Title:
MODIFIED SINA MOLECULES, METHODS AND USES THEREOF
Document Type and Number:
WIPO Patent Application WO/2024/028775
Kind Code:
A3
Abstract:
The present disclosure relates to a double stranded short interfering nucleic acid (siNA) agent for use in medicine, in particular for use in the treatment of ophthalmic diseases comprising an antisense ribonucleotide sequence arranged to base-pair to a substantially complementary sense ribonucleotide sequence; wherein all of the nucleotides of the sense strand and all of the nucleotides of the antisense strand comprise a 2'-O-methyl modification or a 2'-fluoro modification; wherein both the sense strand and the antisense strand independently comprise at least one phosphorothioate internucleotide linkage; wherein the antisense ribonucleotide sequence is substantially complementary to a sense ribonucleotide sequence except in up to 6 nucleotides; wherein the sense strand comprises 15 to 21 nucleotides; wherein each nucleotide or each of the first 18 contiguous nucleotides in the 5'-3' direction comprise a modification that is different from the modification of the adjacent nucleotide; and wherein the first modification in the antisense strand is a 2'-O-methyl modification.
Inventors:
SOARES DA SILVA PATRÍCIO (PT)
Application Number:
PCT/IB2023/057805
Publication Date:
April 11, 2024
Filing Date:
August 01, 2023
Export Citation:
Assignee:
PHYZAT BIOPHARMACEUTICALS LDA (PT)
International Classes:
C12N15/113; A61K31/712; A61K31/7125; A61K31/713; A61P27/02; A61P27/06
Domestic Patent References:
| WO2022107106A2 | 2022-05-27 | |||
| WO2022072356A1 | 2022-04-07 | |||
| WO2016161388A1 | 2016-10-06 |
Other References:
LI A.-J. ET AL: "Simultaneous Silencing of Npy and Dbh Expression in Hindbrain A1/C1 Catecholamine Cells Suppresses Glucoprivic Feeding", THE JOURNAL OF NEUROSCIENCE, vol. 29, no. 1, 7 January 2009 (2009-01-07), US, pages 280 - 287, XP055909519, ISSN: 0270-6474, DOI: 10.1523/JNEUROSCI.4267-08.2009
Attorney, Agent or Firm:
PATENTREE (PT)
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