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1. WO2020112195 - COMPOSITIONS, TECHNOLOGIES AND METHODS OF USING PLERIXAFOR TO ENHANCE GENE EDITING

Publication Number WO/2020/112195
Publication Date 04.06.2020
International Application No. PCT/US2019/049018
International Filing Date 30.08.2019
IPC
C12N 9/22 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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
C12N 15/113 2010.01
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
CPC
A61K 31/395
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
A61K 45/06
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
45Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
C12N 15/113
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
C12N 2310/152
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
2310Structure or type of the nucleic acid
10Type of nucleic acid
15Nucleic acids forming more than 2 strands, e.g. TFOs
152on a single-stranded target, e.g. fold-back TFOs
C12N 2310/3181
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
2310Structure or type of the nucleic acid
30Chemical structure
31of the backbone
318where the PO2 is completely replaced, e.g. MMI or formacetal
3181Peptide nucleic acid, PNA
C12N 2320/31
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
2320Applications; Uses
30Special therapeutic applications
31Combination therapy
Applicants
  • YALE UNIVERSITY [US]/[US]
  • TRUCODE GENE REPAIR, INC. [US]/[US]
Inventors
  • QUIJANO, Elias
  • SALTZMAN, W. Mark
  • GLAZER, Peter
  • COULL, James M.
  • FORDYCE, Marshall
  • SIM, Derek
Agents
  • RAYMOND, Adam S.
  • SHYNTUM, Yvonne Y.
  • YONGYE, Austin B.
  • MONHEIT, Rivka D.
  • HODGES, Robert A.
  • PABST, Patrea L.
  • SARGSYAN, S. Anna
  • ZUNIGA, Carlos A.
  • CALDERON MOLINA, Angie D.
  • XU, Xiaojin
Priority Data
62/773,97730.11.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS, TECHNOLOGIES AND METHODS OF USING PLERIXAFOR TO ENHANCE GENE EDITING
(FR) COMPOSITIONS, TECHNOLOGIES ET PROCÉDÉS D'UTILISATION DE PLÉRIXAFOR POUR AMÉLIORER L'ÉDITION DE GÈNES
Abstract
(EN)
Compositions, technologies and methods for improved gene editing are disclosed. In a preferred method, gene editing involves use of plerixafor as an agent to enhance targeted editing by a gene editing technology, such as a PNA, in combination with a donor oligonucleotide. Genomic modification occurs at a higher frequency when cells are contacted with plerixafor, gene editing technology and donor oligonucleotide, as compared to the absence of the plerixafor. The methods are suitable for in vivo, in vitro or ex vivo approaches to gene editing. Cells modified according to the disclosed methods can be administered to a subject in need thereof in an effective amount to treat a symptom of a disease or disorder. Also disclosed are nanoparticle compositions for intracellular delivery of the gene editing technologies.
(FR)
La présente invention concerne des compositions, des techniques et des procédés permettant d'améliorer l'édition de gènes. Dans un procédé préféré, l'édition de gènes implique l'utilisation de Plérixafor en tant qu'agent pour améliorer l'édition ciblée à l'aide une technique d'édition de gènes, telle que l'utilisation d'un ANP, en combinaison avec un oligonucléotide donneur. La modification génomique se produit à une fréquence plus élevée lorsque les cellules sont mises en contact avec du Plérixafor, une technique d'édition de gènes et un oligonucléotide donneur, par comparaison à l'absence du Plérixafor. Les procédés sont appropriés pour des approches in vivo, in vitro ou ex vivo pour l'édition de gènes. Les cellules modifiées selon les procédés décrits peuvent être administrées à un sujet en ayant besoin en une quantité efficace pour traiter un symptôme d'une maladie ou d'un trouble. L'invention concerne également des compositions de nanoparticules pour l'administration intracellulaire des techniques d'édition de gènes.
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