Title:
BRANCHED POLYPEPTIDE VECTOR FOR EFFECTIVELY DELIVERING NUCLEIC ACIDS AND VARIANT THEREOF
Document Type and Number:
WIPO Patent Application WO/2024/041372
Kind Code:
A1
Abstract:
Provided are a branched polypeptide vector and a variant thereof. The branched polypeptide has a sequence set forth in the following: Xaa1(P2)-Xaa1-Xaa1(P1)-Xaa1-Xaa1(P2), or Xaa1(P1)-Xaa1-Xaa1(P2)-Xaa1-Xaa1(P2)-Xaa1-Xaa1(P1), or Xaa1(P2)-Xaa1-Xaa1(P1)-Xaa1-Xaa1(P1)-Xaa1-Xaa1(P2), or Xaa1(P2)-Xaa1-Xaa1(P1)-Xaa1-Xaa1(P2)-Xaa1-Xaa1(P1)-Xaa1-Xaa1(P2). According to the embodiment, nucleic acids can be efficiently delivered into tissues and organs in vivo to achieve targeted therapies, and the polypeptide has a conformational flexibility and affinity several folds greater than macromolecular drugs (proteins and antibodies). The multi-branched polypeptide has prolonged stability in vivo while maintaining strong affinity and minimal toxicity. By forming stable nanocomplexes or nanoparticles with nucleic acid molecules by means of electrostatic interactions, the polypeptide can achieve the delivery of nucleic acid drugs and the stable release of the nucleic acid drugs in cells, and improve the activity of the nucleic acid drugs.
Inventors:
LU QINGQING (CN)
XIA DAN (CN)
YI CHENG (CN)
GE YOUZHEN (CN)
HU HONGPENG (CN)
JIANG JIANHAO (CN)
ZHONG TIANYI (CN)
ZHANG LIXIA (CN)
JIA KAN (CN)
ZHANG YUYANG (CN)
XIA DAN (CN)
YI CHENG (CN)
GE YOUZHEN (CN)
HU HONGPENG (CN)
JIANG JIANHAO (CN)
ZHONG TIANYI (CN)
ZHANG LIXIA (CN)
JIA KAN (CN)
ZHANG YUYANG (CN)
Application Number:
PCT/CN2023/111747
Publication Date:
February 29, 2024
Filing Date:
August 08, 2023
Export Citation:
Assignee:
SUZHOU HEALIRNA BIOTECHNOLOGY CO LTD (CN)
International Classes:
C07K7/08; A61K38/12; A61K38/16; A61P1/00; A61P5/00; A61P9/00; A61P11/00; A61P19/00; A61P25/00; A61P27/02; A61P27/16; A61P31/04; A61P31/12; A61P37/02; C07K14/00
Domestic Patent References:
| WO2023093596A1 | 2023-06-01 |
Foreign References:
| CN101072581A | 2007-11-14 | |||
| EP2678038B1 | 2019-05-08 | |||
| US20040001827A1 | 2004-01-01 | |||
| US20130259879A1 | 2013-10-03 |
Other References:
MARKUS DE RAAD, ERIK A. TEUNISSEN, DAPHNE LELIEVELD, DAVID A. EGAN, ENRICO MASTROBATTISTA: "High-content screening of peptide-based non-viral gene delivery systems", JOURNAL OF CONTROLLED RELEASE, ELSEVIER, AMSTERDAM, NL, vol. 158, no. 3, 1 March 2012 (2012-03-01), AMSTERDAM, NL , pages 433 - 442, XP055599187, ISSN: 0168-3659, DOI: 10.1016/j.jconrel.2011.09.078
LEWIS R. N. A. H., LIU F., KRIVANEK R., RYBAR P., HIANIK T., FLACH C. R., MENDELSOHN R., CHEN Y., MANT C. T., HODGES R. S., MCELHA: "Studies of the Minimum Hydrophobicity of α-Helical Peptides Required To Maintain a Stable Transmembrane Association with Phospholipid Bilayer Membranes", BIOCHEMISTRY, vol. 46, no. 4, 1 January 2007 (2007-01-01), pages 1042 - 1054, XP093143497, ISSN: 0006-2960, DOI: 10.1021/bi061891b
LEWIS R. N. A. H., LIU F., KRIVANEK R., RYBAR P., HIANIK T., FLACH C. R., MENDELSOHN R., CHEN Y., MANT C. T., HODGES R. S., MCELHA: "Studies of the Minimum Hydrophobicity of α-Helical Peptides Required To Maintain a Stable Transmembrane Association with Phospholipid Bilayer Membranes", BIOCHEMISTRY, vol. 46, no. 4, 1 January 2007 (2007-01-01), pages 1042 - 1054, XP093143497, ISSN: 0006-2960, DOI: 10.1021/bi061891b
Attorney, Agent or Firm:
SUZHOU JIANLI INTELLECTUAL PROPERTY AGENCY CO., LTD. (CN)
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