Source: https://patents.justia.com/patent/10487106
Timestamp: 2020-04-01 08:39:34
Document Index: 465532090

Matched Legal Cases: ['Application No. 2010', 'Application No. 12198517', 'Application No. 10177969', 'Application No. 10177969', 'Application No. 01979073', 'Application No. 05076770', 'Application No. 05076770', 'Application No. 12198517', 'Application No. 12198517', 'application No. 2000']

US Patent for Antisense nucleic acids Patent (Patent # 10,487,106 issued November 26, 2019) - Justia Patents Search
Justia Patents US Patent for Antisense nucleic acids Patent (Patent # 10,487,106)
Mar 29, 2019 - NIPPON SHINYAKU CO., LTD.
Latest NIPPON SHINYAKU CO., LTD. Patents:
Antisense nucleic acid for treating amyotrophy
This is a Continuation of copending application Ser. No. 15/619,996, filed Jun. 12, 2017, which is a Continuation of application Ser. No. 14/615,504, filed Feb. 6, 2015 (now U.S. Pat. No. 9,708,361 issued Jul. 18, 2017), which is a Continuation of application Ser. No. 13/819,520, filed Apr. 10, 2013 (now U.S. Pat. No. 9,079,934 issued Jul. 14, 2015), which is a PCT National Stage of PCT/JP2011/070318 filed Aug. 31, 2011, which claims priority to JP Application No. 2010-196032 filed Sep. 1, 2010, all of which are incorporated by reference in their entireties.
The instant application contains a Sequence Listing which has been submitted in ASCII format via EFS-Web and is hereby incorporated by reference in its entirety. Said ASCII copy, created on Mar. 26, 2019 is named 209658_0001_05_US_587895_ST25.txt and is 24,722 bytes in size.
TABLE 1 Tar- get se- quence in exon SEQ ID 53 Complementary nucleotide sequence NO:
31-53 5′-CCGGTTCTGAAGGTGTTCTTGTA-3′ SEQ ID NO: 2 31-54 5′-TCCGGTTCTGAAGGTGTTCTTGTA-3′ SEQ ID NO: 3 31-55 5′-CTCCGGTTCTGAAGGTGTTCTTGTA-3′ SEQ ID NO: 4 31-56 5′-CCTCCGGTTCTGAAGGTGTTCTTGTA-3′ SEQ ID NO: 5 31-57 5′-GCCTCCGGTTCTGAAGGTGTTCTTGTA-3′ SEQ ID NO: 6 31-58 5′-TGCCTCCGGTTCTGAAGGTGTTCTTGTA-3′ SEQ ID NO: 7 32-53 5′-CCGGTTCTGAAGGTGTTCTTGT-3′ SEQ ID NO: 8 32-54 5′-TCCGGTTCTGAAGGTGTTCTTGT-3′ SEQ ID NO: 9 32-55 5′-CTCCGGTTCTGAAGGTGTTCTTGT-3′ SEQ ID NO: 10 32-56 5′-CCTCCGGTTCTGAAGGTGTTCTTGT-3′ SEQ ID NO: 11 32-57 5′-GCCTCCGGTTCTGAAGGTGTTCTTGT-3′ SEQ ID NO: 12 32-58 5′-TGCCTCCGGTTCTGAAGGTGTTCTTGT-3′ SEQ ID NO: 13 33-53 5′-CCGGTTCTGAAGGTGTTCTTG-3′ SEQ ID NO: 14 33-54 5′-TCCGGTTCTGAAGGTGTTCTTG-3′ SEQ ID NO: 15 33-55 5′-CTCCGGTTCTGAAGGTGTTCTTG-3′ SEQ ID NO: 16 33-56 5′-CCTCCGGTTCTGAAGGTGTTCTTG-3′ SEQ ID NO: 17 33-57 5′-GCCTCCGGTTCTGAAGGTGTTCTTG-3′ SEQ ID NO: 18 33-58 5′-TGCCTCCGGTTCTGAAGGTGTTCTTG-3′ SEQ ID NO: 19 34-53 5′-CCGGTTCTGAAGGTGTTCTT-3′ SEQ ID NO: 20 34-54 5′-TCCGGTTCTGAAGGTGTTCTT-3′ SEQ ID NO: 21 34-55 5′-CTCCGGTTCTGAAGGTGTTCTT-3′ SEQ ID NO: 22 34-56 5′-CCTCCGGTTCTGAAGGTGTTCTT-3′ SEQ ID NO: 23 34-57 5′-GCCTCCGGTTCTGAAGGTGTTCTT-3′ SEQ ID NO: 24 34-58 5′-TGCCTCCGGTTCTGAAGGTGTTCTT-3′ SEQ ID NO: 25 35-53 5′-CCGGTTCTGAAGGTGTTCT-3′ SEQ ID NO: 26 35-54 5′-TCCGGTTCTGAAGGTGTTCT-3′ SEQ ID NO: 27 35-55 5′-CTCCGGTTCTGAAGGTGTTCT-3′ SEQ ID NO: 28 35-56 5′-CCTCCGGTTCTGAAGGTGTTCT-3′ SEQ ID NO: 29 35-57 5′-GCCTCCGGTTCTGAAGGTGTTCT-3′ SEQ ID NO: 30 35-58 5′-TGCCTCCGGTTCTGAAGGTGTTCT-3′ SEQ ID NO: 31 36-53 5′-CCGGTTCTGAAGGTGTTC-3′ SEQ ID NO: 32 36-54 5′-TCCGGTTCTGAAGGTGTTC-3′ SEQ ID NO: 33 36-55 5′-CTCCGGTTCTGAAGGTGTTC-3′ SEQ ID NO: 34 36-56 5′-CCTCCGGTTCTGAAGGTGTTC-3′ SEQ ID NO: 35 36-57 5′-GCCTCCGGTTCTGAAGGTGTTC-3′ SEQ ID NO: 36 36-58 5′-TGCCTCCGGTTCTGAAGGTGTTC-3′ SEQ ID NO: 37
The modification for the other positions of the sugar includes, for example, replacement of 0 at the 4′ position of ribose or deoxyribose with S, bridging between 2′ and 4′ positions of the sugar, e.g., LNA (locked nucleic acid) or ENA (2′-O,4′-C-ethylene-bridged nucleic acids), but is not limited thereto.
