Title:
LIPID NANOPARTICLES AND METHOD FOR PREPARING SAME
Document Type and Number:
WIPO Patent Application WO/2022/270941
Kind Code:
A1
Abstract:
The present invention relates to lipid nanoparticles and a method for preparing same and, more specifically, to particles containing an ionizable lipid and a polyethylene glycol moiety (PEG moiety)-degradable liking functional group-lipid conjugate, which are characterized by minimized side effects in vivo and effective nanoparticle transfer into target cells so as to deliver pharmaceutically effective materials into the cytoplasm.
Inventors:
LEE TAEWOO (KR)
BAEK YU MI (KR)
RYU JIWON (KR)
JANG DAHYE (KR)
BAEK YU MI (KR)
RYU JIWON (KR)
JANG DAHYE (KR)
Application Number:
PCT/KR2022/008941
Publication Date:
December 29, 2022
Filing Date:
June 23, 2022
Export Citation:
Assignee:
THERNA THERAPEUTICS (KR)
International Classes:
A61K9/51; A61K31/7088; A61K47/10; A61K47/18; A61K47/24; A61K47/26; A61K47/28; A61K48/00
Domestic Patent References:
| WO2020061284A1 | 2020-03-26 |
Foreign References:
| KR20200006176A | 2020-01-17 | |||
| KR20180032652A | 2018-03-30 | |||
| US9999673B2 | 2018-06-19 | |||
| KR20180032652A | 2018-03-30 |
Other References:
CHIA-LING CHAN; RAMSEY N. MAJZOUB; RAHAU S. SHIRAZI; KAI K. EWERT; YEN-JU CHEN; KENG S. LIANG; CYRUS R. SAFINYA;: "Endosomal escape and transfection efficiency of PEGylated cationic liposomeDNA complexes prepared with an acid-labile PEG-lipid", BIOMATERIALS, ELSEVIER, AMSTERDAM, NL, vol. 33, no. 19, 10 March 2012 (2012-03-10), AMSTERDAM, NL , pages 4928 - 4935, XP028413204, ISSN: 0142-9612, DOI: 10.1016/j.biomaterials.2012.03.038
KANAMALA MANJU; PALMER BRIAN D.; GHANDEHARI HAMIDREZA; WILSON WILLIAM R.; WU ZIMEI: "PEG-Benzaldehyde-Hydrazone-Lipid Based PEG-Sheddable pH-Sensitive Liposomes: Abilities for Endosomal Escape and Long Circulation", PHARMACEUTICAL RESEARCH, SPRINGER US, NEW YORK, vol. 35, no. 8, 31 May 2018 (2018-05-31), New York, pages 1 - 13, XP036516396, ISSN: 0724-8741, DOI: 10.1007/s11095-018-2429-y
KIM CHANG HYUN; LEE SANG GON; KANG MYUNG JOO; LEE SANGKIL; CHOI YOUNG WOOK: "Surface modification of lipid-based nanocarriers for cancer cell-specific drug targeting", JOURNAL OF PHARMACEUTICAL INVESTIGATION, SPRINGER SINGAPORE, SINGAPORE, vol. 47, no. 3, 17 April 2017 (2017-04-17), Singapore, pages 203 - 227, XP036228613, ISSN: 2093-5552, DOI: 10.1007/s40005-017-0329-5
MOSS, K.H.POPOVA, P.HADRUP, S.R.ASTAKHOVA, K.TASKOVA, M.: "Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides", MOL PHARM, vol. 16, 2019, pages 2265 - 2277, XP055742602, DOI: 10.1021/acs.molpharmaceut.8b01290
KULKARNI, J.A.WITZIGMANN, D.CHEN, S.CULLIS, P.R.VAN DER MEEL, R.: "Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics", ACC CHEM RES, vol. 52, 2019, pages 2435 - 2444, XP055878486, DOI: 10.1021/acs.accounts.9b00368
BUCK, J.GROSSEN, P.CULLIS, P.R.HUWYLER, J.WITZIGMANN, D.: "Lipid-Based DNA Therapeutics: Hallmarks of Non-Viral Gene Delivery", ACS NANO, vol. 13, 2019, pages 3754 - 3782
AKINC, A. ET AL.: "The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs", NAT NANOTECHNOL, vol. 14, 2019, pages 1084 - 1087, XP036953174, DOI: 10.1038/s41565-019-0591-y
SPRINGER, A.D.DOWDY, S.F.: "GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics", NUCLEIC ACID THER, vol. 28, 2018, pages 109 - 118, XP055555952, DOI: 10.1089/nat.2018.0736
