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1. WO2021030773 - EXTRACELLULAR VESICLE-NLRP3 ANTAGONIST

Publication Number WO/2021/030773
Publication Date 18.02.2021
International Application No. PCT/US2020/046556
International Filing Date 14.08.2020
IPC
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
C07D 307/64 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
307Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom
02not condensed with other rings
34having two or three double bonds between ring members or between ring members and non-ring members
56with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
64Sulfur atoms
A61K 31/341 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
335having oxygen as the only ring hetero atom, e.g. fungichromin
34having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide
341not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
A61K 31/7125 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61P 29/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
29Non-central analgesic, antipyretic or antiinflammatory agents, e.g antirheumatic agents; Non-steroidal antiinflammatory drugs (NSAIDs)
C07C 13/553 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
CACYCLIC OR CARBOCYCLIC COMPOUNDS
13Cyclic hydrocarbons containing rings other than, or in addition to, six-membered aromatic rings
28Polycyclic hydrocarbons or acyclic hydrocarbon derivatives thereof
32with condensed rings
54with three condensed rings
547at least one ring not being six-membered, the other rings being at the most six-membered
553Indacenes; Completely or partially hydrogenated indacenes
CPC
C07D 307/64
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
DHETEROCYCLIC COMPOUNDS
307Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom
02not condensed with other rings
34having two or three double bonds between ring members or between ring members and non-ring members
56with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
64Sulfur atoms
C12N 15/1138
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
1138against receptors or cell surface proteins
C12N 2310/11
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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
11Antisense
C12N 2310/315
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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
315Phosphorothioates
C12N 2310/321
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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
32of the sugar
3212'-O-R Modification
C12N 2310/3231
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
32of the sugar
323modified ring structure
3231having an additional ring, e.g. LNA, ENA
Applicants
  • CODIAK BIOSCIENCES, INC. [US]/[US]
Inventors
  • LIM, Joanne
  • KIRWIN, Katherine
  • BROOM, Wendy
  • SATHYANARAYANAN, Sriram
  • VERMA, Ajay
Agents
  • KIM, Ji Eun
  • BANOWIT, Donald R.
  • BEMBEN, Richard M.
  • BEZOS, Salvador M.
  • BLOCK, Daniel S.
  • BODENSTEIN, Matthew S.
  • CALVO, Paul A.
  • CAMARCE, Christian
  • CIMBALA, Michele A.
  • COLLER III, Richard D.
  • CONKLIN, Kyle E.
  • COVERT, John M.
  • DURKIN, Tracy-Gene
  • EISENBERG, Jason D.
  • ELLISON, Eldora L.
  • ESMOND, Robert W.
  • FEATHERSTONE, Donald A.
  • FITZSIMMONS, Jason A.
  • FRUEAUF, Jeremiah B.
  • GAJEWSKI, Daniel A.
  • GILLENTINE, Marsha Rose
  • GOLDSTEIN, Jorge A.
  • HELVEY, Jeffrey T.
  • HICKS, Ross G.
  • HOLOUBEK, Michelle K.
  • JACKMAN, Peter A.
  • KENTON JR., Lestin L.
  • LEE, Michael Q.
  • LONGSWORTH, Gaby L.
  • MILLONIG, Robert C.
  • MUTSCHELKNAUS, Joseph
  • NANNENGA-COMBS, Bonnie W.
  • POWERS III, R. Wilson
  • RAY, Michael B.
  • RICHARDSON, Ryan C.
  • RYGIEL, Mark W.
  • SHEA JR., Timothy J.
  • SOKOHL, Robert E.
  • SPECHT, Michael D.
  • STEFFE, Eric K.
  • STERLING, Deborah A.
  • STERNE, Robert Greene
  • GLENN, Dallin G.
  • HOLMAN, David H.
  • PHERO, Graham C.
Priority Data
62/886,87614.08.2019US
62/989,54113.03.2020US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) EXTRACELLULAR VESICLE-NLRP3 ANTAGONIST
(FR) ANTAGONISTE DE LA VÉSICULE EXTRACELLULAIRE-NLRP3
Abstract
(EN)
The present disclosure relates to extracellular vesicles, e.g., exosomes, comprising an NLRP3 antagonist. In some aspects, the NLRP3 antagonist comprises an antisense oligonucleotide (ASO). Also provided herein are methods for producing the exosomes and methods for using the exosomes to treat and/or prevent diseases or disorders.
(FR)
La présente invention concerne des vésicules extracellulaires, par exemple, des exosomes, comprenant un antagoniste NLRP3. Dans certains aspects, l'antagoniste de NLRP3 comprend un oligonucléotide antisens (ASO). L'invention concerne également des procédés de production des exosomes et des procédés d'utilisation des exosomes pour traiter et/ou prévenir des maladies ou des troubles.
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