Title:
OLIGONUCLEOTIDE DELIVERY AGENTS, PHARMACEUTICAL COMPOSITIONS AND METHODS USING THE SAME
Document Type and Number:
WIPO Patent Application WO/2024/002045
Kind Code:
A1
Abstract:
Provided is an oligonucleotide delivery agent comprising at least one lipid moiety comprising at least one linear alkyl group or linear alkylenyl group directly or indirectly conjugated to a single-stranded oligonucleotide, which can be delivered to various subjects, such as muscle, heart, liver, etc., with enhanced efficiency and thus achieving improved expression regulation of target genes. A pharmaceutical composition comprising the oligonucleotide delivery agent and a method of modulating the expression of a target gene by using the same are also provided.
Inventors:
GAN ZUBAO (CN)
LI LONGCHENG (CN)
KANG MOORIM (CN)
LI LONGCHENG (CN)
KANG MOORIM (CN)
Application Number:
PCT/CN2023/102618
Publication Date:
January 04, 2024
Filing Date:
June 27, 2023
Export Citation:
Assignee:
AOAN BIOSCIENCES (CN)
International Classes:
A61K31/712; C07H19/00; C12N15/113
Domestic Patent References:
| WO2020225371A1 | 2020-11-12 | |||
| WO2019243430A1 | 2019-12-26 | |||
| WO2018057430A1 | 2018-03-29 |
Foreign References:
| US20040048826A1 | 2004-03-11 |
Other References:
ZHANG YUEBAO, SUN CHANGZHEN, WANG CHANG, JANKOVIC KATARINA E., DONG YIZHOU: "Lipids and Lipid Derivatives for RNA Delivery", CHEMICAL REVIEWS, AMERICAN CHEMICAL SOCIETY, US, vol. 121, no. 20, 27 October 2021 (2021-10-27), US , pages 12181 - 12277, XP093122345, ISSN: 0009-2665, DOI: 10.1021/acs.chemrev.1c00244
MOUNA RAOUANE, DIDIER DESMAELE, MARIE GILBERT-SIRIEIX, CLAIRE GUEUTIN, FATIMA ZOUHIRI, CLAUDIE BOURGAUX, ELISE LEPELTIER, RUXANDRA: "Synthesis, Characterization, and in Vivo Delivery of siRNA-Squalene Nanoparticles Targeting Fusion Oncogene in Papillary Thyroid Carcinoma", JOURNAL OF MEDICINAL CHEMISTRY, AMERICAN CHEMICAL SOCIETY, vol. 54, no. 12, 23 June 2011 (2011-06-23), pages 4067 - 4076, XP055100583, ISSN: 00222623, DOI: 10.1021/jm2000272
NIKAN, M., ET AL.: "Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain", MOLECULAR THERAPY—NUCLEIC ACIDS, vol. 5, 9 August 2016 (2016-08-09), XP055623574, DOI: 10.1038/mtna.2016.50
MOUNA RAOUANE, DIDIER DESMAELE, MARIE GILBERT-SIRIEIX, CLAIRE GUEUTIN, FATIMA ZOUHIRI, CLAUDIE BOURGAUX, ELISE LEPELTIER, RUXANDRA: "Synthesis, Characterization, and in Vivo Delivery of siRNA-Squalene Nanoparticles Targeting Fusion Oncogene in Papillary Thyroid Carcinoma", JOURNAL OF MEDICINAL CHEMISTRY, AMERICAN CHEMICAL SOCIETY, vol. 54, no. 12, 23 June 2011 (2011-06-23), pages 4067 - 4076, XP055100583, ISSN: 00222623, DOI: 10.1021/jm2000272
NIKAN, M., ET AL.: "Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain", MOLECULAR THERAPY—NUCLEIC ACIDS, vol. 5, 9 August 2016 (2016-08-09), XP055623574, DOI: 10.1038/mtna.2016.50
Attorney, Agent or Firm:
SHANGHAI PATENT & TRADEMARK LAW OFFICE, LLC (CN)
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