	US 12,076,335 B2
	Nucleic acids for inhibiting expression of TMPRSS6 and iron chelators
Ute Schaeper, Berlin (DE)
Assigned to SILENCE THERAPEUTICS GMBH, Berlin (DE)
Appl. No. 17/263,090
Filed by SILENCE THERAPEUTICS GMBH, Berlin (DE)
PCT Filed Jul. 26, 2019, PCT No. PCT/EP2019/070270 § 371(c)(1), (2) Date Jan. 25, 2021, PCT Pub. No. WO2020/021108, PCT Pub. Date Jan. 30, 2020.
Claims priority of application No. 18185879 (EP), filed on Jul. 26, 2018.
Prior Publication US 2022/0331351 A1, Oct. 20, 2022
Int. Cl. A61K 31/713 (2006.01); A61K 31/4412 (2006.01); A61P 3/02 (2006.01); A61P 7/06 (2006.01); C12N 15/113 (2010.01)

CPC A61K 31/713 (2013.01) [A61K 31/4412 (2013.01); A61P 3/02 (2018.01); A61P 7/06 (2018.01); C12N 15/1137 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2310/322 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/351 (2013.01); C12N 2320/31 (2013.01)]

12 Claims

1. A combination comprising a nucleic acid for inhibiting expression of TMPRSS6 and one or more iron chelators,

wherein the nucleic acid comprises at least one duplex region that comprises at least a portion of a first strand and at least a portion of a second strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from the TMPRSS6 gene, and wherein said first strand comprises the nucleotide sequence 5′aaccagaagaagcagguga 3′ (SEQ ID NO:17) comprising modifications 6273646282647284546, and wherein said second strand comprises the nucleotide sequence 5′ucaccugcuucuucugguu 3′ (SEQ ID NO:18) comprising modifications 1727354715351718451,

wherein the specific modifications are depicted by the following numbers

1=2′F-dU;

2=2′F-dA;

3=2′F-dC;

4=2′F-dG;

5=2′-OMe-rU;

6=2′-OMe-rA;

7=2′-OMe-rC; and

8=2′-OMe-rG.