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US 2016/0237426 A1Published application
Peptide oligonucleotide conjugates
AssigneeSarepta Therapeutics, Inc.
InventorGunnar J. Hanson
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Abstract
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.
Claims33
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.
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Dependencies are inferred from the claim text.