Title:
LIPID NANOPARTICLES FOR DELIVERY OF NUCLEIC ACIDS AND METHODS OF USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2023/230587
Kind Code:
A3
Abstract:
The present disclosure provides for improved compositions of ionizable lipid nanoparticles for the delivery of therapeutic nucleic acids to cells. Anionic phospholipids, including phosphatidyl serine and phosphatidylglycerol are included in the lipid nanoparticles to increase the transfection efficiency in human dendritic cells. The further incorporation of mono-unsaturated alkyl chain analogs in dimethylaminopropyl-di oxolane or heterocyclic ketal ionizable lipids in the formulation demonstrated high levels of transfection in human dendritic cells, compared to other ionizable lipids in the same family, and demonstrated good stability to oxidative damage. Finally, the use of an ammonium salt of phosphatidylserine allows for the efficient production of PS-targeted LNPs.
Inventors:
DRUMMOND DARYL (US)
KIRPOTIN DMITRI (US)
HAYES MARK (US)
KOSHKARYEV ALEXANDER (US)
FULTON ROSS (US)
KIRPOTIN DMITRI (US)
HAYES MARK (US)
KOSHKARYEV ALEXANDER (US)
FULTON ROSS (US)
Application Number:
PCT/US2023/067517
Publication Date:
January 11, 2024
Filing Date:
May 25, 2023
Export Citation:
Assignee:
AKAGERA MEDICINES INC (US)
International Classes:
A61K9/51; A61K9/14; A61K9/16; A61K48/00
Domestic Patent References:
| WO2017201346A1 | 2017-11-23 |
Foreign References:
| US20110060032A1 | 2011-03-10 | |||
| US20120276209A1 | 2012-11-01 | |||
| US20210290756A1 | 2021-09-23 | |||
| US20190262448A1 | 2019-08-29 |
Other References:
HAUSER H., SHIPLEY G.G.: "Crystallization of phosphatidylserine bilayers induced by lithium.", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, US, vol. 256, no. 22, 1 November 1981 (1981-11-01), US , pages 11377 - 11380, XP093128589, ISSN: 0021-9258, DOI: 10.1016/S0021-9258(19)68404-8
SEAN C SEMPLE, AKIN AKINC, JIANXIN CHEN, AMMEN P SANDHU, BARBARA L MUI, CONNIE K CHO, DINAH W Y SAH, DERRICK STEBBING, ERIN J CROS: "Rational design of cationic lipids for siRNA delivery", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 28, no. 2, 1 February 2010 (2010-02-01), New York, pages 172 - 176, XP055539273, ISSN: 1087-0156, DOI: 10.1038/nbt.1602
SEAN C SEMPLE, AKIN AKINC, JIANXIN CHEN, AMMEN P SANDHU, BARBARA L MUI, CONNIE K CHO, DINAH W Y SAH, DERRICK STEBBING, ERIN J CROS: "Rational design of cationic lipids for siRNA delivery", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 28, no. 2, 1 February 2010 (2010-02-01), New York, pages 172 - 176, XP055539273, ISSN: 1087-0156, DOI: 10.1038/nbt.1602
Attorney, Agent or Firm:
DYKEMAN, David, J. (US)
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