Title:
LIPID COMPOSITION AND METHOD OF DELIVERING THERAPEUTIC AGENT
Document Type and Number:
WIPO Patent Application WO/2024/085190
Kind Code:
A1
Abstract:
It is an object of the present invention to provide a lipid composition capable of delivering a nucleic acid such as RNA to a hematopoietic stem/progenitor cell or mesenchymal stem cells, and a method of delivering a therapeutic agent to a cell using the lipid composition. The present invention provides a lipid composition comprising (A) a therapeutic agent and (B) a lipid nanoparticle conjugated to a targeting molecule, wherein the lipid nanoparticle comprises an ionizable lipid, and the targeting molecule specifically binds to a marker of hematopoietic stem / progenitor cells or mesenchymal stem cells.
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Inventors:
TOYONAGA SHO (US)
ANDERSON DANIEL GRIFFITH (US)
RAIMONDO THERESA MARIE (US)
SHI DENNIS ZHENG (US)
ANDERSON DANIEL GRIFFITH (US)
RAIMONDO THERESA MARIE (US)
SHI DENNIS ZHENG (US)
Application Number:
PCT/JP2023/037715
Publication Date:
April 25, 2024
Filing Date:
October 18, 2023
Export Citation:
Assignee:
FUJIFILM CORP (JP)
MASSACHUSETTS INST TECHNOLOGY (US)
MASSACHUSETTS INST TECHNOLOGY (US)
International Classes:
A61K47/42; A61K9/10; A61K31/7088; A61K47/14; A61K47/18; A61K47/22; A61K47/24; A61K47/28; A61K48/00; A61P35/02
Foreign References:
| US20180142261A1 | 2018-05-24 | |||
| US20210128485A1 | 2021-05-06 | |||
| US20200246581A1 | 2020-08-06 |
Other References:
CANNON PAULA, ASOKAN ARAVIND, CZECHOWICZ AGNIESZKA, HAMMOND PAULA, KOHN DONALD B., LIEBER ANDRE, MALIK PUNAM, MARKS PETER, PORTEUS: "Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development", HUMAN GENE THERAPY, MARY ANN LIEBERT, INC. PUBLISHERS, GB, vol. 32, no. 1-2, 1 January 2021 (2021-01-01), GB , pages 31 - 42, XP093002413, ISSN: 1043-0342, DOI: 10.1089/hum.2020.263
CHULPANOVA, D S et al., Generation of genetically engineered human mesenchymal stem cells expressing immunomodulating cytokines and tumour suppressor, Human Gene Therapy, 2017, Vol.28, No.12, Abstracts, A39, P074
DENNIS SHI: "In Vivo RNA Delivery to Hematopoietic Stem and Progenitor Cells via Targeted Lipid Nanoparticles", NANO LETTERS, AMERICAN CHEMICAL SOCIETY, US, vol. 23, no. 7, 12 April 2023 (2023-04-12), US , pages 2938 - 2944, XP093162502, ISSN: 1530-6984, DOI: 10.1021/acs.nanolett.3c00304
CHULPANOVA, D S et al., Generation of genetically engineered human mesenchymal stem cells expressing immunomodulating cytokines and tumour suppressor, Human Gene Therapy, 2017, Vol.28, No.12, Abstracts, A39, P074
DENNIS SHI: "In Vivo RNA Delivery to Hematopoietic Stem and Progenitor Cells via Targeted Lipid Nanoparticles", NANO LETTERS, AMERICAN CHEMICAL SOCIETY, US, vol. 23, no. 7, 12 April 2023 (2023-04-12), US , pages 2938 - 2944, XP093162502, ISSN: 1530-6984, DOI: 10.1021/acs.nanolett.3c00304
Attorney, Agent or Firm:
SIKS & CO. (JP)
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