Peptide-conjugated oligonucleotide therapeutic and method of making and using same
AssigneeSloan-Kettering Institute for Cancer Research
InventorsRobert Benezra, Erik Henke
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Abstract
Conjugates for the efficient delivery of sequence-specific antisense to cells of a selected type for the inhibition of a target protein have the general formula: peptide-HBL-antisense in which the peptide is a homing peptide which directs the conjugate to cells of a particular type, antisense is an antisense oligonucleotide having a sequence selected to provide sequence-specific inhibition of the target protein, and HBL is a heterobifunctional linker having reactivity towards amino and sulfhydryl groups.
Claims25
A peptide-oligonucleotide conjugate having the general formula:
- homing element-GMBS-antisense
- in which homing element is a means for directing the conjugate to cells of a particular type, antisense is an antisense gapmer oligonucleotide having a sequence selected to provide sequence-specific inhibition of a target protein, and GMBS is a linking group formed by reaction of 4-maleimidobutyric acid N-hydroxysuccinimide ester with the homing element and the antisense.
A peptide-oligonucleotide conjugate having the general formula:
- peptide-HBL-antisense
- in which peptide is a homing peptide which directs the conjugate to cells of a particular type, antisense is an antisense oligonucleotide having a sequence selected to provide sequence-specific inhibition of a target protein, and HBL is a heterobifunctional linker having reactivity towards amino and sulfhydryl groups, in which the peptide directs the conjugate to tumor endothelium, and in which the antisense oligonucleotide comprises the Seq ID No. 1 or 31 or an RNA counter-part thereof.
A peptide-oligonucleotide conjugate having the general formula:
- peptide-HBL-antisense
- in which peptide is a homing peptide which directs the conjugate to cells of a particular type, antisense is an antisense oligonucleotide having a sequence selected to provide sequence-specific inhibition of a target protein, and HBL is a heterobifunctional linker having reactivity towards amino and sulfhydryl groups, in which the antisense oligonucleotide comprises Seq. ID No. 1 or 31 or an RNA counter-part thereof.
Dependencies are inferred from the claim text.