Sirna targeting inhbe gene expression, conjugate thereof, and use thereof

WO2026123917A1PCT designated stage Publication Date: 2026-06-18LEADERNA THERAPEUTICS LTD
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Patent Information

Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
LEADERNA THERAPEUTICS LTD
Filing Date
2025-10-09
Publication Date
2026-06-18

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  • Figure PCTCN2025126525-FTAPPB-I100001
    Figure PCTCN2025126525-FTAPPB-I100001
  • Figure PCTCN2025126525-FTAPPB-I100002
    Figure PCTCN2025126525-FTAPPB-I100002
  • Figure PCTCN2025126525-FTAPPB-I100003
    Figure PCTCN2025126525-FTAPPB-I100003
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Abstract

Provided are an siRNA for inhibiting the expression of an inhibin subunit beta E (INHBE) gene and a conjugate thereof. The siRNA comprises a sense strand and an antisense strand. The antisense strand comprises at least 17 consecutive nucleotides that differ from nucleotide sequences set forth in SEQ ID NO: 299 to SEQ ID NO: 595, SEQ ID NO: 751 to SEQ ID NO: 904, and SEQ ID NO: 909 to SEQ ID NO: 911 by no more than 4 nucleotides, and the antisense strand has a length of 17-30 nucleotides; the sense strand has a length of 17-30 nucleotides and is at least partially complementary to the antisense strand. The provided siRNA, siRNA conjugate, and pharmaceutical composition have good stability, excellent INHBE gene inhibitory activity, and satisfactory cytotoxicity and immunostimulatory properties.
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