KULKARNI, J.A.CULLIS, P.R.VAN DER MEEL, R.: "Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility", NUCLEIC ACID THER, vol. 28, 2018, pages 146 - 157, XP055551908, DOI: 10.1089/nat.2018.0721
RIETWYK, S.PEER, D.: "Next-Generation Lipids in RNA Interference Therapeutics", ACS NANO, vol. 11, 2017, pages 7572 - 7586, XP055618179, DOI: 10.1021/acsnano.7b04734
FANG, Y. ET AL.: "Cleavable PEGylation: a strategy for overcoming the ''PEG dilemma'' in efficient drug delivery", DRUG DELIV, vol. 24, 2017, pages 22 - 32, XP055642596, DOI: 10.1080/10717544.2017.1388451
CULLIS, P.R.HOPE, M.J.: "Lipid Nanoparticle Systems for Enabling Gene Therapies", MOL THER, vol. 25, 2017, pages 1467 - 1475, XP055548712, DOI: 10.1016/j.ymthe.2017.03.013
KUMAR, V. ET AL.: "Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy", MOL THER NUCLEIC ACIDS, vol. 3, 2014, pages e210
BELLIVEAU, N.M. ET AL.: "Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA", MOL THER NUCLEIC ACIDS, vol. 1, 2012, pages e37, XP002715253, DOI: 10.1038/mtna.2012.28
SATO, Y. ET AL.: "Elucidation of the physicochemical properties and potency of siRNA-loaded small-sized lipid nanoparticles for siRNA delivery", J CONTROL RELEASE, vol. 229, 2016, pages 48 - 57, XP029521836, DOI: 10.1016/j.jconrel.2016.03.019
BAO, Y. ET AL.: "Effect of PEGylation on biodistribution and gene silencing of siRNA/lipid nanoparticle complexes", PHARM RES, vol. 30, 2013, pages 342 - 51, XP093037641, DOI: 10.1007/s11095-012-0874-6
MUI, B.L. ET AL.: "Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles", MOL THER NUCLEIC ACIDS, vol. 2, 2013, pages e139, XP055898080, DOI: 10.1038/mtna.2013.66
CHEN, S. ET AL.: "Development of lipid nanoparticle formulations of siRNA for hepatocyte gene silencing following subcutaneous administration", J CONTROLRELEASE, vol. 196, 2014, pages 106 - 12, XP029112393, DOI: 10.1016/j.jconrel.2014.09.025
AKINC, A. ET AL.: "Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms", MOL THER, vol. 18, 2010, pages 1357 - 64, XP055016290, DOI: 10.1038/mt.2010.85
CHEN, S. ET AL.: "Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA", J CONTROL RELEASE, vol. 235, 2016, pages 236 - 244
BARGH, J.D.ISIDRO-LLOBET, A.PARKER, J.S.SPRING, D.R.: "Cleavable linkers in antibody-drug conjugates", CHEM SOC REV, vol. 48, 2019, pages 4361 - 4374, XP009517547, DOI: 10.1039/c8cs00676h
ZHAO, G. ET AL.: "Smart pH-sensitive nanoassemblies with cleavable PEGylation for tumor targeted drug delivery", SCI REP, vol. 7, 2017, pages 3383
KANAMALA MANJU; PALMER BRIAN D.; GHANDEHARI HAMIDREZA; WILSON WILLIAM R.; WU ZIMEI: "PEG-Benzaldehyde-Hydrazone-Lipid Based PEG-Sheddable pH-Sensitive Liposomes: Abilities for Endosomal Escape and Long Circulation", PHARMACEUTICAL RESEARCH, SPRINGER US, NEW YORK, vol. 35, no. 8, 31 May 2018 (2018-05-31), New York, pages 1 - 13, XP036516396, ISSN: 0724-8741, DOI: 10.1007/s11095-018-2429-y
KIM CHANG HYUN; LEE SANG GON; KANG MYUNG JOO; LEE SANGKIL; CHOI YOUNG WOOK: "Surface modification of lipid-based nanocarriers for cancer cell-specific drug targeting", JOURNAL OF PHARMACEUTICAL INVESTIGATION, SPRINGER SINGAPORE, SINGAPORE, vol. 47, no. 3, 17 April 2017 (2017-04-17), Singapore, pages 203 - 227, XP036228613, ISSN: 2093-5552, DOI: 10.1007/s40005-017-0329-5
MOSS, K.H.POPOVA, P.HADRUP, S.R.ASTAKHOVA, K.TASKOVA, M.: "Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides", MOL PHARM, vol. 16, 2019, pages 2265 - 2277, XP055742602, DOI: 10.1021/acs.molpharmaceut.8b01290
