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1. WO2020093061 - THERAPEUTIC METHODS

Publication Number WO/2020/093061
Publication Date 07.05.2020
International Application No. PCT/US2019/059711
International Filing Date 04.11.2019
IPC
A61K 47/50 2017.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
50the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
A61K 47/32 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
32Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers
A61K 47/34 2017.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
34Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
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 15/87 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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
Applicants
  • GENEVANT SCIENCES GMBH [CH]/[CH]
Inventors
  • HEYES, James
  • HOLLAND, Richard J.
  • JUDGE, Adam
  • LAM, Kieu Mong
Agents
  • MALEN, Peter L.
  • VIKSNINS, Ann S.
  • HARRIS, Robert J.
  • MICKELSON, John W.
  • KRUEGER, Katherine A.D.
  • PADYS, Danny J.
  • ANDRUS, Alex
  • NIU, Lin
Priority Data
62/755,19602.11.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) THERAPEUTIC METHODS
(FR) PROCÉDÉS THÉRAPEUTIQUES
Abstract
(EN)
The invention provides methods and compositions for delivering a nucleic acid to a cell or the cytosol of the target cell. The method includes contacting the cell with, 1) a membrane-destabilizing polymer; and 2) a nucleic acid conjugate. The nucleic acid conjugate includes a targeting ligand bound to an optional linker and a nucleic acid.
(FR)
L'invention concerne des procédés et des compositions pour administrer un acide nucléique dans une cellule ou dans le cytosol de la cellule cible. Le procédé consiste à mettre la cellule en contact avec 1) un polymère déstabilisateur de membrane, et 2) un conjugué d'acide nucléique. Le conjugué d'acide nucléique comprend un ligand de ciblage lié à un lieur facultatif et un acide nucléique.
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