Claims for Patent: 10,968,453

Summary for Patent: 10,968,453

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:	US16849583
Patent Claims:	1. A method for treating a superoxide dismutase 1 (SOD1) associated neurodegenerative disorder in a human subject in need thereof , the method comprising administering to the human subject a therapeutically effective amount of a pharmaceutical composition comprising a pharmaceutically acceptable carrier or diluent; and an antisense oligonucleotide having the following formula: 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 pharmaceutically acceptable salt thereof., '5′-mCes Aeo Ges Geo Aes Tds Ads mCds Ads Tds Tds Tds mCds Tds Ads mCeo Aes Geo mCes Te -3′ (nucleobase sequence of SEQ ID NO: 725); wherein,'}2. The method of claim 1 , wherein the pharmaceutically acceptable carrier or diluent is a sterile aqueous solution.3. The method of claim 1 , wherein the pharmaceutically acceptable carrier or diluent is phosphate buffered saline (PBS).4. The method of claim 1 , wherein the pharmaceutical composition is administered intrathecally.5. The method of claim 1 , wherein the SOD1 associated neurodegenerative disorder is amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene.6. The method of claim 5 , wherein the mutation is a missense mutation.7. The method of claim 5 , wherein the mutation is a gain of function mutation.8. The method of claim 5 , wherein the ALS is familial SOD1 associated ALS.9. The method of claim 5 , wherein the ALS is sporadic SOD1 associated ALS.10. The method of claim 5 , wherein the pharmaceutical composition is administered intrathecally.11. The method of claim 8 , wherein the pharmaceutical composition is administered intrathecally.12. The method of claim 9 , wherein the pharmaceutical composition is administered intrathecally.14. The method of claim 13 , wherein the pharmaceutically acceptable carrier or diluent is a sterile aqueous solution.15. The method of claim 13 , wherein the pharmaceutically acceptable carrier or diluent is PBS.16. The method of claim 13 , wherein the pharmaceutical composition is administered intrathecally.17. The method of claim 13 , wherein the SOD1 associated neurodegenerative disorder is amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene.18. The method of claim 17 , wherein the mutation is a missense mutation.19. The method of claim 17 , wherein the mutation is a gain of function mutation.20. The method of claim 17 , wherein the ALS is familial SOD1 associated ALS.21. The method of claim 17 , wherein the ALS is sporadic SOD1 associated ALS.22. The method of claim 17 , wherein the pharmaceutical composition is administered intrathecally.23. The method of claim 20 , wherein the pharmaceutical composition is administered intrathecally.24. The method of claim 21 , wherein the pharmaceutical composition is administered intrathecally.