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1. WO2020198509 - MODIFIED OLIGONUCLEOTIDES WITH INCREASED STABILITY

Publication Number WO/2020/198509
Publication Date 01.10.2020
International Application No. PCT/US2020/025017
International Filing Date 26.03.2020
IPC
A61K 31/7084 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
7084Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
A61K 31/7088 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
A61K 31/7105 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
7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 31/711 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
711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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
Applicants
  • UNIVERSITY OF MASSACHUSETTS [US]/[US]
Inventors
  • KHVOROVA, Anastasia
  • ROUX, Loïc Maurice René Jean
  • YAMADA, Ken
Agents
  • VELEMA, James H.
  • COUGHLIN, Sean M.
  • DECONTI, Giulio A.
  • SPELLBERG, Michael
  • STEELE, Alan
  • STONE-HULSLANDER, Judith
  • TRINQUE, Brian C.
  • LABEOTS, Laura A.
Priority Data
62/824,13626.03.2019US
62/826,45429.03.2019US
62/864,79221.06.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) MODIFIED OLIGONUCLEOTIDES WITH INCREASED STABILITY
(FR) OLIGONUCLÉOTIDES MODIFIÉS À STABILITÉ ACCRUE
Abstract
(EN)
This disclosure relates to novel modified oligonucleotides. Novel modified siRNA are also provided. In an aspect of the invention, the oligonucleotides and siRNA provided herein can be incorporated into a CRISPR/Cas system. In one embodiment, the present invention provides novel RNA silencing agents (e.g., siRNA and shRNAs), methods of making said RNA silencing agents, and methods (e.g., research and/or therapeutic methods) for using said improved RNA silencing agents (or portions thereof) for RNA silencing of for example, an ApoE, C9ORF72, or Htt protein.
(FR)
L'invention concerne de nouveaux oligonucléotides modifiés. L'invention concerne également de nouveaux ARNsi modifiés.
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