KULKARNI, J.A.WITZIGMANN, D.CHEN, S.CULLIS, P.R.VAN DER MEEL, R.: "Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics", ACC CHEM RES, vol. 52, 2019, pages 2435 - 2444, XP055878486, DOI: 10.1021/acs.accounts.9b00368
BUCK, J.GROSSEN, P.CULLIS, P.R.HUWYLER, J.WITZIGMANN, D.: "Lipid-Based DNA Therapeutics: Hallmarks of Non-Viral Gene Delivery", ACS NANO, vol. 13, 2019, pages 3754 - 3782
AKINC, A. ET AL.: "The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs", NAT NANOTECHNOL, vol. 14, 2019, pages 1084 - 1087, XP036953174, DOI: 10.1038/s41565-019-0591-y
SPRINGER, A.D.DOWDY, S.F.: "GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics", NUCLEIC ACID THER, vol. 28, 2018, pages 109 - 118, XP055555952, DOI: 10.1089/nat.2018.0736
KULKARNI, J.A.CULLIS, P.R.VAN DER MEEL, R.: "Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility", NUCLEIC ACID THER, vol. 28, 2018, pages 146 - 157, XP055551908, DOI: 10.1089/nat.2018.0721
RIETWYK, S.PEER, D.: "Next-Generation Lipids in RNA Interference Therapeutics", ACS NANO, vol. 11, 2017, pages 7572 - 7586, XP055618179, DOI: 10.1021/acsnano.7b04734
FANG, Y. ET AL.: "Cleavable PEGylation: a strategy for overcoming the ''PEG dilemma'' in efficient drug delivery", DRUG DELIV, vol. 24, 2017, pages 22 - 32, XP055642596, DOI: 10.1080/10717544.2017.1388451
CULLIS, P.R.HOPE, M.J.: "Lipid Nanoparticle Systems for Enabling Gene Therapies", MOL THER, vol. 25, 2017, pages 1467 - 1475, XP055548712, DOI: 10.1016/j.ymthe.2017.03.013
KUMAR, V. ET AL.: "Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy", MOL THER NUCLEIC ACIDS, vol. 3, 2014, pages e210
BELLIVEAU, N.M. ET AL.: "Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA", MOL THER NUCLEIC ACIDS, vol. 1, 2012, pages e37, XP002715253, DOI: 10.1038/mtna.2012.28
SATO, Y. ET AL.: "Elucidation of the physicochemical properties and potency of siRNA-loaded small-sized lipid nanoparticles for siRNA delivery", J CONTROL RELEASE, vol. 229, 2016, pages 48 - 57, XP029521836, DOI: 10.1016/j.jconrel.2016.03.019
BAO, Y. ET AL.: "Effect of PEGylation on biodistribution and gene silencing of siRNA/lipid nanoparticle complexes", PHARM RES, vol. 30, 2013, pages 342 - 51, XP093037641, DOI: 10.1007/s11095-012-0874-6
MUI, B.L. ET AL.: "Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles", MOL THER NUCLEIC ACIDS, vol. 2, 2013, pages e139, XP055898080, DOI: 10.1038/mtna.2013.66
CHEN, S. ET AL.: "Development of lipid nanoparticle formulations of siRNA for hepatocyte gene silencing following subcutaneous administration", J CONTROLRELEASE, vol. 196, 2014, pages 106 - 12, XP029112393, DOI: 10.1016/j.jconrel.2014.09.025
AKINC, A. ET AL.: "Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms", MOL THER, vol. 18, 2010, pages 1357 - 64, XP055016290, DOI: 10.1038/mt.2010.85
CHEN, S. ET AL.: "Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA", J CONTROL RELEASE, vol. 235, 2016, pages 236 - 244
BARGH, J.D.ISIDRO-LLOBET, A.PARKER, J.S.SPRING, D.R.: "Cleavable linkers in antibody-drug conjugates", CHEM SOC REV, vol. 48, 2019, pages 4361 - 4374, XP009517547, DOI: 10.1039/c8cs00676h
ZHAO, G. ET AL.: "Smart pH-sensitive nanoassemblies with cleavable PEGylation for tumor targeted drug delivery", SCI REP, vol. 7, 2017, pages 3383
Attorney, Agent or Firm:
LEE, Cheo Young et al. (KR)
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