COMPLEMENT COMPONENT IRNA COMPOSITIONS AND METHODS OF USE THEREOF

    公开(公告)号:EP3798306A1

    公开(公告)日:2021-03-31

    申请号:EP20200586.4

    申请日:2014-12-12

    摘要: The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting expression of complement component C3 in a cell, wherein said dsRNA comprises a sense strand and an antisense strand, wherein each strand comprises 15-30 nucleotides and said strands form a double-stranded region, and wherein the antisense strand comprising at least 15 contiguous nucleotides differing by no more than 3 nucleotides from any one of the following antisense sequences:
    a. GAGAGAAGACCUUGACCACGUAG (SEQ ID NO:282);
    b. UAGAGAAGACCUUGACCACGUAG (SEQ ID NO:267);
    c. AGAGAGAAGACCUUGACCACGUA (SEQ ID NO:266);
    d. AGAGAAGACCUUGACCACGUAGG (SEQ ID NO:299); and wherein at least one nucleotide in said dsRNA comprises a modification.