MODIFIED SIRNA WITH REDUCED OFF-TARGET ACTIVITY

    公开(公告)号:EP4194553A1

    公开(公告)日:2023-06-14

    申请号:EP21852482.5

    申请日:2021-08-04

    摘要: Disclosed is a modified siRNA with a reduced off-target activity. The siRNA comprises a sense strand and an antisense strand, wherein the antisense strand contains a chemical modification as represented by formula (I) or a tautomeric modification thereof in at least one nucleotide position from position 2 to position 8 of 5' region thereof. A conjugate, a pharmaceutical composition, a cell or a kit containing the siRNA, and the medical use of the siRNA, the conjugate and/or the pharmaceutical composition thereof are also disclosed. Further disclosed are compounds as represented by formula (II) and formula (III) or tautomers thereof, and preparation methods therefor.