 20200507 A CR20200507 A CR 20200507A CR 20200507 A CR20200507 A CR 20200507A CR 20200507 A CR20200507 A CR 20200507A
Authority
CR
Costa Rica
Prior art keywords
methods
making
aav compositions
pharmaceutical compositions
aav
Prior art date
Application number
CR20200507A
Other languages
English (en)
Inventor
Tuyen Ong
Valerie Girard
Richard Truran
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 CR20200507A publication Critical patent/CR20200507A/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/48—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)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (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

<p>Se describen métodos para la purificación de una partícula de AAV recombinante (rAAV) de un cultivo de células hospederas de mamífero. La descripción proporciona además una composición farmacéutica producida mediante los métodos de la descripción, las composiciones farmacéuticas y el método de uso de las composiciones farmacéuticas descritas aquí para el tratamiento de la enfermedad.</p>
CR20200507A 2018-04-05 2019-04-05 Composiciones aav, métodos de fabricación y métodos de uso CR20200507A (es)

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
CR20200507A true CR20200507A (es) 2021-03-02

Family

ID=66223873

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20200507A CR20200507A (es) 2018-04-05 2019-04-05 Composiciones aav, métodos de fabricación y métodos de uso

Country Status (19)

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

Families Citing this family (5)

* 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
CA3171573A1 (en) * 2020-03-16 2021-09-23 Jan Thomas PANTELI Methods for enhancing recombinant adeno-associated virus yield
BR112023003840A2 (pt) 2020-09-02 2023-04-04 4D Molecular Therapeutics Inc Genes de rep1 otimizados por códon e usos dos mesmos
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

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112012018899A2 (pt) * 2010-01-28 2015-09-15 Philadelphia Children Hospital "método para purificar partículas de vetor de vírus adeno-associado."
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
US20210145929A1 (en) 2021-05-20
JP2021520233A (ja) 2021-08-19
TW202000905A (zh) 2020-01-01
JOP20200250A1 (ar) 2020-10-04
CN112384626A (zh) 2021-02-19
PH12020551642A1 (en) 2021-07-26
CA3096051A1 (en) 2019-10-10
AU2019247866A1 (en) 2020-10-22
IL277786A (en) 2020-11-30
KR20210005045A (ko) 2021-01-13
WO2019195729A1 (en) 2019-10-10
MX2020010191A (es) 2021-02-26
BR112020020174A2 (pt) 2021-01-19
CL2020002566A1 (es) 2021-04-16
MA52200A (fr) 2021-02-17
EP3775234A1 (en) 2021-02-17
CO2020013699A2 (es) 2021-04-08
SG11202009698PA (en) 2020-10-29

Similar Documents

Publication Publication Date Title
PH12020551642A1 (en) Aav compositions, methods of making and methods of use
EA202190512A1 (ru) Композиции и способы для производства векторов для генной терапии
MX2020009394A (es) Anticuerpos anti-cd73 y métodos de uso de los mismos.
WO2019126634A3 (en) Targeted integration of nucleic acids
WO2019241535A3 (en) Anion exchange chromatography for recombinant aav production
MX2023013462A (es) Muteinas de interleucina 21 y usos de las mismas.
MX2020004578A (es) Composiciones de casz y metodos de uso.
ZA201705628B (en) Methods and compositions for treating genetic eye diseases
MX2011012039A (es) Anticuerpos bloqueadores anti-dkk-1 y sus usos.
CR20200327A (es) Proteínas de fusión fc il-22 y métodos de uso
WO2018170150A3 (en) Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins
EA201790025A1 (ru) Создание банка мезенхимальных стромальных клеток из объединенных мононуклеарных клеток нескольких доноров костного мозга
PH12022550373A1 (en) Ex vivo gamma delta t cell populations
AU2019433019A8 (en) Antibodies binding CD40 and uses thereof
PH12020550117A1 (en) Variant rnai
MX2021004213A (es) Generacion de pah humana mejorada para el tratamiento de pku grave mediante terapia de sustitucion de genes dirigida al higado.
MX2021012041A (es) Metodos para promover la diferenciacion de celulas epiteliales timicas y progenitores de celulas epiteliales timicas de celulas madre pluripotentes, celulas resultantes y usos de estas.
PH12021552577A1 (en) Engineered producer cell lines and methods of making and using the same
WO2019121961A3 (en) Methods for modulating protein mannosylation profiles using maduramycin, narasin, or salinomycin
MX2021004454A (es) Anticuerpos anti-sinucleina.
MY197684A (en) Oncolytic virus and method
MX2020002148A (es) Vectores adenoasociados recombinantes.
EA201791233A1 (ru) Материал стволовых клеток и способ его получения
AU2020256283A8 (en) Porcine circovirus type 3 (PCV3) vaccines, and production and uses thereof
WO2023023597A8 (en) Virus-like particle