Title:
CATIONIC LIPID ANALOG, AND COMPOSITION AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2023/142167
Kind Code:
A1
Abstract:
A cationic lipid analog, and a composition and use thereof. Aldehyde compounds, amine compounds, carboxylic acid compounds, and isonitrile compounds are used as starting materials, by means of an Ugi reaction, for synthesizing the cationic lipid analog. The obtained cationic lipid analog forms a lipid nanoparticle-like composition with a sterol, a helper lipid, and a polyethylene glycol lipid derivative. The composition can be used for the delivery of drugs including micromolecule drugs, nucleic acid drugs such as mRNA, DNA, and siRNA, protein/polypeptide drugs, and gene-editing complexes such as mRNA/sgRNA and Cas9/sgRNA. By means of different routes of administration, mRNA expression can be achieved in different organs of an animal body, and the application requirements of mRNA therapies, nucleic acid vaccines, gene editing, and the like can be met.
Inventors:
LIU ZHIJIA (CN)
CHEN YONGMING (CN)
HE ZEPENG (CN)
LE ZHICHENG (CN)
CHEN YONGMING (CN)
HE ZEPENG (CN)
LE ZHICHENG (CN)
Application Number:
PCT/CN2022/075911
Publication Date:
August 03, 2023
Filing Date:
February 10, 2022
Export Citation:
Assignee:
GUANGZHOU LIDE BIOMEDICINE TECH CO LTD (CN)
International Classes:
A61K47/18; A61K47/22; A61K48/00; C07C231/06; C07C237/22; C07D295/13
Domestic Patent References:
| WO2022170833A1 | 2022-08-18 | |||
| WO2022170834A1 | 2022-08-18 | |||
| WO2022170835A1 | 2022-08-18 | |||
| WO2003082809A1 | 2003-10-09 |
Foreign References:
| CN114904002A | 2022-08-16 | |||
| CN114904003A | 2022-08-16 | |||
| CN114904004A | 2022-08-16 | |||
| CN103881790A | 2014-06-25 | |||
| KR20140069931A | 2014-06-10 | |||
| US3323884A | 1967-06-06 | |||
| CN102665761A | 2012-09-12 | |||
| CN101674853A | 2010-03-17 | |||
| CN104684543A | 2015-06-03 |
Other References:
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FURUTA TOMOYUKI, MIZUKAMI YUYA, ASANO LISA, KOTAKE KENJIRO, ZIEGLER SLAVA, YOSHIDA HIROKI, WATANABE MIZUKI, SATO SHIN-ICHI, WALDMA: "Nutrient-Based Chemical Library as a Source of Energy Metabolism Modulators", ACS CHEMICAL BIOLOGY, vol. 14, no. 9, 20 September 2019 (2019-09-20), pages 1860 - 1865, XP093080960, ISSN: 1554-8929, DOI: 10.1021/acschembio.9b00444
DOUY ANDRÉ, GALLOT BERNARD: "New Amphipathic Lipopeptides. 1. Synthesis and Mesomorphic Structures of Lipopeptides with Polysarcosine Peptidic Chains.", MAKROMOL. CHEM AND PHYSICS, WILEY, vol. 187, no. 3, 1 March 1986 (1986-03-01), pages 465 - 483, XP093080961, ISSN: 0025-116X, DOI: 10.1002/macp.1986.021870301
PREVIDI DANIEL, RODRIGUES STEPHANIE, COELHO MIKE, CANDIDO ANA CAROLINA, MAGALHÃES LIZANDRA, DONATE PAULO: "Synthesis and Antileishmanial Activity of Some Functionalized Peptoids", JOURNAL OF THE BRAZILIAN CHEMICAL SOCIETY, SOCIEDADE BRASILEIRA DE QUíMICA, SAO PAULO, BR, vol. 30, no. 6, 7 February 2019 (2019-02-07), Sao Paulo, BR , pages 1334 - 1340, XP093080964, ISSN: 0103-5053, DOI: 10.21577/0103-5053.20190023
LEACH KATHRYN E., PEDROSA HERMENEGILDO N., CARLSON LISA J., KRAUSS TODD D.: "Fluorescent Carbon Nanotubes in Cross-Linked Micelles", CHEMISTRY OF MATERIALS, AMERICAN CHEMICAL SOCIETY, US, vol. 21, no. 3, 10 February 2009 (2009-02-10), US , pages 436 - 438, XP093080972, ISSN: 0897-4756, DOI: 10.1021/cm800605p
DATABASE Registry 26 February 2006 (2006-02-26), "Octadecanamide, N,N'-[oxybis[(methylenecarbonylimino)ethylene]]bis[N-2- hydroxyethyl- (5CI) (CA INDEX NAME)", XP093080982, retrieved from STNext Database accession no. 875225-20-0
