MX2021013266A - Nuevas capsides de aav y composiciones que las contienen. - Google Patents

Nuevas capsides de aav y composiciones que las contienen.

Info

Publication number
MX2021013266A
MX2021013266A MX2021013266A MX2021013266A MX2021013266A MX 2021013266 A MX2021013266 A MX 2021013266A MX 2021013266 A MX2021013266 A MX 2021013266A MX 2021013266 A MX2021013266 A MX 2021013266A MX 2021013266 A MX2021013266 A MX 2021013266A
Authority
MX
Mexico
Prior art keywords
novel aav
compositions containing
containing same
aav capsids
novel
Prior art date
Application number
MX2021013266A
Other languages
English (en)
Inventor
James M Wilson
Kalyani Nambiar
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of MX2021013266A publication Critical patent/MX2021013266A/es

Links

Classifications

    • 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
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • 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
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • 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
    • C12N2750/14151Methods of production or purification of viral material
    • 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
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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)
  • Medicinal Preparation (AREA)
  • Materials For Medical Uses (AREA)

Abstract

En el presente documento se proporcionan nuevas cápsides de AAV y rAAV que las comprenden; en una forma de realización, los vectores que emplean una nueva cápside de AAV muestran una mayor transducción de un tejido diana seleccionado en comparación con un AAV de la técnica anterior.
MX2021013266A 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen. MX2021013266A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962840184P 2019-04-29 2019-04-29
US201962913314P 2019-10-10 2019-10-10
US201962924095P 2019-10-21 2019-10-21
PCT/US2020/030273 WO2020223232A1 (en) 2019-04-29 2020-04-28 Novel aav capsids and compositions containing same

Publications (1)

Publication Number Publication Date
MX2021013266A true MX2021013266A (es) 2021-11-17

Family

ID=73028724

Family Applications (3)

Application Number Title Priority Date Filing Date
MX2021013266A MX2021013266A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.
MX2021013267A MX2021013267A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.
MX2021013268A MX2021013268A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.

Family Applications After (2)

Application Number Title Priority Date Filing Date
MX2021013267A MX2021013267A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.
MX2021013268A MX2021013268A (es) 2019-04-29 2020-04-28 Nuevas capsides de aav y composiciones que las contienen.

Country Status (15)

Country Link
US (3) US20220204990A1 (es)
EP (3) EP3976078A4 (es)
JP (3) JP2022530656A (es)
KR (3) KR20220004681A (es)
CN (3) CN113853208A (es)
AU (3) AU2020264976A1 (es)
BR (3) BR112021021502A2 (es)
CA (3) CA3134379A1 (es)
CL (3) CL2021002768A1 (es)
CO (3) CO2021015924A2 (es)
IL (3) IL287492A (es)
MX (3) MX2021013266A (es)
PE (3) PE20212267A1 (es)
SG (3) SG11202111373YA (es)
WO (3) WO2020223232A1 (es)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2022016528A (es) 2020-06-17 2023-06-02 Univ Pennsylvania Composiciones y métodos para el tratamiento de pacientes de terapia génica.
JP2023543125A (ja) 2020-08-24 2023-10-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Glp-1受容体アゴニスト融合物をコードするウイルスベクター及び代謝性疾患の治療におけるその使用
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
JP2023548432A (ja) 2020-11-11 2023-11-16 ヨーロピアン モレキュラー バイオロジー ラボラトリー 遺伝子療法のための改変ウイルス粒子
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
IL303239A (en) * 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome
AU2022262771A1 (en) 2021-04-23 2023-11-02 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023168293A2 (en) * 2022-03-01 2023-09-07 The Trustees Of The University Of Pennsylvania Viral vector genome encoding an insulin fusion protein
WO2023168403A2 (en) * 2022-03-03 2023-09-07 The Trustees Of The University Of Pennsylvania Viral vectors encoding parathyroid hormone fusions and uses thereof in treating hypoparathyroidism
WO2023196893A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2024015966A2 (en) * 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2338900B1 (en) * 2001-11-13 2014-01-01 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) Cy.5 sequences, vectors containing the same and uses thereof
DK2292779T3 (en) * 2003-09-30 2017-02-27 Univ Pennsylvania ADENOASS-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME AND APPLICATIONS THEREOF
CN106906241B (zh) * 2005-04-07 2020-09-11 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
EP2016174A2 (en) * 2006-04-28 2009-01-21 The Trustees of the University of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
JP5963743B2 (ja) * 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Cnsターゲティングaavベクターおよびその使用方法
MX2016011585A (es) * 2014-03-10 2016-11-29 Uniqure Ip Bv Vectores aav mejorados adicionales producidos en celulas de insecto.
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
DK3872085T3 (da) * 2015-07-30 2023-04-03 Massachusetts Eye & Ear Infirmary Stam-virussekvenser og anvendelser heraf
US20180230489A1 (en) * 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
CA3002406A1 (en) * 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
US11326182B2 (en) * 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017267665C1 (en) * 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11192925B2 (en) * 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
WO2018200419A1 (en) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Viral vectors comprising engineered aav capsids and compositions containing the same
JOP20190269A1 (ar) * 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
US11512327B2 (en) * 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV

