MX2021000810A - Sistemas y metodos para producir formulaciones de terapia genetica. - Google Patents

Sistemas y metodos para producir formulaciones de terapia genetica.

Info

Publication number
MX2021000810A
MX2021000810A MX2021000810A MX2021000810A MX2021000810A MX 2021000810 A MX2021000810 A MX 2021000810A MX 2021000810 A MX2021000810 A MX 2021000810A MX 2021000810 A MX2021000810 A MX 2021000810A MX 2021000810 A MX2021000810 A MX 2021000810A
Authority
MX
Mexico
Prior art keywords
methods
aav
particles
systems
formulations
Prior art date
Application number
MX2021000810A
Other languages
English (en)
Inventor
Dinah Wen- Yee Sah
Pengcheng Zhou
Todd Carter
Jacob J Cardinal
Robert Steininger
Lori B Karpes
Christopher J Morrison
Daniel S Hurwit
Matthew Luther
Andrew M Wood
THOMPSON Jeffrey S
Christina Gamba-Vitalo
Soper Jenna Carroll
Steven M Hersch
Original Assignee
Voyager Therapeutics Inc
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 Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of MX2021000810A publication Critical patent/MX2021000810A/es

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    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C12N7/02—Recovery or purification
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Psychology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente divulgacion describe metodos y sistemas para su uso en la produccion de particulas de virus adenoasociado (AAV) y formulaciones de AAV, incluyendo particulas y formulaciones de virus adenoasociado recombinantes (rAAV). En determinadas modalidades, la presente divulgacion presenta metodos y sistemas para clarificar, purificar, formular, filtrar y procesar particulas de AAV y formulaciones de AAV. La presente divulgacion tambien describe composiciones, metodos y procesos para el diseno, preparacion, fabricacion, uso y/o formulacion de particulas de AAV que comprenden polinucleotidos moduladores, por ejemplo, polinucleotidos que codifican moleculas de ARN interferente pequeno (ARNip) que se dirigen al gen de la Huntingtina (HTT) (por ejemplo, el gen HTT expandido con CAG de tipo silvestre o mutado). Tambien se describen metodos para usar particulas de AAV formuladas que comprenden polinucleotidos moduladores para inhibir la expresion del gen HTT en un sujeto con una enfermedad neurodegenerativa (por ejemplo, enfermedad de Huntington (HD)).
MX2021000810A 2018-07-24 2019-07-24 Sistemas y metodos para producir formulaciones de terapia genetica. MX2021000810A (es)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US201862702679P 2018-07-24 2018-07-24
US201862702687P 2018-07-24 2018-07-24
US201862725432P 2018-08-31 2018-08-31
US201862741508P 2018-10-04 2018-10-04
US201962794213P 2019-01-18 2019-01-18
US201962794199P 2019-01-18 2019-01-18
US201962794212P 2019-01-18 2019-01-18
US201962826363P 2019-03-29 2019-03-29
US201962839880P 2019-04-29 2019-04-29
PCT/US2019/043196 WO2020023612A1 (en) 2018-07-24 2019-07-24 Systems and methods for producing gene therapy formulations

Publications (1)

Publication Number Publication Date
MX2021000810A true MX2021000810A (es) 2021-04-28

Family

ID=67766241

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021000810A MX2021000810A (es) 2018-07-24 2019-07-24 Sistemas y metodos para producir formulaciones de terapia genetica.

Country Status (10)

Country Link
US (1) US20210355454A1 (es)
EP (1) EP3826719A1 (es)
JP (1) JP2021530548A (es)
CN (1) CN112770812A (es)
AU (1) AU2019310459A1 (es)
CA (1) CA3107462A1 (es)
IL (1) IL280350A (es)
MX (1) MX2021000810A (es)
SG (1) SG11202100704PA (es)
WO (1) WO2020023612A1 (es)

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US20210355454A1 (en) 2021-11-18
EP3826719A1 (en) 2021-06-02

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