Fehlangepasste Sirna, Sirna-Konjugat, Pharmazeutische Zusammensetzung und Verwendung davon

Details

Veröffentlichungs-Nr.

EP4793354

Anmeldetag

23. Januar 2026

Veröffentlichung

19. August 2026

Rechtsraum

EP

IPC

C12N15/11, C12N15/113

Abstract

The present invention provides a mismatched siRNA for inhibiting gene expression, an siRNA conjugate, a pharmaceutical composition, and uses thereof, the siRNA comprises a sense strand and an antisense strand; wherein the sense strand is 17-21 continuous nucleotides in length and the antisense strand is 19-23 continuous nucleotides in length; the sense strand and the antisense strand comprise one or more of a 2'-methoxy modified nucleotide, a 2'-fluoro modified nucleotide, and a 5'-VP modified nucleotide, and comprise one or more phosphorothioate groups located between two adjacent nucleotides; and at least one nucleotide at a position greater than 15 of the antisense strand in the 5' end to 3' end direction is mismatched to the target gene.

Anmelder

Firma

Chengdu Gowell Biopharmaceutical Co., Ltd.

Land

🇨🇳 China

Fachgebiete