Claims for Patent: 10,669,546
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Summary for Patent: 10,669,546
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: | US16513297 |
Patent Claims: | 2. The method of claim 1 , wherein the SOD1 associated neurodegenerative disorder is SOD1 associated amyotrophic lateral sclerosis (ALS).3. The method of claim 1 , wherein the antisense compound or the pharmaceutically acceptable salt is administered intrathecally.4. The method of claim 1 , wherein the pharmaceutically acceptable salt is a sodium salt.5. The method of claim 1 , wherein the method comprises administering to the human subject a therapeutically effective amount of the antisense compound.6. The method of claim 5 , wherein the antisense compound is administered intrathecally.8. The method of claim 7 , wherein the SOD1 associated neurodegenerative disorder is SOD1 associated amyotrophic lateral sclerosis (ALS).9. The method of claim 7 , wherein the antisense compound or the pharmaceutically acceptable salt thereof is administered intrathecally.10. The method of claim 7 , wherein the pharmaceutically acceptable salt is a sodium salt.11. The method of claim 7 , wherein the method comprises administering to the human subject a therapeutically effective amount of the antisense compound.12. The method of claim 11 , wherein the antisense compound is administered intrathecally.15. The method of claim 13 , wherein the human subject has SOD1 associated amyotrophic lateral sclerosis (ALS).16. The method of claim 13 , wherein the antisense compound or the pharmaceutically acceptable salt thereof is administered intrathecally.17. The method of claim 13 , wherein the pharmaceutically acceptable salt is a sodium salt.18. The method of claim 13 , wherein the method comprises administering to the human subject a therapeutically effective amount of the antisense compound.19. The method of claim 13 , wherein the antisense compound is administered intrathecally.20. The method of claim 14 , wherein the human subject has SOD1 associated amyotrophic lateral sclerosis (ALS).21. The method of claim 14 , wherein the antisense compound or the pharmaceutically acceptable salt thereof is administered intrathecally.22. The method of claim 14 , wherein the pharmaceutically acceptable salt is a sodium salt.23. The method of claim 14 , wherein the method comprises administering to the human subject a therapeutically effective amount of the antisense compound.24. The method of claim 14 , wherein the antisense compound is administered intrathecally.25. The method of claim 1 , wherein the method comprises treating SOD1 associated amyotrophic lateral sclerosis (ALS) in the human subject.26. The method of claim 25 , wherein the antisense compound or the pharmaceutically acceptable salt thereof is administered intrathecally.27. The method of claim 7 , wherein the method comprises treating SOD1 associated amyotrophic lateral sclerosis (ALS) in the human subject.28. The method of claim 27 , wherein the antisense compound or the pharmaceutically acceptable salt thereof is administered intrathecally. |