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1. WO2020002380 - GENE THERAPY

Publication Number WO/2020/002380
Publication Date 02.01.2020
International Application No. PCT/EP2019/066915
International Filing Date 25.06.2019
IPC
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435
from animals; from humans
46
from vertebrates
47
from mammals
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
10
Processes for the isolation, preparation or purification of DNA or RNA
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10
Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
86
Viral vectors
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
Q
MEASURING OR TESTING PROCESSES INVOLVING ENZYMES OR MICRO-ORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1
Measuring or testing processes involving enzymes or micro-organisms; Compositions therefor; Processes of preparing such compositions
68
involving nucleic acids
C07K 14/47 (2006.01)
C12N 15/10 (2006.01)
A61K 48/00 (2006.01)
C12N 5/10 (2006.01)
C12N 15/86 (2006.01)
C12Q 1/68 (2018.01)
CPC
A61K 48/005
C07K 14/4746
C12N 15/102
C12N 15/86
C12N 2310/20
C12N 2750/14143
Applicants
  • OSPEDALE SAN RAFFAELE S.R.L [IT/IT]; Via Olgettina 60 20132 Milan, IT
  • FONDAZIONE TELETHON [IT/IT]; Via Varese 16B 00185 Rome, IT
Inventors
  • GENOVESE, Pietro; IT
  • SCHIROLI, Giulia; IT
  • NALDINI, Luigi; IT
  • JACOB, Aurelien; IT
  • FERRARI, Samuele; IT
Agents
  • O'BRIEN, Simon; GB
Priority Data
18305807.225.06.2018EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) GENE THERAPY
(FR) THÉRAPIE GÉNIQUE
Abstract
(EN)
An agent which promotes homology directed DNA repair for use in haematopoietic stem and/or progenitor cell gene therapy, wherein said haematopoietic stem cells are gene edited. Use of an agent which promotes homology directed DNA repair, for increasing the survival and/or engraftment of gene edited haematopoietic stemand/or progenitorcells or for increasing the efficiency of gene editing of haematopoietic stem and/or progenitor cells.
(FR)
L'invention concerne un agent qui favorise une réparation d'ADN dirigée par homologie pour une utilisation dans une thérapie génique de cellules souches et/ou progénitrices hématopoïétiques, lesdites cellules souches hématopoïétiques étant génétiquement éditées. L'utilisation d'un agent qui favorise la réparation d'ADN dirigée par homologie, pour augmenter la survie et/ou la prise de greffe de cellules souches hématopoïétiques et/ou de cellules souches hématopoïétiques génétiquement éditées ou pour augmenter l'efficacité de l'édition génique de cellules souches hématopoïétiques et/ou progénitrices.
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