Polypeptide oligonucleotide conjugate for inhibiting expression of target gene in central nervous system and use thereof

Abstract

The invention relates to a polypeptide oligonucleotide conjugate with a structure as shown in a formula (I) or (II) or a pharmaceutically acceptable salt or a stereoisomer thereof, and an application of the polypeptide oligonucleotide conjugate or the pharmaceutically acceptable salt or the stereoisomer thereof. The polypeptide oligonucleotide conjugate can be used for inhibiting expression of one or more target genes of central nervous system tissue cells, and has the advantages of good in-vivo effect, long action time and good safety.

Description

(19) State Intellectual Property Office (12) Invention Patent Application (10) Application Publication Number (43) Application Publication Date (21) Application Number 202580000534.0 (22) Application Date 2025.03.27 (66) Domestic Priority Data 202410368843.0 2024.03.28 CN 202411353131.8 2024.09.26 CN (85) PCT International Application Entering National Phase Date 2025.04.23 (86) PCT International Application Application Data PCT / CN2025 / 085394 2025.03.27 (87) PCT International Application Publication Data WO2025 / 201467 ZH 2025.10.02 (71) Applicant Guangzhou Bibet Pharmaceutical Co., Ltd. Address 510660 Room 802, 8th Floor, Building A-3, No. 25, Yayingshi Road, Science City, Guangzhou High-tech Industrial Development Zone, Guangdong Province (72) Inventors: Fan Fushun, Liu Xinjian, Fu Tiancheng, Lin Yingying, Liang Hao, Weng Yunwo, Qian Changgeng (74) Patent Agency: Guangzhou Guangdian Intellectual Property Agency (General Partnership) 44365 Patent Attorney: Zeng Yinfeng (51) Int.Cl. A61K 47 / 64 (2017.01) A61K 31 / 713 (2006.01) A61K 31 / 7105 (2006.01) A61P 25 / 00 (2006.01) A61P 25 / 16 (2006.01) A61P 25 / 28 (2006.01) A61P 25 / 14 (2006.01) A61P 21 / 00(2006.01) A61P 31 / 14(2006.01) C07K 1 / 00(2006.01) C07K 7 / 06(2006.01) C12N 15 / 11(2006.01) (54) Invention Title: Polypeptide Oligonucleotide Conjugate for Inhibiting the Expression of Target Genes in the Central Nervous System and Its Application (57) Abstract: This invention relates to a polypeptide oligonucleotide conjugate having the structure shown in formula (I) or (II) below, or a pharmaceutically acceptable salt thereof or a stereoisomer thereof, and its application. The polypeptide oligonucleotide conjugate of this invention can be used to inhibit the expression of one or more target genes in central nervous system tissue cells, and has the advantages of good in vivo efficacy, long duration of action, and good safety. CN 121127266 A 2025.12.12 CN 1 21 12 72 66 A

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