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1. WO2020113034 - AAV VIRAL VECTORS AND USES THEREOF

Publication Number WO/2020/113034
Publication Date 04.06.2020
International Application No. PCT/US2019/063649
International Filing Date 27.11.2019
IPC
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
C12N 15/864 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
864Parvoviral vectors
CPC
A61K 48/005
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
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
C07K 14/4702
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
4701not used
4702Regulators; Modulating activity
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
  • AVEXIS, INC. [US]/[US]
Inventors
  • HATFIELD, James Michael
  • HODGE, Robert Emil
  • FELTNER, Douglas
  • BALLEYDIER, Joseph
  • MERIGGIOLI, Matthew
  • KASPAR, Brian K.
  • KASPAR, Allan Arman
Agents
  • JACOBSTEIN, Jeffrey M.
Priority Data
62/773,89430.11.2018US
62/835,24217.04.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) AAV VIRAL VECTORS AND USES THEREOF
(FR) VECTEURS VIRAUX AAV ET LEURS UTILISATIONS
Abstract
(EN)
Disclosed herein are compositions comprising AAV9 viral vectors and methods of using them to treat SMA patients, e.g., Type II and Type III Spinal Muscular Atrophy (SMA) patients.
(FR)
L'invention concerne des compositions contenant des vecteurs viraux AAV9 et leurs procédés d'utilisation pour traiter des patients atteints d'une AMS, par exemple, des patients atteints d'une amyotrophie musculaire spinale (AMS) de type II et de type III.
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