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1. WO2021211641 - ENHANCING AAV-MEDIATED DELIVERY AND TRANSDUCTION WITH POLYVINYL ALCOHOL

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Publication Number WO/2021/211641
Publication Date 21.10.2021
International Application No. PCT/US2021/027169
International Filing Date 13.04.2021
IPC
A61K 48/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/075 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
01DNA viruses
075Adenoviridae
C07K 14/015 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005from viruses
01DNA viruses
015Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
C12N 7/00 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
7Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
CPC
A61K 48/0025
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
0008characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
0025wherein the non-active part clearly interacts with the delivered nucleic acid
C12N 15/86
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
C12N 2750/14143
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
2750ssDNA Viruses
00011ssDNA Viruses
14011Parvoviridae
14111Dependovirus, e.g. adenoassociated viruses
14141Use of virus, viral particle or viral elements as a vector
14143viral genome or elements thereof as genetic vector
Applicants
  • UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INCORPORATED [US]/[US]
Inventors
  • QING, Keyun
  • SRIVASTAVA, Arun
Agents
  • GREENWALD, Eric, P.
  • ALAM, Saad
  • ATTISHA, Michael, J.
  • BAKER, C., Hunter
  • AMUNDSEN, Eric, L.
Priority Data
63/010,03014.04.2020US
63/024,01513.05.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) ENHANCING AAV-MEDIATED DELIVERY AND TRANSDUCTION WITH POLYVINYL ALCOHOL
(FR) AMÉLIORATION DE L'ADMINISTRATION ET DE LA TRANSDUCTION MÉDIÉES PAR AAV AVEC DE L'ALCOOL POLYVINYLIQUE
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Abstract
(EN) Disclosed herein are novel methods for administration of rAAV particles having enhanced transduction properties, comprising the pre-incubation and co-administration of AAV capsids and polyvinyl alcohol (PVA). The disclosed methods of rAAV particle administration have improved efficiency in transducing mammalian liver and hematopoetic cells in vivo. The disclosed methods are suitable for use with a variety of AAV capsid serotypes and presudotypes and improve vector potency, thus lowering the concentration of rAAV particle required to achieve the desired effect. Further disclosed herein are buffers for storage and manufacturing of rAAV particles comprising PVA.
(FR) L'invention concerne de nouveaux procédés d'administration de particules de rAAV ayant des propriétés de transduction améliorées, consistant à incuber préalablement et à co-administrer des capsides d'AAV et de l'alcool polyvinylique (PVA). Les procédés décrits de l'administration de particules de rAAV ont une efficacité améliorée dans la transduction de cellules hépatiques et hématopoïétiques de mammifères in vivo. Les procédés de l'invention sont appropriés pour une utilisation avec divers sérotypes et présudotypes de capsides d'AAV présumés et améliorent la puissance de vecteur, réduisant ainsi la concentration de particules de rAAV requise pour obtenir l'effet souhaité. L'invention concerne en outre des tampons pour le stockage et la fabrication de particules de rAAV comprenant du PVA.
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