Claims for Patent: 10,351,854

Summary for Patent: 10,351,854

Title:	Therapeutic inhibition of lactate dehydrogenase and agents therefor
Abstract:	This invention relates to compounds, compositions, and methods useful for reducing lactate dehydrogenase target RNA and protein levels via use of dsRNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.
Inventor(s):	Brown Bob D., Dudek Henryk T., Lai Cheng
Assignee:	Dicerna Pharmaceuticals, Inc.
Application Number:	US15518009
Patent Claims:	2. The dsNA of claim 1 , wherein the sense strand and antisense strand form a duplex such that the antisense strand comprises a two nucleotide overhang at its 3′ end.3. The dsNA of claim 1 , wherein the dsNA comprises a duplex of 19 to 25 base pairs in length.4. The dsNA of claim 2 , wherein the sense strand is 25 to 53 nucleotides in length.5. The dsNA of claim 2 , wherein the sense strand is 36 nucleotides in length and/or wherein the antisense strand is 22 nucleotides in length.6. The dsNA of claim 1 , wherein the sense strand and the antisense strand are each in the range of 21 to 23 nucleotides in length.7. The dsNA of claim 1 , wherein the sense strand is 21 nucleotides in length and the antisense strand is 23 nucleotides in length.8. The dsNA of claim 7 , wherein the 3′ terminus of the sense strand and the 5′ terminus of the antisense strand form a blunt end.9. The dsNA of claim 1 , wherein the sense strand comprises a tetraloop at its 3′ end.10. The dsNA of claim 6 , wherein the sense strand comprises a tetraloop at its 3′ end.11. The dsNA of claim 9 , wherein the tetraloop comprises a single stranded loop having a sequence of GAAA.12. The dsNA of claim 1 , wherein the dsNA comprises at least one phosphorothioate linkage.13. The dsNA of claim 11 , wherein the dsNA comprises at least one phosphorothioate linkage.14. The dsNA of claim 1 , wherein the one or more modified nucleotides contains a 2′ modification.15. The dsNA of claim 14 , wherein the one or more modified nucleotides are selected from a 2′-O-methyl nucleotide and a 2′-fluoro nucleotide.16. The dsNA of claim 15 , wherein the dsNA comprises at least one 2′-fluoro nucleotide and at least one 2′-O-methyl nucleotide.17. The dsNA of claim 1 , wherein the dsNA is attached to a moiety selected from the group consisting of a GalNAc moiety claim 1 , a cholesterol moiety claim 1 , and a cholesterol targeting ligand.18. The dsNA of claim 15 , wherein the dsNA is attached to a GalNAc moiety.19. A pharmaceutical composition comprising the dsNA of and a pharmaceutically acceptable carrier.21. A mammalian cell containing the nucleic acid of .22. A pharmaceutical composition comprising the nucleic acid of and a pharmaceutically acceptable carrier.24. The dsNA of claim 23 , wherein the complementary region of the antisense strand contains three claim 23 , two claim 23 , or one mismatched nucleotide residues relative to SEQ ID NO: 401.25. The dsNA of claim 23 , wherein the sense strand and antisense strand form a duplex such that the antisense strand comprises a two nucleotide overhang at its 3′ end.26. The dsNA of claim 23 , wherein the dsNA comprises a duplex of 19 to 25 base pairs in length.27. The dsNA of claim 25 , wherein the sense strand is 25 to 53 nucleotides in length.28. The dsNA of claim 25 , wherein the sense strand is 36 nucleotides in length and/or wherein the antisense strand is 22 nucleotides in length.29. The dsNA of claim 23 , wherein the sense strand and the antisense strand are each in the range of 21 to 23 nucleotides in length.30. The dsNA of claim 23 , wherein the sense strand is 21 nucleotides in length and the antisense strand is 23 nucleotides in length.31. The dsNA of claim 30 , wherein the 3′ terminus of the sense strand and the 5′ terminus of the antisense strand form a blunt end.32. The dsNA of claim 23 , wherein the sense strand comprises a tetraloop at its 3′ end.33. The dsNA of claim 29 , wherein the sense strand comprises a tetraloop at its 3′ end.34. The dsNA of claim 32 , wherein the tetraloop comprises a single stranded loop having a sequence of GAAA.