US2017369876A1PendingUtilityA1
Exon skipping compositions for treating muscular dystrophy
Est. expiryMar 14, 2033(~6.7 yrs left)· nominal 20-yr term from priority
A61P 43/00A61P 21/00A61P 21/04C12N 2310/3233C12N 2310/3513C12N 2310/351A61K 47/60C12N 15/113C12N 2310/33C12N 15/111C12N 2320/33C12N 2310/11C12N 2310/3535A61K 31/713A61K 47/6455C12N 2320/30A61K 31/7088C12N 15/11Y02A50/30
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Claims
Abstract
Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.
Claims
exact text as granted — not AI-modified1 . An antisense oligonucleotide of 22 bases comprising a base sequence that is 100% complementary to a target region of exon 44 of the human dystrophin pre-mRNA, wherein the target region is the annealing site H44A(−07+15), wherein the antisense oligonucleotide is a morpholino antisense oligonucleotide, and wherein the antisense oligonucleotide induces exon 44 skipping;
or a pharmaceutically acceptable salt thereof.
2 . An isolated antisense oligonucleotide of 22 bases comprising the base sequence GAT CTG TCA AAT CGC CTG CAG G (SEQ ID NO: 5), in which thymine bases are optionally uracil bases;
or a pharmaceutically acceptable salt thereof.
3 - 7 . (canceled)
8 . The antisense oligonucleotide of claim 1 , wherein the oligonucleotide is chemically linked to a polyethylene glycol molecule.
9 . The antisense oligonucleotide of claim 2 , wherein the antisense oligonucleotide is chemically linked to a polyethylene glycol molecule.
10 - 15 . (canceled)
16 . An antisense oligonucleotide having the following structure:
wherein
and wherein ̂=the stereochemistry of the phosphorous center is not defined.
17 - 20 . (canceled)
21 . The antisense oligonucleotide of claim 1 , wherein the antisense oligonucleotide is conjugated to a triethyleneglycol moiety via a piperazinyl moiety.
22 . The antisense oligonucleotide of claim 2 , wherein the antisense oligonucleotide is conjugated to a triethyleneglycol moiety via a piperazinyl moiety.
23 . The antisense oligonucleotide of claim 1 , wherein thymine bases are uracil bases.
24 . The antisense oligonucleotide of claim 2 , wherein thymine bases are uracil bases.
25 . The antisense oligonucleotide of claim 8 , wherein thymine bases are uracil bases.
26 . The antisense oligonucleotide of claim 9 , wherein thymine bases are uracil bases.
27 . The antisense oligonucleotide of claim 16 , wherein thymine bases are uracil bases.
28 . The antisense oligonucleotide of claim 21 , wherein thymine bases are uracil bases.
29 . The antisense oligonucleotide of claim 22 , wherein thymine bases are uracil bases.
30 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 1 and a pharmaceutically acceptable carrier.
31 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 2 and a pharmaceutically acceptable carrier.
32 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 8 and a pharmaceutically acceptable carrier.
33 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 9 and a pharmaceutically acceptable carrier.
34 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 16 and a pharmaceutically acceptable carrier.
35 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 21 and a pharmaceutically acceptable carrier.
36 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 22 and a pharmaceutically acceptable carrier.
37 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 23 and a pharmaceutically acceptable carrier.
38 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 24 and a pharmaceutically acceptable carrier.
39 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 25 and a pharmaceutically acceptable carrier.
40 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 26 and a pharmaceutically acceptable carrier.
41 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 27 and a pharmaceutically acceptable carrier.
42 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 28 and a pharmaceutically acceptable carrier.
43 . A pharmaceutical composition comprising the antisense oligonucleotide of claim 29 and a pharmaceutically acceptable carrier.
44 . The pharmaceutical composition of claim 30 for use in the treatment of Duchenne muscular dystrophy (DMD).
45 . The pharmaceutical composition of claim 31 for use in the treatment of Duchenne muscular dystrophy (DMD).
46 . The pharmaceutical composition of claim 32 for use in the treatment of Duchenne muscular dystrophy (DMD).
47 . The pharmaceutical composition of claim 33 for use in the treatment of Duchenne muscular dystrophy (DMD).
48 . The pharmaceutical composition of claim 34 for use in the treatment of Duchenne muscular dystrophy (DMD).
49 . The pharmaceutical composition of claim 35 for use in the treatment of Duchenne muscular dystrophy (DMD).
50 . The pharmaceutical composition of claim 36 for use in the treatment of Duchenne muscular dystrophy (DMD).
51 . The pharmaceutical composition of claim 37 for use in the treatment of Duchenne muscular dystrophy (DMD).
52 . The pharmaceutical composition of claim 38 for use in the treatment of Duchenne muscular dystrophy (DMD).
53 . The pharmaceutical composition of claim 39 for use in the treatment of Duchenne muscular dystrophy (DMD).
54 . The pharmaceutical composition of claim 40 for use in the treatment of Duchenne muscular dystrophy (DMD).
55 . The pharmaceutical composition of claim 41 for use in the treatment of Duchenne muscular dystrophy (DMD).
56 . The pharmaceutical composition of claim 42 for use in the treatment of Duchenne muscular dystrophy (DMD).
57 . The pharmaceutical composition of claim 43 for use in the treatment of Duchenne muscular dystrophy (DMD).Cited by (0)
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