Reduced size self-delivering rnai compounds
AssigneePhio Pharmaceuticals Corp.
InventorsAnastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, James Cardia
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Abstract
The present invention relates to methods for in vivo administration of sd-rxRNA molecules.
Claims29
An sd-rxRNA® comprising a guide strand and a passenger strand, wherein the sd-rxRNA® includes a double stranded region and a single stranded region, wherein the double stranded region is from 8-15 nucleotides long, wherein the single stranded region is at the 3′ end of the guide strand and is 4-12 nucleotides long, wherein the single stranded region contains 3, 4, 5, 6, 7, 8, 9, 10, 11, or 12 phosphorothioate modifications, wherein at least 40% of the nucleotides are modified, and wherein at least two Us and/or Cs include a hydrophobic modification comprising an octyl or a pyridyl amide modification.
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A method for inhibiting the expression of a target gene of a mammal, comprising
- administering to a mammal an sd-rxRNA® in an effective amount to inhibit the expression of a target gene of the mammal, wherein the sd-rxRNA® comprises a guide strand and a passenger strand, wherein the sd-rxRNA® includes a double stranded region and a single stranded region wherein the double stranded region is from 8-15 nucleotides long, wherein the single stranded region is at the 3′ end of the guide strand and is 4-12 nucleotides long, wherein the single stranded region contains 3, 4, 5, 6, 7, 8, 9, 10, 11, or 12 phosphorothioate modifications, wherein at least 40% of the nucleotides are modified, and wherein at least two Us and/or Cs include a hydrophobic modification comprising an octyl or a pyridyl amide modification.
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Dependencies are inferred from the claim text.