[go: up one dir, main page]

MX2020010191A - Composiciones de virus adenoasociado (aav), métodos de fabricación y métodos de uso. - Google Patents

Composiciones de virus adenoasociado (aav), métodos de fabricación y métodos de uso.

Info

Publication number
MX2020010191A
MX2020010191A MX2020010191A MX2020010191A MX2020010191A MX 2020010191 A MX2020010191 A MX 2020010191A MX 2020010191 A MX2020010191 A MX 2020010191A MX 2020010191 A MX2020010191 A MX 2020010191A MX 2020010191 A MX2020010191 A MX 2020010191A
Authority
MX
Mexico
Prior art keywords
methods
making
aav compositions
pharmaceutical compositions
aav
Prior art date
Application number
MX2020010191A
Other languages
English (en)
Inventor
Valerie Girard
Richard Truran
Tuyen Ong
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 MX2020010191A publication Critical patent/MX2020010191A/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
    • 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
    • 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
    • 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
    • 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
    • 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)

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

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.
MX2020010191A 2018-04-05 2019-04-05 Composiciones de virus adenoasociado (aav), métodos de fabricación y métodos de uso. MX2020010191A (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
MX2020010191A true MX2020010191A (es) 2021-02-26

Family

ID=66223873

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020010191A MX2020010191A (es) 2018-04-05 2019-04-05 Composiciones de virus adenoasociado (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 (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 (es) * 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
JOP20200250A1 (ar) 2020-10-04
CN112384626A (zh) 2021-02-19
SG11202009698PA (en) 2020-10-29
CR20200507A (es) 2021-03-02
US20210145929A1 (en) 2021-05-20
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

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
WO2019126634A3 (en) Targeted integration of nucleic acids
WO2019173692A3 (en) Anti-cd73 antibodies and methods of use thereof
ZA202210065B (en) Interleukin-21 muteins and methods of treatment
WO2019241535A3 (en) Anion exchange chromatography for recombinant aav production
BR112021022874A2 (pt) Proteínas de ligação epcam e métodos de uso
MX2020010871A (es) Compuestos de 5'-caperuza-trinucleótidos u oligonucleótidos superiores y sus usos en estabilizar arn, que expresan proteínas y en la terapia.
EP4435007A3 (en) Trispecific proteins and methods of use
MX2021006615A (es) Proteinas de union triespecificas y metodos de uso.
EA202091712A1 (ru) МОДИФИЦИРОВАННЫЙ КАПСИДНЫЙ БЕЛОК rAAV ДЛЯ ГЕННОЙ ТЕРАПИИ
ZA201804654B (en) Anti-cd74 antibody conjugates, compositions comprising anti-cd74 antibody conjugates and methods of using anti-cd74 antibody conjugates
EP4512429A3 (en) Production of oversized adeno-associated vectors
WO2018170150A3 (en) Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins
MX2013000958A (es) Fabricación del inhibidor-inter-alfa (iaip) a partir de plasma.
PH12022500003A1 (en) Il-2 conjugates and methods of use to treat autoimmune diseases
PH12022550460A1 (en) Anti-cd96 antibodies and methods of use thereof
MX2022013963A (es) Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas.
HK1252778A1 (zh) 細胞擴增方法和治療組合物
WO2019121961A3 (en) Methods for modulating protein mannosylation profiles using maduramycin, narasin, or salinomycin
PH12021552577A1 (en) Engineered producer cell lines and methods of making and using the same
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.
MX2021004454A (es) Anticuerpos anti-sinucleina.
MX2021000322A (es) Enzimas de variante fosfopentomutasa modificada geneticamente.
AU2020256283A8 (en) Porcine circovirus type 3 (PCV3) vaccines, and production and uses thereof