IP Library › Patent Application 16178129
Patent Application
App. No. 16/178,129

COMPOSITIONS AND METHODS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY

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Patent No.
US None
App. No.
16/178,129
Abstract

Disclosed herein are compounds, compositions and methods for treatment of diseases and disorders, including spinal muscular atrophy.

Claims (21)

1 - 70 . (canceled)

71 . A method of modulating the GF/IGF-1 axis, comprising administering at least one GF/IGF-1 axis modulator, wherein the GF/IGF-1 axis modulator is a GF/IGF-1 axis molecule selected from among: IGF-1 and insulin-like growth factor binding acid labile subunit (IGFALS), to a human subject having spinal muscular atrophy (SMA).

72 . The method of claim 71 , wherein at least one GF/IGF-1 axis modulator is IGF-binding-protein acid labile subunit (IGFALS).

73 . The method of claim 71 , wherein at least one GF/IGF-1 axis modulator is IGF-1.

74 . The method of claim 71 , wherein the at least one GF/IGF-1 axis modulator is administered systemically.

75 . The method of claim 71 , wherein at least one growth hormone axis modulator is administered by intraperitoneal injection.

76 . The method of claim 71 , wherein at least one growth hormone axis modulator is administered by subcutaneous injection.

77 . The method of claim 71 , wherein at least one growth hormone axis modulator is administered by intramuscular injection.

78 . The method of claim 71 , wherein at least one growth hormone axis modulator is administered into the cerebrospinal fluid.

79 . The method of claim 71 , comprising administering at least one antisense oligonucleotide to the subject having spinal muscular atrophy.

80 . The method of claim 79 , wherein the antisense compound comprises an antisense oligonucleotide complementary to a nucleic acid encoding human SMN2.

81 . The method of claim 80 , wherein the oligonucleotide is complementary to a portion of intron 7 of the nucleic acid encoding human SMN2.

82 . The method of claim 80 , wherein the antisense oligonucleotide is at least 90% complementary to the nucleic acid encoding human SMN2.

83 . The method of claim 80 , wherein the antisense oligonucleotide is fully complementary to the nucleic acid encoding human SMN2.

84 . The method of claim 80 , wherein the oligonucleotide has a nucleobase sequence comprising at least 10 contiguous nucleobases of the nucleobase sequence SEQ ID NO: 1.

85 . The method of claim 80 , wherein the oligonucleotide has a nucleobase sequence comprising at least 15 contiguous nucleobases of the nucleobase sequence SEQ ID NO: 1.

86 . The method of claim 80 , wherein the oligonucleotide has a nucleobase sequence comprising the nucleobase sequence SEQ ID NO: 1.

87 . The method of claim 80 , wherein the oligonucleotide has a nucleobase sequence consisting of the nucleobase sequence SEQ ID NO: 1.

88 . The method of claim 80 , wherein at least one nucleoside of the antisense oligonucleotide comprises a modified sugar moiety.

89 . The method of claim 88 , wherein the at least one modified sugar moiety comprises a 2′-methoxyethyl sugar moiety.

90 . The method of claim 88 , wherein essentially each nucleoside of the antisense oligonucleotide comprises a modified sugar