发明名称 |
PEPTIDE OLIGONUCLEOTIDE CONJUGATES |
摘要 |
Oligonucleotide analogues conjugated to carrier peptides are provided. The disclosed compounds are useful for the treatment of various diseases, for example diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects. |
申请公布号 |
US2016237426(A1) |
申请公布日期 |
2016.08.18 |
申请号 |
US201514851434 |
申请日期 |
2015.09.11 |
申请人 |
SAREPTA THERAPEUTICS, INC. |
发明人 |
Hanson Gunnar J. |
分类号 |
C12N15/113;A61K47/48;A61K31/712 |
主分类号 |
C12N15/113 |
代理机构 |
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代理人 |
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主权项 |
1. A conjugate comprising:
(a) a carrier peptide comprising amino acid subunits, the carrier peptide comprising a proline amino acid at the carboxy terminus of the carrier peptide; and (b) a nucleic acid analogue comprising a substantially uncharged backbone and a targeting base sequence for sequence-specific binding to a target nucleic acid; and (c) a covalent attachment between the nucleic acid analog and the carrier peptide, the covalent attachment comprising the carboxy-terminal proline and an optional linker group;wherein:
two or more of the amino acid subunits are positively charged amino acids, no more than seven contiguous amino acid subunits are arginine, the carrier peptide is covalently attached to the nucleic acid analogue, and the covalent attachment between the nucleic acid analog and the carrier peptide is not 6-aminohexanoic acid or β-alanine. |
地址 |
Corvallis OR US |