CL2019002474A1 - Vector clado f de virus adeno-asociado (vaa) y usos relacionados. - Google Patents

Vector clado f de virus adeno-asociado (vaa) y usos relacionados.

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Publication number
CL2019002474A1
CL2019002474A1 CL2019002474A CL2019002474A CL2019002474A1 CL 2019002474 A1 CL2019002474 A1 CL 2019002474A1 CL 2019002474 A CL2019002474 A CL 2019002474A CL 2019002474 A CL2019002474 A CL 2019002474A CL 2019002474 A1 CL2019002474 A1 CL 2019002474A1
Authority
CL
Chile
Prior art keywords
vector
associated virus
raav
seq
aavhu68
Prior art date
Application number
CL2019002474A
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English (en)
Inventor
James M Wilson
Qiang Wang
April Giles
Kevin Turner
Original Assignee
Univ Pennsylvania
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Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of CL2019002474A1 publication Critical patent/CL2019002474A1/es

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    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/32Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

UN VECTOR DE VIRUS ADENOASOCIADO RECOMBINANTE (RAAV), QUE COMPRENDE UNA CÁPSIDE DE AAVHU68 PRODUCIDA EN UN SISTEMA DE PRODUCCIÓN QUE COMPRENDE UNA SECUENCIA DE NUCLEÓTIDOS DE LA SEQ ID NO: 1, O UNA SECUENCIA AL MENOS 75 % IDÉNTICA A ESTA, QUE CODIFICA LA SEQ ID NO:2. LA CÁPSIDE DE AAVHU68 COMPRENDE SUBPOBLACIONES DE RESIDUOS DE ASPARAGINA MUY DESAMIDADOS EN LOS PARES DE ASPARAGINA-GLICINA EN LA SECUENCIA DE AMINOÁCIDOS DE LA SEQ ID NO: 2. TAMBIÉN SE PROPORCIONAN COMPOSICIONES QUE CONTIENEN EL RAAV Y USOS DE ESTAS. ADEMÁS, SE PROPORCIONAN RAAV QUE TIENEN UNA CÁPSIDE DE AAV MODIFICADA, QUE COMPRENDE AL MENOS UNA SUBPOBLACIÓN DE PROTEÍNAS VP1 O VP2 CON UNA VAL EN LA POSICIÓN DE AMINOÁCIDOS 157, CON REFERENCIA A LA NUMERACIÓN DE VP1 DE AAVHU68.
CL2019002474A 2017-02-28 2019-08-27 Vector clado f de virus adeno-asociado (vaa) y usos relacionados. CL2019002474A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762464748P 2017-02-28 2017-02-28
US201762591002P 2017-11-27 2017-11-27
US201862614002P 2018-01-05 2018-01-05

Publications (1)

Publication Number Publication Date
CL2019002474A1 true CL2019002474A1 (es) 2019-11-29

Family

ID=61628475

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2019002474A CL2019002474A1 (es) 2017-02-28 2019-08-27 Vector clado f de virus adeno-asociado (vaa) y usos relacionados.

Country Status (28)

Country Link
US (2) US11827906B2 (es)
EP (2) EP4272765A3 (es)
JP (2) JP7455579B2 (es)
KR (1) KR20190132639A (es)
CN (1) CN110741082B (es)
AU (1) AU2018227440B2 (es)
BR (1) BR112019017839A2 (es)
CA (1) CA3053399A1 (es)
CL (1) CL2019002474A1 (es)
CO (1) CO2019009525A2 (es)
DK (1) DK3589730T5 (es)
ES (1) ES2971872T3 (es)
FI (1) FI3589730T3 (es)
HR (1) HRP20240257T1 (es)
HU (1) HUE065885T2 (es)
IL (1) IL268644A (es)
LT (1) LT3589730T (es)
MD (1) MD3589730T2 (es)
MX (1) MX2019010275A (es)
PE (1) PE20191362A1 (es)
PL (1) PL3589730T3 (es)
PT (1) PT3589730T (es)
RS (1) RS65241B1 (es)
SA (1) SA519402565B1 (es)
SG (4) SG10201912976WA (es)
SI (1) SI3589730T1 (es)
WO (1) WO2018160582A1 (es)
ZA (1) ZA201905407B (es)

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