Title:
CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE
Document Type and Number:
Japanese Patent JP2023116506
Kind Code:
A
Abstract:
To provide techniques for facilitating uptake and distribution of antisense compounds into other cell types, such as CNS tissue or muscle tissue.SOLUTION: The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, where the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound. In certain embodiments, the compounds disclosed herein are useful for modulating the expression of extra-hepatic target nucleic acids.SELECTED DRAWING: None
Inventors:
MICHAEL OESTERGAARD
SETH PUNIT P
FRANK RIGO
CHRISSA A DWYER
SETH PUNIT P
FRANK RIGO
CHRISSA A DWYER
Application Number:
JP2023085094A
Publication Date:
August 22, 2023
Filing Date:
May 24, 2023
Export Citation:
Assignee:
IONIS PHARMACEUTICALS INC
International Classes:
C12N15/113; A61K31/7088; A61P1/16; A61P21/00
Attorney, Agent or Firm:
Osamu Yamamoto
Toru Miyamae
Junichi Matsuo
Reiko Izumitani
Toru Miyamae
Junichi Matsuo
Reiko Izumitani
Previous Patent: Polymer having fluorine-containing aniline structure
Next Patent: Compositions and methods for treating cancer
Next Patent: Compositions and methods for treating cancer