MX2023003807A - Virus adenoasociados recombinantes para administracion en el snc o el musculo. - Google Patents

Virus adenoasociados recombinantes para administracion en el snc o el musculo.

Info

Publication number
MX2023003807A
MX2023003807A MX2023003807A MX2023003807A MX2023003807A MX 2023003807 A MX2023003807 A MX 2023003807A MX 2023003807 A MX2023003807 A MX 2023003807A MX 2023003807 A MX2023003807 A MX 2023003807A MX 2023003807 A MX2023003807 A MX 2023003807A
Authority
MX
Mexico
Prior art keywords
cns
associated viruses
recombinant adeno
muscle delivery
amino acid
Prior art date
Application number
MX2023003807A
Other languages
English (en)
Spanish (es)
Inventor
Ye Liu
Olivier Danos
Andrew Mercer
Samantha Yost
Subha Karumuthil-Melethil
Elad Firnberg
Joseph Bruder
Randolph Qian
April R Tepe
Jennifer M Egley
Original Assignee
Regenxbio 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 Regenxbio Inc filed Critical Regenxbio Inc
Priority claimed from PCT/US2021/054058 external-priority patent/WO2022076750A2/en
Publication of MX2023003807A publication Critical patent/MX2023003807A/es

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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurosurgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
MX2023003807A 2020-10-07 2021-10-07 Virus adenoasociados recombinantes para administracion en el snc o el musculo. MX2023003807A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063088988P 2020-10-07 2020-10-07
PCT/US2021/054058 WO2022076750A2 (en) 2020-10-07 2021-10-07 Recombinant adeno-associated viruses for cns or muscle delivery

Publications (1)

Publication Number Publication Date
MX2023003807A true MX2023003807A (es) 2023-04-12

Family

ID=86184347

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023003807A MX2023003807A (es) 2020-10-07 2021-10-07 Virus adenoasociados recombinantes para administracion en el snc o el musculo.

Country Status (9)

Country Link
US (1) US20230374541A1 (OSRAM)
EP (1) EP4225778A2 (OSRAM)
JP (1) JP2023545384A (OSRAM)
KR (1) KR20230082613A (OSRAM)
CN (1) CN116783209A (OSRAM)
AU (1) AU2021358546A1 (OSRAM)
CA (1) CA3193685A1 (OSRAM)
IL (1) IL301563A (OSRAM)
MX (1) MX2023003807A (OSRAM)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20260055144A1 (en) * 2022-08-24 2026-02-26 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018119330A2 (en) * 2016-12-22 2018-06-28 Oregon Health & Science University Adeno associated viral vectors
CA3052829A1 (en) * 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
CN113924115A (zh) * 2019-01-31 2022-01-11 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法

Also Published As

Publication number Publication date
AU2021358546A9 (en) 2024-02-08
AU2021358546A1 (en) 2023-06-08
JP2023545384A (ja) 2023-10-30
EP4225778A2 (en) 2023-08-16
US20230374541A1 (en) 2023-11-23
IL301563A (en) 2023-05-01
CN116783209A (zh) 2023-09-19
CA3193685A1 (en) 2022-04-14
KR20230082613A (ko) 2023-06-08

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