Title:
OLIGONUCLEOTIDE MODULATORS ACTIVATING UTROPHIN EXPRESSION
Document Type and Number:
WIPO Patent Application WO/2023/138502
Kind Code:
A1
Abstract:
Provided is an oligonucleotide modulator for preventing or treating dystrophin-deficient-related disorders (DDD) including Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). 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 UTRN.
Inventors:
KANG MOORIM (CN)
PLACE ROBERT (CN)
LI LONGCHENG (CN)
PLACE ROBERT (CN)
LI LONGCHENG (CN)
Application Number:
PCT/CN2023/072076
Publication Date:
July 27, 2023
Filing Date:
January 13, 2023
Export Citation:
Assignee:
RACTIGEN THERAPEUTICS (CN)
International Classes:
C12N15/113; A61K31/713; A61P21/00; C12N15/63
Domestic Patent References:
| WO2021230385A1 | 2021-11-18 |
Foreign References:
| US20150099791A1 | 2015-04-09 | |||
| US20170044540A1 | 2017-02-16 | |||
| US20120046345A1 | 2012-02-23 | |||
| CN102459597A | 2012-05-16 |
Other References:
LONG-CHENG LI , STEVEN T OKINO , HONG ZHAO , DEEPA POOKOT , ROBERT F PLACE , SHINJI URAKAMI , HIDEKI ENOKIDA , RAJIV OAHIYA: "Small dsRNAs induce transcriptional activation in human cells", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 103, no. 46, 14 November 2006 (2006-11-14), pages 17337 - 17342, XP002589998, ISSN: 0027-8424, DOI: 10.1073/PNAS.0607015103
Attorney, Agent or Firm:
SHANGHAI PATENT & TRADEMARK LAW OFFICE , LLC (CN)
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