Compositions and methods for inhibiting transmembrane serine protease 6 (TMPRSS6) expression
AssigneesNovo Nordisk A/S, Dicerna Pharmaceuticals, Inc.
InventorsAnna Linda Blois, Christina Marie Priest, Jihye Park, Henryk Dudek
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Abstract
Oligonucleotides are provided herein that inhibit TMPRSS6 expression. Also provided are compositions including the same and uses thereof, particularly uses relating to treating diseases, disorders and/or conditions associated with hepcidin deficiency or suppression.
Claims28
An RNAi oligonucleotide for reducing transmembrane serine protease 6 (TMPRSS6) expression, comprising a sense strand and an antisense strand, wherein the sense strand and the antisense strand form a duplex region, and wherein the sense strand comprises a nucleotide sequence as set forth in SEQ ID NO:
- 844 and the antisense strand comprises the nucleotide sequences as set forth in SEQ ID NO:
- 600.
An RNAi oligonucleotide for reducing TMPRSS6 expression, the oligonucleotide comprising a sense strand and an antisense strand, wherein
- the sense strand comprises the sequence and all of the modifications of 5′-[mGs][mG][mU][mG][mC][mU][mA][fC][fU][fC][fU][mG][mG][mU][mA][mU][mU][mU][mC][mA][mG][mC]
- [mA][mG][mC][mC][mG][ademA-GalNAc][ademA-GalNAc][ademA-GalNAc][mG][mG][mC][mU][mG][mC]-3′ (SEQ ID NO:
- 621), and
- wherein the antisense strand comprises the sequence and all of the modifications of 5′-[MePhosphonate-4O-mUs][fGs][fAs][fA][fA][mU][fA][mC][mC][fA][mG][mA][mG][fU][mA][mG][mC][mA][mC][mCs][mGs][mG]-3′ (SEQ ID NO:
- 642), wherein mC, mA, mG, mU=2′-OMe ribonucleosides;
- fA, fC, fG, fU=2′F ribonucleosides;
- s=phosphorothioate, and wherein ademA-GalNAc=
Dependencies are inferred from the claim text.