Products and compositions
AssigneeSilence Therapeutics GmbH
InventorsJudith Hauptmann, Dmitry Samarsky, Christian Frauendorf
Life of this patent
Estimating the term…
Abstract
The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with gene expression or inhibits its expression and therapeutic uses such as for the treatment of disease and disorders.
Claims17
Nucleic acid for inhibiting expression of a target gene in a cell, comprising at least one duplex region that comprises at least a portion of a first strand and at least a portion of a second strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene, wherein said first strand includes modified nucleotides at a plurality of positions, and wherein the nucleotides at positions 2 and 14 from the 5′ end of the first strand are not modified with a 2′ O-methyl modification.
A nucleic acid of any preceding claim wherein the nucleotides on the second strand corresponding to positions 11 and/or 13 from the 5′ end of the first strand are modified.
A nucleic acid according to any preceding claim wherein greater than 50% of the nucleotides of the first and/or second strand comprise a 2′ O-methyl modification, such as greater than 55%, 60%, 65%, 70%, 75%, 80%, or 85%, or more, of the first and/or second strand comprise a 2′ O-methyl modification, preferably measured as a percentage of the total nucleotides of both the first and second strands.
A nucleic acid according to any preceding claim comprising no more than 20%, (such as no more than 15% or no more than 10%) of 2′ fluoro modifications on the first and/or second strand, as a percentage of the total nucleotides of both strands.
A nucleic acid according to any preceding claim wherein the terminal nucleotide at the 3′ end of at least one of the first strand and the second strand is an inverted nucleotide and is attached to the adjacent nucleotide via the 3′ carbon of the terminal nucleotide and the 3′ carbon of the adjacent nucleotide and/or the terminal nucleotide at the 5′ end of at least one of the first strand and the second strand is an inverted nucleotide and is attached to the adjacent nucleotide via the 5′ carbon of the terminal nucleotide and the 5′ carbon of the adjacent nucleotide, or wherein the nucleic acid comprises a phosphorodithioate linkage.
Nucleic acid according to any preceding claim, conjugated with a ligand.
Dependencies are inferred from the claim text.