US 11,655,473 B2
Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (MARC1) expression
Henryk Dudek, Belmont, MA (US); Wen Han, Boston, MA (US); Natalie Wayne Pursell, Westborough, MA (US); Chengjung Lai, Hudson, NH (US); William Geoffrey Haynes, Oxford (GB); and Zhihao Ding, Biberach an der Riss (DE)
Assigned to Novo Nordisk A/S, Bagsvaerd (DK); and Dicerna Pharmaceuticals, Inc., Lexington, MA (US)
Filed by Novo Nordisk A/S, Bagsvaerd (DK); and Dicerna Pharmaceuticals, Inc., Lexington, MA (US)
Filed on May 27, 2022, as Appl. No. 17/826,211.
Claims priority of provisional application 63/194,395, filed on May 28, 2021.
Claims priority of application No. 21183860 (EP), filed on Jul. 6, 2021.
Prior Publication US 2023/0042451 A1, Feb. 9, 2023
Int. Cl. C12N 15/113 (2010.01); A61P 1/16 (2006.01)
CPC C12N 15/1137 (2013.01) [A61P 1/16 (2018.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/351 (2013.01); C12N 2310/531 (2013.01)] 27 Claims
 
1. A double stranded RNAi oligonucleotide (dsRNAi) capable of inhibiting the protein expression of MARC1, wherein said dsRNAi comprises a sense strand and an antisense strand, wherein the sense strand and the antisense strand form a duplex region, and wherein the sense and antisense strands are selected from the group consisting of
(i) the sense and antisense strands comprise the nucleotide sequences set forth in SEQ ID NOs: 1543 and 1579, respectively;
(ii) the sense and antisense strands comprise the nucleotide sequences set forth in SEQ ID NOs: 1560 and 1596, respectively;
(iii) the sense and antisense strands comprise the nucleotide sequences set forth in SEQ ID NOs: 1568 and 1604, respectively; and
(iv) the sense and antisense strands comprise the nucleotide sequences set forth in SEQ ID NOs: 1553 and 1589, respectively.