Claims for Patent: 8,653,119

Summary for Patent: 8,653,119

Title:	Methods for treating transthyretin amyloid diseases
Abstract:	Kinetic stabilization of the native state of transthyretin is an effective mechanism for preventing protein misfolding. Because transthyretin misfolding plays an important role in transthyretin amyloid diseases, inhibiting such misfolding can be used as an effective treatment or prophylaxis for such diseases. Treatment methods are disclosed.
Inventor(s):	Kelly; Jeffery W. (La Jolla, CA), Powers; Evan T. (La Jolla, CA), Razavi; Hossein (Danbury, CT)
Assignee:	The Scripps Research Institute (La Jolla, CA)
Application Number:	13/303,060
Patent Claims:	1. A method of treating a transthyretin amyloid disease, comprising administering to a subject in need thereof a therapeutically effective amount of a pharmaceutically acceptable salt of 6-Carboxy-2-(3,5-dichlorophenyl)-benzoxazole. 2. The method of claim 1, wherein the pharmaceutically acceptable salt of 6-Carboxy-2-(3,5-dichlorophenyl)-benzoxazole is an N-methyl-D-glucamine salt of 6-Carboxy-2-(3,5-dichlorophenyl)-benzoxazole. 3. A method of treating a transthyretin amyloid disease, comprising administering to a subject in need thereof a therapeutically effective amount of a pharmaceutical composition comprising a pharmaceutically acceptable salt of 6-Carboxy-2-(3,5-dichlorophenyl)-benzoxazole and a pharmaceutically acceptable carrier. 4. The method of claim 3, wherein the pharmaceutically acceptable salt of 6-Carboxy-2-(3,5-dichlorophenyl)-benzoxazole is an N-methyl-D-glucamine salt of 6-Carboxy-2-(3,5-dichlorophenyl)-benzoxazole. 5. The method of claim 1, wherein the transthyretin amyloid disease is familial amyloid polyneuropathy, familial amyloid cardiomyopathy, senile systemic amyloidosis, or cardiac amyloidosis following liver transplantation. 6. The method of claim 2, wherein the transthyretin amyloid disease is familial amyloid polyneuropathy, familial amyloid cardiomyopathy, senile systemic amyloidosis, or cardiac amyloidosis following liver transplantation. 7. The method of claim 3, wherein the transthyretin amyloid disease is familial amyloid polyneuropathy, familial amyloid cardiomyopathy, senile systemic amyloidosis, or cardiac amyloidosis following liver transplantation. 8. The method of claim 4, wherein the transthyretin amyloid disease is familial amyloid polyneuropathy, familial amyloid cardiomyopathy, senile systemic amyloidosis, or cardiac amyloidosis following liver transplantation. 9. The method of claim 1, wherein the transthyretin amyloid disease is familial amyloid cardiomyopathy. 10. The method of claim 2, wherein the transthyretin amyloid disease is familial amyloid cardiomyopathy. 11. The method of claim 3, wherein the transthyretin amyloid disease is familial amyloid cardiomyopathy. 12. The method of claim 4, wherein the transthyretin amyloid disease is familial amyloid cardiomyopathy. 13. The method of claim 1, wherein the transthyretin amyloid disease is senile systemic amyloidosis. 14. The method of claim 2, wherein the transthyretin amyloid disease is senile systemic amyloidosis. 15. The method of claim 3, wherein the transthyretin amyloid disease is senile systemic amyloidosis. 16. The method of claim 4, wherein the transthyretin amyloid disease is senile systemic amyloidosis. 17. The method of claim 1, wherein the transthyretin amyloid disease is cardiac amyloidosis following liver transplantation. 18. The method of claim 2, wherein the transthyretin amyloid disease is cardiac amyloidosis following liver transplantation. 19. The method of claim 3, wherein the transthyretin amyloid disease is cardiac amyloidosis following liver transplantation. 20. The method of claim 4, wherein the transthyretin amyloid disease is cardiac amyloidosis following liver transplantation. 21. The method of claim 1, wherein the transthyretin amyloid disease is familial amyloid polyneuropathy. 22. The method of claim 2, wherein the transthyretin amyloid disease is familial amyloid polyneuropathy. 23. The method of claim 3, wherein the transthyretin amyloid disease is familial amyloid polyneuropathy. 24. The method of claim 4, wherein the transthyretin amyloid disease is familial amyloid polyneuropathy.