IP Library › Granted Patent US 10,407,461
Granted Patent B2
US 10,407,461 · App. 16/364,451 · Granted Sep 10, 2019

Antisense nucleic acids

Inventors: Naoki Watanabe (Tsukuba, JP); Youhei Satou (Tsukuba, JP); Shin'ichi Takeda (Kodaira, JP); Tetsuya Nagata (Kodaira, JP)
Assignees: NIPPON SHINYAKU CO., LTD.; NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY
C07H21/04C07H21/00C12N15/111C12N15/113C12N2310/11C12N2310/315C12N2310/3145C12N2310/321C12N2310/3525C12N2320/33
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Quick Facts
Patent No.
US 10,407,461
App. No.
16/364,451
Granted
Sep 10, 2019
Kind
B2
Abstract

The present invention provides an oligomer which efficiently enables to cause skipping of the 53rd exon in the human dystrophin gene. Also provided is a pharmaceutical composition which causes skipping of the 53rd exon in the human dystrophin gene with a high efficiency.

Claims (7)

1. A phosphorodiamidate morpholino oligomer (PMO) antisense oligomer that causes skipping of the 53 rd exon in a human dystrophin pre-mRNA, consisting of a 25-mer oligomer that is 100% complementary to the target sequence 5′-GAACACCUUCAGAACCGGAGGCAAC-3′ (SEQ ID NO: 124) of said human dystrophin pre-mRNA, wherein said PMO antisense oligomer hybridizes to said target sequence with Watson-Crick base pairing under physiological conditions, wherein each phosphorodiamidate morpholino monomer of said PMO antisense oligomer has the formula:

wherein each of R 2 and R 3 represents a methyl; and

wherein Base is a nucleobase selected from the group consisting of uracil, cytosine, thymine, adenine, and guanine.

2. A phosphorodiamidate morpholino oligomer (PMO) antisense oligomer that causes skipping of the 53 rd exon in a human dystrophin pre-mRNA, consisting of a 25-mer oligomer that is 100% complementary to the target sequence 5′-GAACACCUUCAGAACCGGAGGCAAC-3′ (SEQ ID NO: 124) of said human dystrophin pre-mRNA, wherein said PMO antisense oligomer hybridizes to said target sequence with Watson-Crick base pairing under physiological conditions, wherein each phosphorodiamidate morpholino monomer of said PMO antisense oligomer has the formula:

wherein each of R 2 and R 3 represents a methyl;

wherein Base is a nucleobase selected from the group consisting of uracil, cytosine, thymine, adenine, and guanine; and

wherein the 5′ end of said PMO antisense oligomer has the formula:

Assignments (2)
CORRECTIVE ASSIGNMENT TO CORRECT THE RECEIVING PARTY POSTAL CODE PREVIOUSLY RECORDED AT REEL: 486999 FRAME: 218. ASSIGNOR(S) HEREBY CONFIRMS THE ASSIGNMENT. Recorded Apr 17, 2019
From: WATANABE, NAOKI; SATOU, YOUHEI; TAKEDA, SHIN'ICHI; NAGATA, TETSUYA
To: NIPPON SHINYAKU CO., LTD.; NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY
Reel/Frame 050175/0156 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 26, 2019
From: NAGATA, TETSUYA; WATANABE, NAOKI; SATOU, YOUHEI; TAKEDA, SHIN'ICHI
To: NIPPON SHINYAKU CO., LTD.; NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY
Reel/Frame 048699/0218 →
Priority Claims (1)
JP 2010-196032 · Sep 1, 2010 · national
Continuity (4)
Continuation 15619996 · Jun 12, 2017
Continuation 14615504 · Feb 6, 2015
Continuation 13819520
Related Publication 20190211050A1 · Jul 11, 2019
Cited By (13)
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