Title:
ANTISENSE OLIGONUCLEOTIDE, COMPOSITIONS AND PHARMACEUTICAL FORMULATIONS THEREOF FOR EXON SKIPPING
Document Type and Number:
WIPO Patent Application WO/2024/067885
Kind Code:
A1
Abstract:
Provided are novel antisense oligonucleotides ("ASOs") that generate or promote exon skipping during pre-mRNA splicing, pharmaceutical compositions containing such ASOs, and their use. ASOs (e.g., bipartite ASOs) comprise a targeting sequence and a 5'-splice-site decoy sequence operably connected at the 5'and/or 3'end of the targeting sequence. The decoy sequence may have 5, 6, 7, 8, 9, 10, 11, 12 or 13 nucleotides in length, a portion of which is complementary to the single-strand 5'end of U1 snRNA. The targeting sequence hybridizes to a targeted region, such as an exon of interest, a flanking intron sequence upstream or downstream of the exon of interest, or an intron-exon junction upstream or downstream of the exon of interest.
Inventors:
HUA YIMIN (US)
FENG PENGCHAO (CN)
FENG PENGCHAO (CN)
Application Number:
PCT/CN2023/123640
Publication Date:
April 04, 2024
Filing Date:
October 09, 2023
Export Citation:
Assignee:
ASOCURA PHARMACEUTICALS SUZHOU CO LTD (CN)
International Classes:
C12N15/113; A61K31/7088
Domestic Patent References:
| WO2021089736A1 | 2021-05-14 |
Foreign References:
| CN114206352A | 2022-03-18 | |||
| US20130203836A1 | 2013-08-08 | |||
| US20170143847A1 | 2017-05-25 |
Other References:
CAZZELLA,V. ET AL.: "Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts", MOLECULAR THERAPY, vol. 20, no. 11, 11 September 2012 (2012-09-11), pages 2134 - 2142, XP009168733, DOI: 10.1038/mt.2012.178
VICKERS TIMOTHY A., SABRIPOUR MAHYAR, CROOKE STANLEY T.: "U1 adaptors result in reduction of multiple pre-mRNA species principally by sequestering U1snRNP", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, GB, vol. 39, no. 10, 1 May 2011 (2011-05-01), GB , pages e71 - e71, XP093100703, ISSN: 0305-1048, DOI: 10.1093/nar/gkr150
VICKERS TIMOTHY A., SABRIPOUR MAHYAR, CROOKE STANLEY T.: "U1 adaptors result in reduction of multiple pre-mRNA species principally by sequestering U1snRNP", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, GB, vol. 39, no. 10, 1 May 2011 (2011-05-01), GB , pages e71 - e71, XP093100703, ISSN: 0305-1048, DOI: 10.1093/nar/gkr150
Attorney, Agent or Firm:
COHORIZON INTELLECTUAL PROPERTY INC. (CN)
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