Claims for Patent: 10,385,341

Summary for Patent: 10,385,341

Title:	Compositions for modulating SOD-1 expression
Abstract:	Disclosed herein are antisense compounds and methods for decreasing SOD-1 mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate SOD-1 associated diseases, disorders, and conditions. Such SOD-1 associated diseases include amyotrophic sclerosis (ALS).
Inventor(s):	Eric E. Swayze
Assignee:	Ionis Pharmaceuticals Inc , Biogen MA Inc
Application Number:	US15/301,004
Patent Claims:	1. A salt of an antisense compound, wherein the anion of the antisense compound has the following formula: 2. An antisense compound according to the following formula: mCes Aeo Ges Geo Aes Tds Ads mCds Ads Tds Tds Tds mCds Tds Ads mCeo Aes Geo mCes Te (nucleobase sequence of SEQ ID NO: 725); wherein A=an adenine, mC=a 5-methylcytosine, G=a guanine, T=a thymine, e=a 2′-O-methoxyethylribose modified sugar, d=a 2′-deoxyribose sugar, s=a phosphorothioate internucleoside linkage, and o=a phosphodiester internucleoside linkage, or a salt thereof. 3. A composition comprising the antisense compound or salt thereof of claim 2 and at least one of a pharmaceutically acceptable carrier or diluent. 4. A composition comprising the salt of the antisense compound of claim 1 and at least one of a pharmaceutically acceptable carrier or diluent. 5. The composition of claim 3, wherein the pharmaceutically acceptable carrier or diluent is phosphate-buffered saline (PBS). 6. The composition of claim 4, wherein the pharmaceutically acceptable carrier or diluent is PBS. 7. The composition of claim 3, which is formulated for intrathecal administration. 8. The composition of claim 4, which is formulated for intrathecal administation. 9. The antisense compound of claim 2. 10. The salt of the antisense compound of claim 2. 11. A composition comprising the antisense compound of claim 9 and at least one of a pharmaceutically acceptable carrier or diluent. 12. The composition of claim 11, wherein the pharmaceutically acceptable carrier or diluent is PBS. 13. The composition of claim 11, which is formulated for intrathecal administration. 14. A composition comprising the salt of the antisense compound of claim 10 and at least one of a pharmaceutically acceptable carrier or diluent. 15. The composition of claim 14, wherein the pharmaceutically acceptable carrier or diluent is PBS. 16. The composition of claim 14, which is formulated for intrathecal administration. 17. An antisense compound according to the following formula: or a salt thereof. 18. A composition comprising the antisense compound or salt thereof of claim 17 and at least one of a pharmaceutically acceptable carrier or diluent. 19. The composition of claim 18, wherein the pharmaceutically acceptable carrier or diluent is PBS. 20. The composition of claim 18, which is formulated for intrathecal administration. 21. The antisense compound of claim 17. 22. A composition comprising the antisense compound of claim 21 and at least one of a pharmaceutically acceptable carrier or diluent. 23. The composition of claim 22, wherein the pharmaceutically acceptable carrier or diluent is PBS. 24. The composition of claim 22, which is formulated for intrathecal administration.