Title:
OLIGONUCLEOTIDE MODULATORS ACTIVATING COMPLEMENT FACTOR H EXPRESSION
Document Type and Number:
WIPO Patent Application WO/2024/001172
Kind Code:
A1
Abstract:
Provided is an oligonucleotide modulator for preventing or treating complementary factor H deficiency (CFHD) and use thereof. The oligonucleotide modulator comprises a sense nucleic acid strand and an antisense nucleic acid strand, wherein the sense nucleic acid strand and the antisense nucleic acid strand are independently an oligonucleotide strand of 16 to 35 nucleotides in length, in which one nucleotide strand has at least 75%base homology or complementarity to a target selected from a promoter region of a target gene CFH. Also provided is a pharmaceutical composition comprising the oligonucleotide modulator disclosed herein and optionally, a pharmaceutically acceptable carrier, and a method for up-regulating the expression of a target gene in cells and methods for preventing or treating a disorder induced by insufficient expression of a target gene with the oligonucleotide modulator or the pharmaceutical composition comprising the oligonucleotide modulator disclosed herein.
Inventors:
KANG MOORIM (CN)
LI LONGCHENG (CN)
LI LONGCHENG (CN)
Application Number:
PCT/CN2023/072927
Publication Date:
January 04, 2024
Filing Date:
January 18, 2023
Export Citation:
Assignee:
RACTIGEN THERAPEUTICS (CN)
International Classes:
C12N15/113; A61K31/713; A61K48/00; A61P3/00; C07H21/04; C12N15/10
Domestic Patent References:
| WO2016170348A2 | 2016-10-27 | |||
| WO2021026476A1 | 2021-02-11 | |||
| WO2007078599A2 | 2007-07-12 |
Foreign References:
| US20200208152A1 | 2020-07-02 | |||
| US20200255827A1 | 2020-08-13 | |||
| CN110959042A | 2020-04-03 |
Other References:
RAM,S. ET AL.: "Infections of people with complement deficiencies and patients who have undergone splenectomy", CLIN.MICROBIOL.REV., vol. 23, no. 4, 31 October 2021 (2021-10-31), pages 740 - 780, XP055339676, DOI: 10.1128/CMR.00048-09
Attorney, Agent or Firm:
SHANGHAI PATENT & TRADEMARK LAW OFFICE , LLC (CN)
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