Under argon atmosphere, 22.0 g of N-{1-[(2R,6S)-6-(hydroxymethyl)-4-tritylmorpholin-2-yl]-2-oxo-1,2-dihydropyrimidin-4-yl}benzamide and 7.04 g of 4-dimethylaniinopyridine (4-DMAP) were suspended in 269 mL of dichloromethane, and 5.76 g of succinic anhydride was added to the suspension, followed by stirring at room temperature for 3 hours. To the reaction solution was added 40 mL of methanol, and the mixture was concentrated under reduced pressure. The residue was extracted using ethyl acetate and 0.5M aqueous potassium dihydrogenphosphate solution. The resulting organic layer was washed sequentially with 0.5M aqueous potassium dihydrogenphosphate solution, water and brine in the order mentioned. The resulting organic layer was dried over sodium sulfate and concentrated under reduced pressure to give 25.9 g of the product.
Reference Example 2 4-Oxo-4-{[(2S,6R)-6-(6-oxo-2-[2-phenoxyacetamido]-1H-purin-9-yl)-4-tritylmorphotin-2-yl]methoxy}butanoic acid loaded onto 2-aminomethylpolystyrene resin Step 1: Production of N2-(phenoxyacetyl)guanosine
Guanosine, 100 g, was dried at 80° C. under reduced pressure for 24 hours. After 500 mL of pyridine (anhydrous) and 500 mL of dichloromethane (anhydrous) were added thereto, 401 mL of chlorotrimethylsilane was dropwise added to the mixture under an argon atmosphere at 0° C., followed by stirring at room temperature for 3 hours. The mixture was again ice-cooled and 66.3 g of phenoxyacetyl chloride was dropwise added thereto. Under ice cooling, the mixture was stirred for further 3 hours. To the reaction solution was added 500 mL of methanol, and the mixture was stirred at room temperature overnight. The solvent was then removed by distillation under reduced pressure. To the residue was added 500 mL of methanol, and concentration under reduced pressure was performed 3 times. To the residue was added 4 L of water, and the mixture was stirred for an hour under ice cooling. The precipitates formed were taken out by filtration, washed sequentially with water and cold methanol and then dried to give 150.2 g of the objective compound (yield: 102%) (cf. Org. Lett. (2004), Vol. 6, No. 15, 2555-2557).
Forward primer: 5′-AGGATTTGGAACAGAGGCGTC-3′ (SEQ ID NO: 40)
Reverse primer: 5′-GTCTGCCACTGGCGGAGGTC-3′ (SEQ ID NO: 41)
Forward primer: 5′-CATCAAGCAGAAGGCAACAA-3′ (SEQ ID NO: 42)
Reverse primer: 5′-GAAGTTTCAGGGCCAAGTCA-3′ (SEQ ID NO: 43)
hEX51F: 5′-CGGGCTTGGACAGAACTTAC-3′ (SEQ ID NO: 45)
hEx55R: 5′-TCCTTACGGGTAGCATCCTG-3′ (SEQ ID NO: 46)
hEx44F: 5′-TGTTGAGAAATGGCGGCGT-3′ (SEQ ID NO: 48)
TABLE 7 Antisense SEQ ID oligomer Nucleotide sequence NO:
1. A phosphorodiamidate morpholino oligomer (PMO) consisting of a 25-mer antisense oligomer that is 100% complementary, according to Watson-Crick base pairing, to the 36th to the 60th nucleotides from the 5′ end of the 53rd exon in a human dystrophin pre-mRNA, wherein the 53rd exon in said human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO: 1, wherein each phosphorodiamidate morpholino monomer of said PMO has the formula:
wherein each of R2 and R3 represents a methyl;
wherein Base is a nucleobase selected from the group consisting of cytosine, thymine, adenine, and guanine, and
wherein the 5′ end of said PMO has a formula selected from the group consisting of:
2. A phosphorodiamidate morpholino oligomer (PMO) consisting of a 25-mer antisense oligomer that is 100% complementary, according to Watson-Crick base pairing, to the 36th to the 60th nucleotides from the 5′ end of the 53rd exon in a human dystrophin pre-mRNA, wherein the 53rd exon in said human dystrophin pre-mRNA consists of a nucleotide sequence corresponding to SEQ ID NO: 1, wherein each phosphorodiamidate morpholino monomer of said PMO has the formula:
wherein Base is a nucleobase selected from the group consisting of uracil, cytosine, thymine, adenine, and guanine; and
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Patent Publication Number: 20190218244
Assignees: NIPPON SHINYAKU CO., LTD. (Kyoto-shi, Kyoto), NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY (Kodaira-shi, Tokyo)
Inventors: Naoki Watanabe (Tsukuba), Youhei Satou (Tsukuba), Shin'ichi Takeda (Kodaira), Tetsuya Nagata (Kodaira)
Application Number: 16/369,427
International Classification: C07H 21/04 (20060101); C12N 15/113 (20100101); C12N 15/11 (20060101); C07H 21/00 (20060101); C12N 5/00 (20060101);