Rna interference in dermal and fibrotic indications
AssigneePhio Pharmaceuticals Corp.
InventorsAnastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Pamela A. Pavco, Lyn Libertine, James Cardia, Karen G. Bulock
Life of this patent
Estimating the term…
Abstract
The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal and fibrotic applications.
Claims20
1-55. (canceled)
A double-stranded ribonucleic acid (dsRNA) comprising a sense strand and an antisense strand, wherein the sense and/or antisense strand comprises at least 12 contiguous nucleotides of a sequence selected from the sequences within Tables 4, 5, 6, 7, 16, 17, 21, 22, 26, and 27, wherein the dsRNA is an sd-rxRNA, wherein the antisense strand is 16-23 nucleotides long and the sense strand is 12-15 nucleotides long, 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 antisense 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, and wherein at least 40% of the nucleotides of the double-stranded nucleic acid molecule are modified.
A method for treating or preventing a fibrotic disorder, the method comprising administering to a subject in need thereof a therapeutically effective amount of a double stranded ribonucleic acid (dsRNA) comprising a sense strand and an antisense strand, wherein the sense and/or antisense strand comprises at least 12 contiguous nucleotides of a sequence selected from the sequences within Tables 4, 5, 6, 7, 16, 17, 21, 22, 26, and 27, wherein the dsRNA is an sd-rxRNA, wherein the antisense strand is 16-23 nucleotides long and the sense strand is 12-15 nucleotides long, 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 antisense 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, and wherein at least 40% of the nucleotides of the double-stranded nucleic acid molecule are modified.
A method comprising administering to a subject in need thereof a therapeutically effective amount of a double stranded ribonucleic acid (dsRNA) comprising a sense strand and an antisense strand, wherein the sense and/or antisense strand comprises at least 12 contiguous nucleotides of a sequence selected from the sequences within Tables 4, 5, 6, 7, 16, 17, 21, 22, 26, and 27, wherein the dsRNA is an sd-rxRNA, wherein the antisense strand is 16-23 nucleotides long and the sense strand is 12-15 nucleotides long, 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 antisense 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, and wherein at least 40% of the nucleotides of the double-stranded nucleic acid molecule are modified.
Dependencies are inferred from the claim text.