Claims for Patent: 10,385,341
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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): | Swayze Eric E. |
| Assignee: | Biogen MA Inc. |
| Application Number: | US15301004 |
| Patent Claims: | 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); whereinA=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, ando=a phosphodiester internucleoside linkage,or a salt thereof.3. A composition comprising the antisense compound or salt thereof of and at least one of a pharmaceutically acceptable carrier or diluent.4. A composition comprising the salt of the antisense compound of 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 .10. The salt of the antisense compound of .11. A composition comprising the antisense compound of 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 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.18. A composition comprising the antisense compound or salt thereof of 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 .22. A composition comprising the antisense compound of 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. |
