Chemically Modified saRNA Compositions and Methods of Use
AssigneeMina Therapeutics Limited
InventorsTroels Koch, Henrik Hansen, Hannah Pendergraff, Sara Aguti, Jean-Paul Desaulniers, Matthew Hammill, Ifrodet Giorgees, Joseph Ieuan Hoare
Life of this patent
Estimating the term…
Abstract
The disclosure relates to a saRNA useful in upregulating the expression of a target gene and therapeutic compositions comprising the saRNA, wherein the saRNA is chemically modified. Methods of using the saRNA and the therapeutic compositions are also provided.
Claims113
A double-stranded synthetic isolated small activating RNA (saRNA) which up-regulates the expression of a target gene, comprising an antisense strand and a sense strand, wherein each strand has 14-30 nucleotides, and wherein the sense strand and/or the antisense strand comprises an alkyl spacer.
A method of increasing the stability, reducing melting temperature (Tm), increasing activity, and/or reducing off-target effect of a small activating RNA (saRNA), wherein the saRNA up-regulates the expression of a target gene, and wherein the saRNA comprises an antisense strand and a sense strand, each strand having 14-30 nucleotides, comprising adding an alkyl spacer to the sense strand and/or the antisense strand of the saRNA.
A double-stranded synthetic isolated small activating RNA (saRNA) that up-regulates the expression of a target gene, comprising an antisense strand and a sense strand, wherein each strand consists of 14-30 nucleotides, and wherein at least one nucleotide of the sense strand and/or the antisense strand is an unlocked nucleic acid (UNA) having the structure
A method of increasing the stability, reducing melting temperature (Tm), increasing activity, and/or reducing off-target effect of a small activating RNA (saRNA), wherein the saRNA up-regulates the expression of a target gene, and wherein the saRNA comprises an antisense strand and a sense strand, each strand having 14-30 nucleotides, comprising replacing at least one nucleic acid of the sense strand and/or the antisense strand of the saRNA with an unlocked nucleic acid (UNA).
A double-stranded synthetic isolated small activating RNA (saRNA) that up-regulates the expression of a target gene, comprising an antisense strand and a sense strand, wherein each strand has 14-30 nucleotides, and wherein the sense strand and/or the antisense strand comprises a moiety that reduces the affinity between the strands.
Dependencies are inferred from the claim text.