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1. WO2021062419 - NOVEL VECTORS AND USES THEREOF

Publication Number WO/2021/062419
Publication Date 01.04.2021
International Application No. PCT/US2020/053353
International Filing Date 30.09.2020
IPC
C12N 15/86 2006.01
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
A61P 31/14 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
12Antivirals
14for RNA viruses
C12N 7/00 2006.01
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
A61P 31/12 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
31Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
12Antivirals
A61K 48/00 2006.01
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
Applicants
  • BURKE, John, Douglas [US]/[US]
  • CANDOTTI, Fabio [CH]/[CH]
Inventors
  • BURKE, John, Douglas
  • CANDOTTI, Fabio
Common Representative
  • BURKE, John, Douglas
Priority Data
62/907,71829.09.2019US
63/038,88414.06.2020US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) NOVEL VECTORS AND USES THEREOF
(FR) NOUVEAUX VECTEURS ET UTILISATIONS ASSOCIÉES
Abstract
(EN)
Provided is a viral particle comprising a genomic RNA suitable for delivering a polynucleotide sequence of interest (SOI) into a cell and/or a subject. The genomic RNA comprises, from 5' to 3': (a) the SOI replacing the upstream R, (b) a U5, (c) a primer binding site (PBS), (d) an encapsidation signal (Psi), (e) polypurine tract(s) (PPT), and (f) a U3. The SOI preferably takes the form of single-stranded DNA or a DNA-RNA hybrid after initiation of reverse transcription. Additionally provided are polynucleotides, vectors, cells, components, compositions, kits, methods and uses of the viral particle.
(FR)
L'invention concerne une particule virale comprenant un ARN génomique approprié pour administrer une séquence polynucléotidique d'intérêt (SOI) dans une cellule et/ou un sujet. L'ARN génomique comprend, de l'extrémité 5' à l'extrémité 3' : (a) la SOI remplaçant le R amont, (b) un U5, (c) un site de liaison d'amorce (PBS), (d) un signal d'encapsidation (Psi), (e) une ou plusieurs voies de polypurine (PPT) et (f) un U3. La SOI se présente de préférence sous la forme d'un ADN simple brin ou d'un hybride ADN-ARN après initiation de la transcription inverse. L'invention concerne en outre des polynucléotides, des vecteurs, des cellules, des constituants, des compositions, des kits, des méthodes et des utilisations de la particule virale.
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