GALETTI, M.D. ET AL.: "Multicomponent Synthesis of Acylated Short Peptoids with Antifungal Activity against Plant Pathogens.", MOLECULAR DIVERSITY., vol. 16, no. 1, 16 September 2011 (2011-09-16), XP035031341, DOI: 10.1007/s11030-011-9334-1
CHRISMENT J., DELPUECH J.-J.: "Proton Transfers on Amphiphilic Molecules. A Nonionic Alkyllipopeptide in Dimethyl Sulfoxide", LANGMUIR, AMERICAN CHEMICAL SOCIETY, US, vol. 12, no. 10, 1 January 1996 (1996-01-01), US , pages 2441 - 2445, XP093080983, ISSN: 0743-7463, DOI: 10.1021/la950786s
MOLLA MIJANUR R., CHAKRABORTY SHRADDHA, MUNOZ−SAGREDO LEONEL, DRECHSLER MARKUS, ORIAN−ROUSSEAU VÉRONIQUE, LEVKIN PAVEL A.: "Combinatorial Synthesis of a Lipidoid Library by Thiolactone Chemistry: In Vitro Screening and In Vivo Validation for siRNA Delivery", BIOCONJUGATE CHEMISTRY, AMERICAN CHEMICAL SOCIETY, US, vol. 31, no. 3, 18 March 2020 (2020-03-18), US , pages 852 - 860, XP093080665, ISSN: 1043-1802, DOI: 10.1021/acs.bioconjchem.0c00013
FURUTA TOMOYUKI, MIZUKAMI YUYA, ASANO LISA, KOTAKE KENJIRO, ZIEGLER SLAVA, YOSHIDA HIROKI, WATANABE MIZUKI, SATO SHIN-ICHI, WALDMA: "Nutrient-Based Chemical Library as a Source of Energy Metabolism Modulators", ACS CHEMICAL BIOLOGY, vol. 14, no. 9, 20 September 2019 (2019-09-20), pages 1860 - 1865, XP093080960, ISSN: 1554-8929, DOI: 10.1021/acschembio.9b00444
DOUY ANDRÉ, GALLOT BERNARD: "New Amphipathic Lipopeptides. 1. Synthesis and Mesomorphic Structures of Lipopeptides with Polysarcosine Peptidic Chains.", MAKROMOL. CHEM AND PHYSICS, WILEY, vol. 187, no. 3, 1 March 1986 (1986-03-01), pages 465 - 483, XP093080961, ISSN: 0025-116X, DOI: 10.1002/macp.1986.021870301
PREVIDI DANIEL, RODRIGUES STEPHANIE, COELHO MIKE, CANDIDO ANA CAROLINA, MAGALHÃES LIZANDRA, DONATE PAULO: "Synthesis and Antileishmanial Activity of Some Functionalized Peptoids", JOURNAL OF THE BRAZILIAN CHEMICAL SOCIETY, SOCIEDADE BRASILEIRA DE QUíMICA, SAO PAULO, BR, vol. 30, no. 6, 7 February 2019 (2019-02-07), Sao Paulo, BR , pages 1334 - 1340, XP093080964, ISSN: 0103-5053, DOI: 10.21577/0103-5053.20190023
LEACH KATHRYN E., PEDROSA HERMENEGILDO N., CARLSON LISA J., KRAUSS TODD D.: "Fluorescent Carbon Nanotubes in Cross-Linked Micelles", CHEMISTRY OF MATERIALS, AMERICAN CHEMICAL SOCIETY, US, vol. 21, no. 3, 10 February 2009 (2009-02-10), US , pages 436 - 438, XP093080972, ISSN: 0897-4756, DOI: 10.1021/cm800605p
DATABASE Registry 26 February 2006 (2006-02-26), "Octadecanamide, N,N'-[oxybis[(methylenecarbonylimino)ethylene]]bis[N-2- hydroxyethyl- (5CI) (CA INDEX NAME)", XP093080982, retrieved from STNext Database accession no. 875225-20-0
GALETTI, M.D. ET AL.: "Multicomponent Synthesis of Acylated Short Peptoids with Antifungal Activity against Plant Pathogens.", MOLECULAR DIVERSITY., vol. 16, no. 1, 16 September 2011 (2011-09-16), XP035031341, DOI: 10.1007/s11030-011-9334-1
CHRISMENT J., DELPUECH J.-J.: "Proton Transfers on Amphiphilic Molecules. A Nonionic Alkyllipopeptide in Dimethyl Sulfoxide", LANGMUIR, AMERICAN CHEMICAL SOCIETY, US, vol. 12, no. 10, 1 January 1996 (1996-01-01), US , pages 2441 - 2445, XP093080983, ISSN: 0743-7463, DOI: 10.1021/la950786s
MOLLA MIJANUR R., CHAKRABORTY SHRADDHA, MUNOZ−SAGREDO LEONEL, DRECHSLER MARKUS, ORIAN−ROUSSEAU VÉRONIQUE, LEVKIN PAVEL A.: "Combinatorial Synthesis of a Lipidoid Library by Thiolactone Chemistry: In Vitro Screening and In Vivo Validation for siRNA Delivery", BIOCONJUGATE CHEMISTRY, AMERICAN CHEMICAL SOCIETY, US, vol. 31, no. 3, 18 March 2020 (2020-03-18), US , pages 852 - 860, XP093080665, ISSN: 1043-1802, DOI: 10.1021/acs.bioconjchem.0c00013
Attorney, Agent or Firm:
SCIHEAD IP LAW FIRM (CN)
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