Also Published As

Publication number Publication date
WO2020223231A1 (en) 2020-11-05
PE20212267A1 (es) 2021-11-30
US20220204990A1 (en) 2022-06-30
CL2021002824A1 (es) 2022-05-27
EP3962507A1 (en) 2022-03-09
EP3976078A4 (en) 2023-08-30
JP2022530544A (ja) 2022-06-29
BR112021020545A2 (pt) 2022-01-04
CA3134468A1 (en) 2020-11-05
KR20220004681A (ko) 2022-01-11
SG11202111373YA (en) 2021-11-29
SG11202111375RA (en) 2021-11-29
CA3134507A1 (en) 2020-11-05
CN113853208A (zh) 2021-12-28
JP2022530656A (ja) 2022-06-30
JP2022530633A (ja) 2022-06-30
KR20220004695A (ko) 2022-01-11
BR112021021502A2 (pt) 2022-07-19
EP3976078A1 (en) 2022-04-06
CL2021002768A1 (es) 2022-05-27
CO2021015924A2 (es) 2021-11-30
KR20220004680A (ko) 2022-01-11
IL287498A (en) 2021-12-01
AU2020266523A1 (en) 2021-11-04
IL287491A (en) 2021-12-01
AU2020264975A1 (en) 2021-11-11
EP3976077A1 (en) 2022-04-06
CN113853209A (zh) 2021-12-28
EP3976077A4 (en) 2023-08-30
CL2021002811A1 (es) 2022-05-27
PE20212334A1 (es) 2021-12-14
BR112021020054A2 (pt) 2021-12-07
CN113853207A (zh) 2021-12-28
IL287492A (en) 2021-12-01
PE20220014A1 (es) 2022-01-11
US20220249705A1 (en) 2022-08-11
WO2020223232A1 (en) 2020-11-05
CO2021015916A2 (es) 2021-11-30
CO2021015928A2 (es) 2021-11-30
AU2020264976A1 (en) 2021-11-11
US20220220453A1 (en) 2022-07-14
MX2021013268A (es) 2021-11-17
CA3134379A1 (en) 2020-11-05
EP3962507A4 (en) 2023-10-11
WO2020223236A1 (en) 2020-11-05
SG11202111102SA (en) 2021-11-29
MX2021013267A (es) 2021-11-17

Similar Documents

Publication Publication Date Title
MX2021013267A (es) Nuevas capsides de aav y composiciones que las contienen.
MX2023001863A (es) Nuevas cápsides de aav y composiciones que las contienen.
EP3758724A4 (en) NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSID DEAMIDATION AND USES THEREOF
WO2019191701A8 (en) Novel recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
EP4303225A3 (en) Polyploid adeno-associated virus vectors and methods of making and using the same
EP3768695A4 (en) NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
EP4041755A4 (en) MODIFIED AAV CAPSIDS AND USES THEREOF
MX2021012311A (es) Variantes de capsides de aav para liberacion intravitrea.
MX2023005113A (es) Cápsides de aav y composiciones que las contienen.
MX2022012855A (es) Virus adenoasociado con cápside modificada geneticamente.
AU2020299026A8 (en) Methods and AAV vectors for in vivo transduction
JOP20220043A1 (ar) بروتين كابسيد vp1 معدل معزول من aav5
MX2021012489A (es) Lineas celulares productoras dise?adas geneticamente y metodos de elaboracion y uso de las mismas.
WO2019222314A8 (en) Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a
EA202192955A1 (ru) Новые капсиды aav и композиции, содержащие их
PH12020551319A1 (en) Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
AR120259A1 (es) Variantes de aav3b con rendimiento mejorado de la producción y tropismo hepático
WO2024015966A3 (en) Recombinant aav having aav clade d and clade e capsids and compositions containing same